Aurigene Pharmaceutical Services Header Aurigene Pharmaceutical Services Header

X
[{"orgOrder":0,"company":"Infinity Pharmaceuticals","sponsor":"BVF Partners L.P","pharmaFlowCategory":"D","amount":"$71.5 million","upfrontCash":"Undisclosed","newsHeadline":"Infinity Pharmaceuticals Raises $20 Million Through an Innovative Non-Dilutive Asset-Backed Financing from BVF","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Undisclosed","graph2":"Phase II"},{"orgOrder":0,"company":"Apotex Inc","sponsor":"CHIESI USA INC","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Chiesi Group and Apotex Inc. finalize agreement for acquisition of Ferriprox\u00ae (deferiprone)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"Bayer AG","sponsor":"WuXi Biologics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"WuXi Biologics and Bayer enter into acquisition agreement","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"GERMANY","productType":"Large molecule","productStatus":"Approved","date":"January 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Timber Pharmaceuticals","sponsor":"Timber Pharmaceuticals","pharmaFlowCategory":"D","amount":"$20.0 million","upfrontCash":"Undisclosed","newsHeadline":"BioPharmX And Timber Pharmaceuticals Announce Entry into Merger Agreement","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme reports positive arimoclomol data from open-label phase 2\/3 extension in Niemann-Pick disease Type C","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Protagonist Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protagonist Therapeutics Initiates Phase 2 Study of Novel Hepcidin Mimetic PTG-300 in the Treatment of Patients with Hereditary Hemochromatosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Peptide","graph2":"Phase II"},{"orgOrder":0,"company":"Escape Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ESCAPE Bio Announces Excellent Safety and Tolerability in Phase 1 Study of Oral S1P5 Receptor Agonist ESB1609","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Enterprise Therapeutics Publishes Paper on Novel Therapeutic Approach for Treatment of All Cystic Fibrosis Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Applied Therapeutics Announces Positive Topline Results of Pivotal Phase 2 ACTION-Galactosemia Study of AT-007 in Galactosemia Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AMO Pharma Announces Initiation of Planned Pivotal Clinical Trial for Myotonic Dystrophy Following $35m Fund Raise","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Prevail Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Prevail Therapeutics Provides PR001 Program Update","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IND Enabling"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Announces Positive Topline Cohort 3 Results and Improved Longer-Term Cohort 2 Results from Phase 1\/2 Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Retrotope","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Retrotope Expands its Drug Pipeline with the First Dosing of RT001 in patients with Friedreich\u2019s ataxia (FA)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"First Wave BioPharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AzurRx BioPharma To Present Interim Phase 2 Clinical Study Data for MS1819-SD, in combination with standard PERT for Cystic Fibrosis Patients with Severe Exocrine Pancreatic Insufficiency, at Biotech Showcase 2020 Conference (January 13) in San Francisco","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"Undisclosed","date":"January 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Initiates Rolling Submission of New Drug Application (NDA) to U.S. Food and Drug Administration (FDA) for Lumasiran for the Treatment of Primary Hyperoxaluria Type 1 (PH1)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioCryst to Provide Berotralstat and BCX9930 Program Updates at 38th Annual J.P. Morgan Healthcare Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Abeona Therapeutics Initiates Pivotal Phase 3 Clinical Trial Evaluating EB-101 Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase III"},{"orgOrder":0,"company":"Aeglea BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aeglea BioTherapeutics Submits CTA Application for its Novel Engineered Human Enzyme Designed to Treat Homocystinuria (ACN00177)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"IND Enabling"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Zynerba Pharmaceuticals Announces that Enrollment is Nearing Completion in Pivotal CONNECT-FX Trial in Fragile X Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Eloxx Pharmaceuticals Provides Update on Progress of Its Phase 2 Clinical Programs for ELX-02 and ERSG Pipeline at Biotech Showcase\u2122","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Xenetic Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Xenetic Biosciences (XBIO) Announces Publication of Data from Partner\\'s Phase 1\/2 Study Evaluating Program Leveraging Polyxen Platform Technology","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Mereo BioPharma Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mereo BioPharma Announces Additional Positive Data from Phase 2b ASTEROID Study of Setrusumab in Adults with Osteogenesis Imperfecta and Provides Update on Regulatory Progress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GeneTx and Ultragenyx Announce Investigational New Drug (IND) Application Active for GTX-102 in Patients with Angelman Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"IND Enabling"},{"orgOrder":0,"company":"Fortress Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fortress Biotech Announces Rare Pediatric Disease Designation for CUTX-101 for the Treatment of Menkes Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Seelos Therapeutics Announces Updates to SLS-005 (trehalose) Programs for Europe and U.S.","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Khondrion","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Khondrion announces first patients dosed in Phase IIb study of Sonlicromanol for mitochondrial diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Emerald Health Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Emerald Health Pharmaceuticals Granted Orphan Designation in Europe for Huntington\u2019s Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"IND Enabling"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Positive topline results demonstrated by olipudase alfa, first and only investigational therapy in late-stage development for acid sphingomyelinase deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Rubius Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rubius Therapeutics Announces First Patient Dosed in Phase 1b Trial of RTX-134 for the Treatment of Patients with Phenylketonuria","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ProQR Receives Rare Pediatric Disease Designation from FDA for QR-421a","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Albireo Achieves Significant Milestones for Odevixibat Across Multiple Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Passage Bio","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","amount":"$125.0 million","upfrontCash":"Undisclosed","newsHeadline":"Gene Therapy Developer Passage Bio Files for $125M IPO","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IND Enabling"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"General Atlantic","pharmaFlowCategory":"D","amount":"$98.0 million","upfrontCash":"Undisclosed","newsHeadline":"CANbridge Pharmaceuticals Completes US$98 Million Series D Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Spruce Biosciences","sponsor":"Omega Funds","pharmaFlowCategory":"D","amount":"$88.0 million","upfrontCash":"Undisclosed","newsHeadline":"Spruce Biosciences Raises $88 Million in Series B Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Imara","sponsor":"Undisclosed","pharmaFlowCategory":"D","amount":"$86.0 million","upfrontCash":"Undisclosed","newsHeadline":"NEA-founded Imara pitches mid-stage alternative for $86M IPO","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Torii Pharmaceutical","pharmaFlowCategory":"D","amount":"$42.0 million","upfrontCash":"$22.0 million","newsHeadline":"BioCryst Submits Japanese NDA for Oral, Once Daily Berotralstat","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Azafaros","sponsor":"Forbion","pharmaFlowCategory":"D","amount":"$28.0 million","upfrontCash":"Undisclosed","newsHeadline":"Azafaros Completes EUR 25 Million Series A Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Undisclosed"},{"orgOrder":0,"company":"AgeX Therapeutics","sponsor":"University of California, Irvine","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"AgeX Therapeutics to Collaborate with University of California","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Undisclosed"},{"orgOrder":0,"company":"Light Chain Bioscience","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Novimmune unit nabs Takeda milestone payment for hemophilia work","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Discovery","country":"SWITZERLAND","productType":"Large molecule","productStatus":"Undisclosed","date":"February 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Discovery"},{"orgOrder":0,"company":"Aradigm","sponsor":"Grifols International","pharmaFlowCategory":"D","amount":"$6.2 million","upfrontCash":"$3.2 million","newsHeadline":"Aradigm Announces Entering into an Asset Purchase Agreement with Grifols, S.A.","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Aptevo Therapeutics","sponsor":"Medexus Pharmaceuticals","pharmaFlowCategory":"D","amount":"$30.0 million","upfrontCash":"$30.0 million","newsHeadline":"Medexus expands US product portfolio by acquiring hematology asset IXINITY","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"February 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Inventiva Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inventiva to Present Results from its Phase IIa Odiparcil Study","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Zynerba Pharma Achieves Patient Screening Target for Zygel","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protalix Presents Key Clinical Data of Pegunigalsidase Alfa","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ISRAEL","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Moderna Announces First Patient Enrolled in mRNA-3704 Phase II","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Vaccine","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Vaccine","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Priority Review for Avalglucosidase Alfa, a Potential New Therapy for Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Regulus Therapeutics Reinitiates Multiple Ascending Dose Study of RGLS4326 for the Treatment of ADPKD","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"JCR Announces Presentations on JR-141 and JR-162 at the 16th Annual WORLDSymposium 2020","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"JAPAN","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Undisclosed","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GeneTx Announces IRB Approval to Begin Clinical Study of GTX-102 for the Treatment of Angelman Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Stealth BioTherapeutics Announces Results From Study to Evaluate Efficacy of Elamipretide","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Peptide","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Arrevus","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Arrevus Receives FDA Orphan Drug Designation for ARV-1801 (sodium fusidate)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Completes Enrollment of HELIOS-A Phase 3 Study of Vutrisiran for the Treatment of hATTR Amyloidosis with Polyneuropathy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Wave Life Sciences to Present Preclinical In Vivo and In Vitro Data for SNP3-Targeting Huntington\u2019s Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Accepts BioCryst\u2019s NDA for Oral, Once Daily Berotralstat (BCX7353) to Prevent HAE Attacks","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Chugai\u2019s Huntington\\'s disease drug, RG6042 gets Japanese orphan drug designation","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FORMA Therapeutics Announces FT-4202 Granted Fast Track Designation and Rare Pediatric Disease Designation for Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Magenta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Magenta Therapeutics Announces Updated Phase 2 Data on MGTA-456 Cell Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cyclo Therapeutics Completes Enrollment in its Phase I\/II Trial to Evaluate Trappsol\u00ae Cyclo\u2122 for Niemann-Pick Disease Type C","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Catabasis Pharmaceuticals to Present Edasalonexent at the XVIII International Conference on Duchenne and Becker Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Magenta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Magenta Therapeutics Completes Dosing in Phase 1 MGTA-145 Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Acer Therapeutics Announces ACER-001 for Urea Cycle Disorders Trial Shows Bioequivalence to BUPHENYL\u00ae","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Marinus Pharmaceuticals Completes Targeted Enrollment in Pivotal Phase 3 Study for CDKL5 Deficiency Disorder","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cyclo Therapeutics Announces Completion of Enrollment in its Extension Protocol to the Phase I Trial to Evaluate Trappsol Cyclo","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Eloxx Strengthens its Commitment to Cystic Fibrosis Through Reallocating Resources","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Translate Bio Receives FDA Fast Track Designation for MRT5005 for the Treatment of Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Zynerba Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Zynerba Pharmaceuticals Announces the Completion of Enrollment in the Pivotal CONNECT-FX Trial of Zygel\u2122 in Fragile X Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Psagot Investment House","pharmaFlowCategory":"D","amount":"$43.7 million","upfrontCash":"Undisclosed","newsHeadline":"Protalix BioTherapeutics Announces $43.7 Million in Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ISRAEL","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Imara","sponsor":"Undisclosed","pharmaFlowCategory":"D","amount":"$75.2 million","upfrontCash":"Undisclosed","newsHeadline":"A biotech tests Wall Street's roiled waters and comes away with a $75M IPO haul","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Sigilon Therapeutics","sponsor":"Canada Pension Plan Investment Board ","pharmaFlowCategory":"D","amount":"$80.3 million","upfrontCash":"Undisclosed","newsHeadline":"Sigilon Therapeutics Announces $80.3 Million Series B Financing to Advance","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Undisclosed","graph2":"IND Enabling"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"NewCo","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"FORMA Therapeutics Announces Divestiture of Select Hit Discovery Capabilities","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable ","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cyclo Therapeutics Announces Positive Feedback from FDA on its Pivotal Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Rare Pediatric Disease Designation to Stealth BioTherapeutics for Elamipretide for the Treatment of Barth Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Peptide","graph2":"Phase III"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Roche Trial of Ionis Huntington\u2019s Disease Drug Halted After Side Effects","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Acceleron Pharma","sponsor":"Not Applicable ","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Acceleron Announces Topline Results from the Phase 2 Trial of ACE-083","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable ","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Berotralstat (BCX7353) Significantly Reduced Use of Acute On-Demand Medicine in HAE Patients in APeX-2","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Not Applicable ","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GeneTx and Ultragenyx Announce First Patient Dosed in Phase 1\/2 Clinical Trial of GTX-102 in Patients with Angelman Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rhythm Pharmaceuticals Receives Orphan Drug Designation from U.S. FDA for Setmelanotide for the Treatment of Alstr\u00f6m Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Peptide","graph2":"Phase III"},{"orgOrder":0,"company":"Pharnext","sponsor":"Not Applicable ","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pharnext Receives Promising Innovative Medicine Designation for PXT3003 ","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Small molecule","productStatus":"Approved","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Eiger BioPharmaceuticals Completes Submission of New Drug Application to FDA for Lonafarnib","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cyclo Therapeutics Inc. Announces Positive Safety Profile of Its Drug Trappsol\u00ae Cyclo","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Castle Creek Biosciences","sponsor":"Fidelity Management & Research Company","pharmaFlowCategory":"D","amount":"$55.0 million","upfrontCash":"Undisclosed","newsHeadline":"Castle Creek Biosciences Announces $75 Million Investment","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase III"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"MPM Capital","pharmaFlowCategory":"D","amount":"$80.0 million","upfrontCash":"Undisclosed","newsHeadline":"ReCode Therapeutics Raises $80 Million in Oversubscribed Series A Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Large molecule","productStatus":"Undisclosed","date":"March 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Undisclosed"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"CureDuchenne Ventures","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Dyne Therapeutics Announces Equity Investment from CureDuchenne Ventures","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Large molecule","productStatus":"Undisclosed","date":"March 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Undisclosed"},{"orgOrder":0,"company":"Vaccinex","sponsor":"Keystone Capital Partners","pharmaFlowCategory":"D","amount":"$16.5 million","upfrontCash":"Undisclosed","newsHeadline":"Vaccinex Announces up to $16.5 Million in Equity Financing Agreements","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"IntraBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"IntraBio Receives FDA Fast Track Designation for Niemann-Pick Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Eloxx Pharmaceuticals Provides Update on the Impact of Novel Coronavirus (COVID-19) on Phase 2 Clinical Trials in Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Orphan Technologies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphan Technologies Receives Rare Pediatric Disease Designation from FDA for OT-58","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"SWITZERLAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Inflazome","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inzomelid completes Phase I studies and shows positive results in the treatment of Cryopyrin-Associated Periodic Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"IRELAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"APR Applied Pharma Research","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Orphan Drug Designation to APR-OD031 for the Treatment of Phenylketonuria (PKU)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"SWITZERLAND","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Undisclosed","graph2":"Preclinical"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Translate Bio Provides MRT5005 Program Updates","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Ovid Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ovid Therapeutics Announces Initial Data with Soticlestat in CDKL5 Deficiency Disorder and Dup15q Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Receives Positive Opinion on Orphan Drug Designation from the European Medicines Agency for Mitapivat","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ProQR Announces Positive Findings From an Interim Analysis in the Phase 1\/2 trial of QR-421","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Accepts IND Application for CRISPR\/Cas9-Based Sickle Cell Disease Therapeutic Candidate","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Dynacure","sponsor":"Perceptive Advisors","pharmaFlowCategory":"D","amount":"$54.6 million","upfrontCash":"Undisclosed","newsHeadline":"Dynacure Announces \u20ac50M ($55M) Series C Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Timber Pharmaceuticals","sponsor":"U.S. Food & Drug Administration ","pharmaFlowCategory":"D","amount":"$1.5 million","upfrontCash":"$0.5 million","newsHeadline":"Timber Pharmaceuticals Announces Award of Second Tranche of FDA Orphan Products Clinical Trials Grant","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"LFB","sponsor":"HEMA Biologics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"LFB Announces FDA Approval of SEVENFACT for the Treatment of Adults and Adolescents with Hemophilia A or B with Inhibitors","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Large molecule","productStatus":"Approved","date":"April 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"OxThera AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"OxThera Announces Completion of Recruitment in Phase 3 ePHex Study With Oxabact\u00ae in Patients With Primary Hyperoxaluria","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWEDEN","productType":"Large molecule","productStatus":"Undisclosed","date":"April 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Millendo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Millendo Therapeutics Announces Topline Results for Pivotal Phase 2b Study of Livoletide\r\n","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Peptide","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Completes Rolling Submission of New Drug Application to the U.S.FDA and Submits Marketing Authorization Application","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"KalVista Pharmaceuticals Provides Operational and Financial Update","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Vaccinex","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vaccinex Delivers Virtual Presentation at the Advances in Alzheimer\u2019s and Parkinson\u2019s Therapies AAT-AD\/PD\u2122 Focus Meeting 2020","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Arrowhead Pharmaceuticals Files for Regulatory Clearance to Begin Phase 1\/2a Study of ARO-ENaC for Treatment of Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IND Enabling"},{"orgOrder":0,"company":"Cyclerion Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cyclerion Updates Corporate Progress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Arcturus Therapeutics Announces Allowance of IND & Approval of Clinical Trial Application (CTA) for ARCT-810","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fulcrum Therapeutics Announces Completion of IND-enabling Safety Studies for FTX-6058 in Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"IND Enabling"},{"orgOrder":0,"company":"Proteostasis Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Proteostasis Therapeutics Announces Completion of Scientific Advice Meeting with the Dutch Medicines Evaluation Board","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Guggenheim Securities","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Arcturus Therapeutics Announces Proposed Public Offering of Common Stock","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Large molecule","productStatus":"Undisclosed","date":"April 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Undisclosed"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Receives Fast Track Designation for Vutrisiran for the Treatment of the Polyneuropathy of hATTR Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FORMA Therapeutics Receives FDA Orphan Drug Designation for FT-4202 for the Treatment of Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Catabasis Pharmaceuticals to Present at 2020 Muscular Dystrophy Association Virtual Poster Session","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Emmaus Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Emmaus Life Sciences Announces SFDA (Saudi Food & Drug Authority) Accepts Endari\u00ae Priority Review Request","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ionis and Partner Announce Enrollment Completion of Global Phase 3 GENERATION HD1 Study for Huntington's Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Proteostasis Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Proteostasis Therapeutics Announces Keystone Symposia Presentation of Organoid Study from More than 300 Adult CF Patients\n","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"FSIO","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Vertex Announces Innovative Reimbursement Agreement in Switzerland for ORKAMBI\u00ae and SYMDEKO\u00ae for Eligible Cystic Fibrosis Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"BrainVectis","sponsor":"AskBio","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"AskBio Acquires BrainVectis to Expand its Clinical Pipeline for Neurodegenerative Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Aeglea BioTherapeutics","sponsor":"J.P. Morgan Securities","pharmaFlowCategory":"D","amount":"$100.0 million ","upfrontCash":"Undisclosed","newsHeadline":"Aeglea BioTherapeutics Announces Proposed Public Offering","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","amount":"$2.0 million","upfrontCash":"Undisclosed","newsHeadline":"Cyclo Therapeutics Closes $2.0 Million Private Placement","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Applied Therapeutics Announces Full Data and Scientific Presentations from the Pivotal Phase 2 ACTION-Galactosemia Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Catalyst Biosciences Completes Phase 2b Trial of Subcutaneous Factor IX Dalcinonacog Alfa (DalcA)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Soleno Therapeutics Announces Publication Evaluating Role of Activating ATP-Sensitive Potassium Channel in Treatment of Hyperphagic Obesity","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Eiger BioPharmaceuticals Announces EMA Validation of Lonafarnib MAA for Treatment of Progeria and Progeroid Laminopathies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Italfarmaco S.p.A","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Italfarmaco Provides Update on Advanced-Stage Clinical Development Program with Givinostat in Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ITALY","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"First Wave BioPharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AzurRx BioPharma Receives Approval to Conduct Phase 2 OPTION 2 Clinical Trial of MS1819 in Cystic Fibrosis ","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Seelos Therapeutics Receives Orphan Drug Designation for SLS-005 (Trehalose) in Sanfilippo Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"GeneTx Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GeneTx and Ultragenyx Announce GTX-102 Granted Fast Track Designation by U.S. FDA for Treatment of Angelman Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Censa Pharmaceuticals","sponsor":"PTC Therapeutics","pharmaFlowCategory":"D","amount":"$366.0 million","upfrontCash":"$10.0 million","newsHeadline":"PTC Therapeutics to Acquire Censa Pharmaceuticals","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"jCyte","sponsor":"Santen Pharmaceutical","pharmaFlowCategory":"D","amount":"$252.0 million","upfrontCash":"$50.0 million ","newsHeadline":"jCyte Inc. Enters into Ex-US Licensing and Commercialization Agreement for jCell Therapy with Santen Pharmaceutical","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"New Composition of Matter Patent Allowance Extends Protection for Berotralstat in U.S. Market by Four Years to 2039","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Ovid Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ovid Therapeutics Announces Positive Topline Results from the Phase 2 ROCKET Trial of OV101 for the Treatment of Fragile X Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protalix BioTherapeutics Announces Topline Results from the BRIDGE Phase III Open-Label Trial of Pegunigalsidase Alfa in Fabry Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ISRAEL","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"DiNAQOR AG","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"BioMarin Extends Gene Therapy Leadership with DiNAQOR in a Preclinical Collaboration and License Agreement","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"SWITZERLAND","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IND Enabling"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AVROBIO to Present New Clinical and Preclinical Data at Upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Unveils New Strategic Plan","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"LogicBio Therapeutics to Present New Data on GeneRide\u2122 Platform and Next Generation Capsid Development Program at ASGCT 2020\r\n","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IND Enabling"},{"orgOrder":0,"company":"Timber Pharmaceuticals","sponsor":"BioPharmX","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Timber Pharmaceuticals Announces Merger Closing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Aptevo Therapeutics","sponsor":"Medexus Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Medexus Provides an Update on the IXINITY\u00ae Acquisition and its Overall Business in the COVID-19 Environment","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Vanda Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vanda Pharmaceuticals Inks Agreement with FDA on Resubmission of the Application for HETLIOZ to Treat Patients with Smith-Magenis Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rhythm Pharmaceuticals Gets FDA Acceptance of New Drug Application for Setmelanotide for the Treatment of POMC and LEPR Deficiency Obesities","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Rezolute","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rezolute, Inc. Announces Updates to Clinical and Business Operations in Response to COVID-19 Pandemic","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Seelos Therapeutics Receives Rare Pediatric Disease Designation for SLS-005 (Trehalose) in Sanfilippo Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Inventiva Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inventiva\u2019s Latest Research on Odiparcil\u2019s Mechanism of Action Published in Leading Peer-Reviewed Scientific Journal PLOS ONE","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cyclo Therapeutics Announces Positive Feedback from The EMA on its Pivotal Trial Design for Trappsol Cyclo for Niemann-Pick Disease Type C1","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Eiger Announces FDA Acceptance of NDA for Filing with Priority Review of Zokinvy for Treatment of Progeria and Progeroid Laminopathies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Aeglea BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aeglea BioTherapeutics to Present Late-Breaking 1-Year Data for Pegzilarginase in Patients with Arginase 1 Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Santhera Completes Enrollment of Phase 3 SIDEROS Study with Puldysa\u00ae (Idebenone) in Duchenne Muscular Dystrophy (DMD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Bristol Myers Squibb","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Bristol Myers Squibb Research at EHA 2020 Demonstrates Continued Advances Across Multiple Blood Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Antisense Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Antisense ATL1102 Final Phase II DMD Results Meet Primary Endpoint and Exceed Expectations on Secondary Endpoints","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"AUSTRALIA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Imara","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Imara to Present Data on IMR-687 in Sickle Cell Disease at the 25th Annual European Hematology Association (EHA) Congress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Chiesi Group Receives FDA Approval for Ferriprox\u00ae (deferiprone) twice-a-day tablets","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"ITALY","productType":"Small molecule","productStatus":"Approved","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios to Present Updated Clinical Proof-of-Concept Data from the Phase 2 Study of Mitapivat in Thalassemia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma\u2019s Phoenix Tissue Repair Provides Updates to its Recessive Dystrophic Epidermolysis Bullosa Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Inozyme Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inozyme and GACI Global Initiate Study to Understand Severity and Impact of Disease in Patients with ENPP1 Deficiency and ABCC6 Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"IND Enabling"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Catabasis Pharmaceuticals Presents Edasalonexent, A Potential Foundational Therapy For Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Minoryx Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Minoryx Therapeutics Doses First Patient With Leriglitazone In Registration-Enabling Cald Nexus Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"SPAIN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Spruce Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Spruce Biosciences Announces Launch of CAHstudy.com to Initiate Screening of Patients for CAH Clinical Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Capricor Announces Positive Top-Line Final Results from HOPE-2 Study in Patients with DMD Treated with Lead Candidate CAP-1002","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II"},{"orgOrder":0,"company":"AmideBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"U.S. FDA Grants Orphan Drug Designation to Amidebio's Glucagon Analog for the Treatment of Congenital Hyperinsulinism","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"IND Enabling"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Zafgen","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Zafgen and Chondrial Therapeutics Announce Definitive Merger Agreement","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Cowen","pharmaFlowCategory":"D","amount":"$40.0 million","upfrontCash":"Undisclosed","newsHeadline":"Marinus Pharmaceuticals Announces Proposed Public Offering of Common Stock","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Aeglea BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aeglea BioTherapeutics Announces 1-Year Data for Pegzilarginase in Patients with Arginase 1 Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioCryst to Present New Berotralstat Data at European Academy of Allergy and Clinical Immunology Digital Congress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Announces FDA Has Granted Priority Review of the Lumasiran New Drug Application for the Treatment of Primary Hyperoxaluria Type 1","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Genmab","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Genmab Announces Positive Topline Results in Phase III ANDROMEDA Study of Daratumumab in Light-chain (AL) Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"DENMARK","productType":"Large molecule","productStatus":"Approved","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Takeda Gets Positive CHMP Opinion for Pre-Filled Syringe Presentation of TAKHZYRO for Preventive Treatment for Hereditary Angioedema Attacks","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protalix BioTherapeutics and Chiesi Global Rare Diseases Submits BLA to U.S. Food and Drug Administration for Pegunigalsidase Alfa","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ISRAEL","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Ligand Pharmaceuticals","sponsor":"Palvella Therapeutics","pharmaFlowCategory":"D","amount":"$3.0 milion","upfrontCash":"$2.0 milion","newsHeadline":"Ligand Earns $3 Million Milestone Payment from Palvella Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Vigene Bioscience","sponsor":"ASC Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"ASC Therapeutics and Vigene Announce Long-Term Strategic Partnership for Gene Therapy Development and Manufacturing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"January 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Undisclosed"},{"orgOrder":0,"company":"Audentes Therapeutics","sponsor":"Astellas Pharma","pharmaFlowCategory":"D","amount":"$3,000.0 million","upfrontCash":"$3,000.0 million","newsHeadline":"Astellas Enters into Definitive Agreement to Acquire Audentes Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2019","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"LogicBio Therapeutics Announces Submission of Investigational New Drug Application (IND) for LB-001 for Methylmalonic Acidemia (MMA) and Highlights Key Corporate Milestones","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IND Enabling"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioMarin, Pioneer in Phenylketonuria, to Begin Clinical Trial with BMN 307 Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IND Enabling"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AVROBIO Announces Clinical and Regulatory Progress for Gaucher Disease Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Aruvant Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aruvant Receives Orphan Drug Designation for ARU-1801 for the Treatment of Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"SWITZERLAND","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Announces FDA Granted Orphan Drug Designation for OTL-102 for the Treatment of X-linked Chronic Granulomatous Disease (X-CGD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Undisclosed"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AVROBIO Announces Clinical Data Presentation","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AVROBIO Presents Initial Data for Cystinosis Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"LogicBio Therapeutics Provides Update on FDA Review of LB-001 NDA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IND Enabling"},{"orgOrder":0,"company":"CRISPR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CRISPR Therapeutics Provides Research Update for CTX120","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Freeline Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Freeline presents data on its AAV-based gene therapies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Prevail Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Prevail Therapeutics\u2019 PR001 Gets Orphan Drug Designation By FDA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Reneo Pharmaceuticals Initiates Clinical Trial in McArdle Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioMarin's Biologics License Application for Valoctocogene Roxaparvovec Accepted for Priority Review by FDA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase III"},{"orgOrder":0,"company":"Lysogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Lysogene Receives FDA Fast Track Designation for LYS-SAF302 Gene Therapy in MPS IIIA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"uniQure Presents Multiple New Preclinical Data on AMT-130 at the CHDI\u2019s 15th Annual Huntington\u2019s Disease Therapeutics Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I"},{"orgOrder":0,"company":"University of North Carolina","sponsor":"AskBio","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"AskBio Enters Research Collaboration and Licensing Agreement with University of North Carolina (UNC) for Angelman Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"March 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Undisclosed"},{"orgOrder":0,"company":"GenSight Biologics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GenSight Biologics Announces Presentation of Bilateral Visual Recovery From GS010 Phase III Trials","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase III"},{"orgOrder":0,"company":"Freeline Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Freeline receives Orphan Drug Designation from the European Commission","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Homology Medicines Announces Peer-Reviewed Publication Of HMI-102 Investigational Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"uniQure Announces Achievement of Target Patient Dosing in HOPE-B Pivotal Trial of AMT-061 in Hemophilia B","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase III"},{"orgOrder":0,"company":"Dicerna Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Dicerna Provides Initial Observations From PHYOX 3 Trial of Nedosiran","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase III"},{"orgOrder":0,"company":"Genespire","sponsor":"Sofinnova Partners","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Sofinnova Telethon Fund Backs Naldini's Gene Therapy Biotech","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"April 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Undisclosed"},{"orgOrder":0,"company":"Myosana Therapeutics","sponsor":"CureDuchenne Ventures","pharmaFlowCategory":"D","amount":"$1.0 million","upfrontCash":"Undisclosed","newsHeadline":"Myosana Therapeutics, Inc. Raises Up To $1M from CureDuchenne Ventures","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"April 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Undisclosed"},{"orgOrder":0,"company":"Affinia Therapeutics","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Vertex Pharmaceuticals and Affinia Therapeutics Set Up Collaboration to Discover and Develop Novel AAV Capsids for Genetic Therapies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Undisclosed"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"PBM Capital","pharmaFlowCategory":"D","amount":"$30.0 million","upfrontCash":"Undisclosed","newsHeadline":"Taysha Gene Therapies Announce $30 Million Seed Financing and Strategic Partnership with the UT Southwestern Gene Therapy Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IND Enabling"},{"orgOrder":0,"company":"Genespire","sponsor":"Sofinnova Partners","pharmaFlowCategory":"D","amount":"$17.3 million","upfrontCash":"Undisclosed","newsHeadline":"Genespire Secures \u20ac16 Million Series A Financing from Sofinnova Partners to Advance Transformative Gene Therapies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"ITALY","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"April 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Undisclosed"},{"orgOrder":0,"company":"Mustang Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mustang Bio Receives Advanced Therapy Medicinal Product Classification from EMA for MB-107 Lentiviral Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Passage Bio Orphan Drug Designation for PBGM01 for Treatment of Infantile GM1 Gangliosidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IND Enabling"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Announces First Patient Dosed with OTL-201 Gene Therapy in Proof-of-Concept Clinical Trial for Sanfilippo Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"4D Molecular Therapeutics Announces Upcoming Oral and Poster Presentations at the American Society of Gene and Cell Therapy Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IND Enabling"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Announces Eight Presentations Across Gene Therapy Portfolio at the ASGCT 23rd Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Undisclosed"},{"orgOrder":0,"company":"SR-Tiget","sponsor":"Genespire","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Genespire and SR-Tiget Announce Alliance for the Development of Transformative Gene Therapies for Genetic Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Undisclosed"},{"orgOrder":0,"company":"Hitachi Chemicals","sponsor":"Bluebird Bio","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Hitachi Chemical Advanced Therapeutics Solutions and apceth Biopharma ink Clinical and Commercial Manufacturing Pact with Bluebird Bio","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"JAPAN","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Axplora","sponsor":"Lysogene","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Novasep and Lysogene Announce their New Collaboration for Development and Production of GM1 Gangliosidosis Gene Therapy Product","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"GERMANY","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I"},{"orgOrder":0,"company":"University of North Carolina","sponsor":"AskBio","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"AskBio and UNC Team Up to Develop Angelman Syndrome Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Phoenix Nest","sponsor":"Cure Sanfilippo Foundation","pharmaFlowCategory":"D","amount":"$0.4 million","upfrontCash":"Undisclosed","newsHeadline":"Cure Sanfilippo Grants $380,000 to Type C Gene Therapy Development","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Sio Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Axovant Announces Upcoming Presentations at the Annual Meeting of the American Society of Gene and Cell Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Undisclosed"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Solid Biosciences Provides Update regarding SGT-001 Phase I\/II Clinical Hold on IGNITE DMD","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Avrobio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AVROBIO Reports Updated Clinical Data from Investigational Gene Therapy Programs for Fabry Disease and Cystinosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Stoke Therapeutics's Preclinical Data reveal In-Vitro and In-Vivo Target Engagement and Protein Upregulation in OPA1 Protein Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Dyne Therapeutics Demonstrates FORCE\u2122 Platform\u2019s Potential to Deliver Potent Exon-Skipping Molecules Directly to Muscle","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Passage Bio Presents Data from Animal Models of Krabbe Disease at the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Announces Positive Longer-term Results from First Three Cohorts of Phase 1\/2 Study of DTX301 Gene Therapy OTC Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Magenta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Magenta Therapeutics Presents Data on MGTA-145 at The Annual Meeting of American Society of Gene and Cell Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Krystal Biotech Shares Positive Interim Results from Phase 1\/2 Trial of KB105 in TGM1-related Autosomal Recessive Congenital Ichthyosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Presents New Interim Data from OTL-203 Proof-of-concept Study for MPS-I","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Undisclosed"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"3 Hemophilia B Patients Report Uniqure's AMT-060 Gene Therapy Gave Them Normalcy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Agtc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AGTC Shares Positive Preclinical Data With Scientific Evidence for its Ongoing Clinical Program in X-Linked Retinitis Pigmentosa Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Passage Bio Receives Rare Pediatric Disease Designation for PBGM01 for Patients with GM1 Gangliosidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IND Enabling"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Novartis Wins Conditional EU Approval for Gene Therapy Zolgensma","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"SWITZERLAND","productType":"Cell and Gene therapy","productStatus":"Approved","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Approved"},{"orgOrder":0,"company":"Expression Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Agrees to Phase 1 Trial of ET3, Gene Therapy for Hemophilia A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioMarin Provides 4 Years of Clinical Data from Ongoing Phase 1\/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase III"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"REGENERON","pharmaFlowCategory":"D","amount":"$100.0 million","upfrontCash":"$70.0 million","newsHeadline":"Regeneron and Intellia Therapeutics Expand Collaboration to Develop Crispr\/Cas9-Based Treatments","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Personalis","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Personalis Announces Scientific Collaboration with Sarepta Therapeutics on Immune Response to Precision Genetic Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Undisclosed"},{"orgOrder":0,"company":"Lupin Ltd","sponsor":"Exeltis Healthcare","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Lupin Signs Distribution Agreement in Key EU Territories for Orphan Drug NaMuscla\u00ae","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"INDIA","productType":"Small molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"American Porphyria Foundation","sponsor":"Alnylam Pharmaceuticals","pharmaFlowCategory":"D","amount":"$0.3 million","upfrontCash":"Undisclosed","newsHeadline":"Alnylam Announces Recipients of Second Annual Advocacy for Impact Grants Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"","productType":"Cell and Gene therapy","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Approved"},{"orgOrder":0,"company":"Duchenne research","sponsor":"Undisclosed","pharmaFlowCategory":"D","amount":"$1.0 million","upfrontCash":"Undisclosed","newsHeadline":"$1M Gene Therapy Research Grant Launched by PPMD, Duchenne UK","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Undisclosed"},{"orgOrder":0,"company":"Prilenia therapeutics","sponsor":"Morningside Venture","pharmaFlowCategory":"D","amount":"$62.5 million","upfrontCash":"Undisclosed","newsHeadline":"Prilenia Therapeutics Raises $62.5M to Fund Late Stage Trials In HD and ALS","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ISRAEL","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Highbridge Capital Management","pharmaFlowCategory":"D","amount":"$21.1 million","upfrontCash":"Undisclosed","newsHeadline":"Santhera Receives Financing Commitment of up to CHF 20 Million from Fund Managed by Highbridge Capital Management","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Rezolute","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rezolute to Present Clinical Data for RZ358, Lead Candidate in Phase 2b Trial in Congenital Hyperinsulinism","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Seattle Children\u2019s Research Institute","sponsor":"Moderna Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"RNA Gene Therapy Seen to Effectively Treat Hemophilia A Mouse Model","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vivet\u2019s Second Gene Therapy Product, VTX-803 for PFIC3, Receives US and European Orphan Drug Designation","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme Initiates Rolling Submission Of New Drug Application For Arimoclomol With FDA in Niemann-Pick Disease Type C","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam to Webcast Conference Call Discussing Full Results from ILLUMINATE-A Phase 3 Study of Lumasiran at ERA-EDTA International Congress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Corbus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Corbus Pharmaceuticals Announces Publication of Phase 2b Cystic Fibrosis Study Patient Demographics and Baseline Disease Characteristics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Santhera Announces Completion of ReveraGen\u2019s Long-Term Extension Study with Vamorolone in Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum\u2019s Maralixibat Demonstrates Transplant-Free Survival for Pediatric Patients with Progressive Familial Intrahepatic Cholestasis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CSL Behring and Seattle Children's Research Institute to Advance Gene Therapy Treatments for Primary Immunodeficiency Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Undisclosed"},{"orgOrder":0,"company":"Protagonist Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protagonist Therapeutics to Present at the 25th European Hematology Association (EHA) Annual Congress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Lysogene","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Lysogene Announces Important Updates on AAVance Phase 2\/3 Clinical Trial with LYS-SAF302 in MPS IIIA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Arcturus Therapeutics Announces First Healthy Volunteer Dosed in Phase 1 Study of ARCT-810 for Ornithine Transcarbamylase (OTC) Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta to Share Clinical Update from SRP-9003 Gene Therapy Trial for Limb-girdle Muscular Dystrophy Type 2E, or Beta-Sarcoglycanopathy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"New Data from the Phase 3 HELP Study Open-Label Extension Evaluate the Long-Term Safety and Efficacy of TAKHZYRO\u00ae (lanadelumab) in Reducing Hereditary Angioedema Attacks","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Duchenne UK","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Catabasis Pharmaceuticals and Duchenne UK Announce Partnership to Evaluate Edasalonexent in a Phase 2 Non-Ambulatory Duchenne Muscular Dystrophy Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Oppenheimer & Co.","pharmaFlowCategory":"D","amount":"$23.0 million","upfrontCash":"Undisclosed","newsHeadline":"Catabasis Pharmaceuticals Announces Pricing of $23 Million Underwritten Public Offering","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Evox Therapeutics","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","amount":"$882.0 million","upfrontCash":"$44.0 million","newsHeadline":"Evox Therapeutics and Takeda Sign Multi-target Rare Disease Collaboration","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"Undisclosed","date":"March 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Moderna Highlights Advances in Platform Science and Innovative Vaccine Research at Third Annual Science Day","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Announces FDA Orphan Drug Designation Granted to Mitapivat for Treatment of Thalassemia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Oxford Biomedica","sponsor":"Sanofi","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Sanofi Returns Rights to Gene Therapies to Oxford Biomedica","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II"},{"orgOrder":0,"company":"University of Massachusetts Medical School","sponsor":"CANbridge Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"CANbridge Pharmaceuticals Enters Into Rare Disease Gene Therapy Research Agreement With UMass Medical School","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Undisclosed"},{"orgOrder":0,"company":"Beam Therapeutics","sponsor":"Magenta Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Magenta's Technology Attracts Another Gene Therapy Developer Beam Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"IND Enabling"},{"orgOrder":0,"company":"Ipsen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ipsen Demonstrates Continued Commitment to Rare Diseases with Eight Abstracts Accepted at ENDO 2020","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GBT Announces Plans to Seek Expanded Labeling for Oxbryta\u00ae (voxelotor) to Treat Children Ages 4 to 11 Years with Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Saniona Receives Positive Feedback from FDA on Regulatory Path for Tesomet in Prader-Willi Syndrome (PWS)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Soleno's Drug Misses Key Goal in Prader-Willi Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Sio Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Axovant Completes Enrollment of Low-Dose Cohort in Phase 1\/2 Study of AXO-AAV-GM1 and Expands Study to Include Type I Patients with GM1 Gangliosidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Catalyst Biosciences Announces Oral and Poster Presentations at the World Federation of Hemophilia Virtual Summit 2020","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Ovid Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ovid Therapeutics Announces Multiple Presentations on the 2020 American Academy of Neurology Science Highlights Platform","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioCryst Announces Berotralstat Expanded Access Program for Patients with Hereditary Angioedema in United States","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Forma Therapeutics Announces Phase I Clinical Data for Investigational Agent FT-4202 in Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Rezolute","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rezolute Receives Rare Pediatric Disease Designation for RZ358","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Catalyst Biosciences Completes MarzAA Pharmacokinetic & Pharmacodynamic Study","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios PKR Activator Mitapivat Demonstrates Sustained Hemoglobin Responses in Non-transfusion-dependent \u03b1- and \u03b2-Thalassemia in Phase 2 Study","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Antisense Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Antisense Therapeutics Announces a Poster Presentation on the Muscular Dystrophy Association Virtual Conference 2020 website","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"AUSTRALIA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma\u2019s ML Bio Solutions Dosed First Subject in Phase 1 Clinical Trial of BBP-418 For Limb Girdle Muscular Dystrophy Type 2i","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Editas Medicine Presents Pre-Clinical Data from a Study of EDIT-301 with Sickle Patient Cells for Potential Treatment of Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IND Enabling"},{"orgOrder":0,"company":"Imara","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Imara Presents Positive Interim Results from Phase 2a Study of IMR-687 in Sickle C