[{"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","year":"2020","type":"Financing","leadProduct":"Eganelisib","moa":"PI3K-gamma","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Infinity Pharmaceuticals","amount2":0.070000000000000007,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.070000000000000007,"dosageForm":"Oral","sponsorNew":"Infinity Pharmaceuticals \/ BVF Partners L.P","highestDevelopmentStatusID":"8","companyTruncated":"Infinity Pharmaceuticals \/ BVF Partners L.P"},{"orgOrder":0,"company":"Genethon","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Genethon strengthens its collaboration with Sarepta Therapeutics for the development of the gene therapy product GNT0004 in Duchenne muscular dystrophy","therapeuticArea":"Musculoskeletal","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"January 2020","year":"2020","type":"Collaboration","leadProduct":"GNT0004","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Genethon","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase I","highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Genethon \/ Sarepta Therapeutics","highestDevelopmentStatusID":"6","companyTruncated":"Genethon \/ Sarepta Therapeutics"},{"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","year":"2020","type":"Acquisition","leadProduct":"Deferiprone","moa":"Iron","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Apotex Inc","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Approved","highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","productSubType":"Small molecule","amount2New":0,"dosageForm":"","sponsorNew":"Apotex Inc \/ Chiesi Group","highestDevelopmentStatusID":"12","companyTruncated":"Apotex Inc \/ Chiesi Group"},{"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","year":"2020","type":"Acquisition","leadProduct":"Koate DVI","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Bayer AG","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Approved","highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Bayer AG \/ WuXi Biologics","highestDevelopmentStatusID":"12","companyTruncated":"Bayer AG \/ WuXi Biologics"},{"orgOrder":0,"company":"Armata Pharmaceuticals","sponsor":"Innoviva","pharmaFlowCategory":"D","amount":"$25.0 million","upfrontCash":"Undisclosed","newsHeadline":"Armata Pharmaceuticals and Innoviva Announce $25 Million Strategic Investment to Support Advancement of Armata\\'s Bacteriophage Development Programs","therapeuticArea":"Infections and Infectious Diseases","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","year":"2020","type":"Financing","leadProduct":"AP-PA02","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Armata Pharmaceuticals","amount2":0.029999999999999999,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase I\/ Phase II","highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.029999999999999999,"dosageForm":"","sponsorNew":"Armata Pharmaceuticals \/ Innoviva","highestDevelopmentStatusID":"7","companyTruncated":"Armata Pharmaceuticals \/ Innoviva"},{"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","year":"2020","type":"Merger","leadProduct":"Isotretinoin","moa":"RAR","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Timber Pharmaceuticals","amount2":0.02,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","productSubType":"Small molecule","amount2New":0.02,"dosageForm":"Topical","sponsorNew":"Timber Pharmaceuticals \/ Timber Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Timber Pharmaceuticals \/ Timber Pharmaceuticals"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II\/ Phase III","highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","productSubType":"Small molecule","amount2New":0,"dosageForm":"","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"PTG-300","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Protagonist Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Protagonist Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Protagonist Therapeutics \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"ESB1609","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Escape Bio","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase I","highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Escape Bio \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Escape Bio \/ Not Applicable"},{"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":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","year":"2020","type":"Not Applicable","leadProduct":"ETX001","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Enterprise Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Preclinical","highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Enterprise Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Enterprise Therapeutics \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"AT-007","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Applied Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Applied Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Applied Therapeutics \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Tideglusib","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"AMO Pharma","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"Small molecule","amount2New":0,"dosageForm":"","sponsorNew":"AMO Pharma \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"AMO Pharma \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"PR001","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Prevail Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Prevail Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Prevail Therapeutics \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"DTX301","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Ultragenyx Pharmaceutical","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase I\/ Phase II","highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Ultragenyx Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Ultragenyx Pharmaceutical \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Linoleic Acid","moa":"Lipid peroxidation","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Retrotope","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II\/ Phase III","highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Retrotope \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Retrotope \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"MS1819","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"First Wave BioPharma","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Capsule","sponsorNew":"First Wave BioPharma \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"First Wave BioPharma \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Lumasiran","moa":"Glycolate oxidase","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Alnylam Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Alnylam Pharmaceuticals \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Berotralstat","moa":"Plasma kallikrein","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"BioCryst Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"BioCryst Pharmaceuticals \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"LZRSE-Col7A1 Engineered Autologous Epidermal Sheets","moa":"COL7A1","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Abeona Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Topical","sponsorNew":"Abeona Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Abeona Therapeutics \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"ACN00177","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Aeglea BioTherapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase I\/ Phase II","highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Aeglea BioTherapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Aeglea BioTherapeutics \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Cannabidiol","moa":"CB1 receptor","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","productSubType":"Controlled Substance","amount2New":0,"dosageForm":"Transdermal Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"ELX-02","moa":"Ribosomal protein","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Eloxx Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"SHP656","moa":"Blood coagulation factor","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Xenetic Biosciences","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase I\/ Phase II","highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Xenetic Biosciences \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Xenetic Biosciences \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Setrusumab","moa":"Sclerostin","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mereo BioPharma Group","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Mereo BioPharma Group \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Mereo BioPharma Group \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"GTX-102","moa":"UBE3A-AS expression","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"GeneTx Biotherapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"GeneTx Biotherapeutics \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"GeneTx Biotherapeutics \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Copper histidinate","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Fortress Biotech","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","productSubType":"Vitamins\/Minerals\/Inorganic Salts","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Fortress Biotech \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Fortress Biotech \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Trehalose API","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Seelos Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"Small molecule","amount2New":0,"dosageForm":"","sponsorNew":"Seelos Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Seelos Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Genentech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Genentech\u2019s Risdiplam Meets Primary Endpoint in Pivotal FIREFISH Trial in Infants With Type 1 Spinal Muscular Atrophy","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","year":"2020","type":"Not Applicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Genentech","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Approved","highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","productSubType":"Small molecule","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Genentech \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Genentech \/ Not Applicable"},{"orgOrder":0,"company":"Ipsen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ipsen\u2019s palovarotene clinical program in fibrodysplasia ossificans progressiva reaches prespecified interim analysis futility criteria","therapeuticArea":"Musculoskeletal","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","year":"2020","type":"Not Applicable","leadProduct":"Palovarotene","moa":"RAR-gamma","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Ipsen","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Approved","highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","productSubType":"Small molecule","amount2New":0,"dosageForm":"","sponsorNew":"Ipsen \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Ipsen \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Sonlicromanol","moa":"Lipid peroxidation","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Khondrion","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"Small molecule","amount2New":0,"dosageForm":"Oral","sponsorNew":"Khondrion \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Khondrion \/ Not Applicable"},{"orgOrder":0,"company":"Arrevus","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Arrevus Receives FDA Qualified Infectious Disease Product (QIDP) Designation for the Treatment of Cystic Fibrosis Pulmonary Exacerbations","therapeuticArea":"Pulmonary\/Respiratory Diseases","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"January 2020","year":"2020","type":"Not Applicable","leadProduct":"Sodium Fusidate","moa":"Bacterial protein synthesis","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arrevus","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"Antibiotic","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Arrevus \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Arrevus \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"EHP-102","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Emerald Health Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Oral","sponsorNew":"Emerald Health Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Emerald Health Pharmaceuticals \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"RTX-134","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Rubius Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase I","highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Rubius Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Rubius Therapeutics \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"QR-421a","moa":"USH2A protein","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ProQR Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase I\/ Phase II","highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"ProQR Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"ProQR Therapeutics \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"10153627","moa":"IBAT","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Albireo Pharma","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Oral","sponsorNew":"Albireo Pharma \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Albireo Pharma \/ Not Applicable"},{"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","year":"2020","type":"Public Offering","leadProduct":"PBGM01","moa":"Beta-galactosidase","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0.13,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase I\/ Phase II","highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.13,"dosageForm":"","sponsorNew":"Passage Bio \/ J.P. Morgan","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ J.P. Morgan"},{"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","year":"2020","type":"Series D Financing","leadProduct":"Idursulfase beta","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"CANbridge Pharmaceuticals","amount2":0.10000000000000001,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Approved","highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.10000000000000001,"dosageForm":"Intravenous Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ General Atlantic","highestDevelopmentStatusID":"12","companyTruncated":"CANbridge Pharmaceuticals \/ General Atlantic"},{"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","year":"2020","type":"Series B Financing","leadProduct":"Tildacerfont","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spruce Biosciences","amount2":0.089999999999999997,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"Small molecule","amount2New":0.089999999999999997,"dosageForm":"Tablet","sponsorNew":"Spruce Biosciences \/ Omega Funds","highestDevelopmentStatusID":"8","companyTruncated":"Spruce Biosciences \/ Omega Funds"},{"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","year":"2020","type":"Public Offering","leadProduct":"Tovinontrine","moa":"PDE9","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Imara","amount2":0.089999999999999997,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.089999999999999997,"dosageForm":"Oral","sponsorNew":"Imara \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Imara \/ Undisclosed"},{"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","year":"2020","type":"Agreement","leadProduct":"Berotralstat","moa":"Plasma kallikrein","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioCryst Pharmaceuticals","amount2":0.040000000000000001,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Approved","highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.040000000000000001,"dosageForm":"Oral","sponsorNew":"BioCryst Pharmaceuticals \/ Torii Pharmaceutical","highestDevelopmentStatusID":"12","companyTruncated":"BioCryst Pharmaceuticals \/ Torii Pharmaceutical"},{"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","year":"2020","type":"Series A Financing","leadProduct":"AZ-3102","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Azafaros","amount2":0.029999999999999999,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Undisclosed","highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.029999999999999999,"dosageForm":"Oral","sponsorNew":"Azafaros \/ Forbion","highestDevelopmentStatusID":"1","companyTruncated":"Azafaros \/ Forbion"},{"orgOrder":0,"company":"Armata Pharmaceuticals","sponsor":"Innoviva","pharmaFlowCategory":"D","amount":"$25.0 million","upfrontCash":"Undisclosed","newsHeadline":"Armata Closes First $25 Million Securities Purchase Agreement","therapeuticArea":"Infections and Infectious Diseases","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2020","year":"2020","type":"Agreement","leadProduct":"AP-PA02","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Armata Pharmaceuticals","amount2":0.029999999999999999,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase I\/ Phase II","highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.029999999999999999,"dosageForm":"","sponsorNew":"Armata Pharmaceuticals \/ Innoviva","highestDevelopmentStatusID":"7","companyTruncated":"Armata Pharmaceuticals \/ Innoviva"},{"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","year":"2020","type":"Collaboration","leadProduct":"Neural stem cells","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"AgeX Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Undisclosed","highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"AgeX Therapeutics \/ University of California, Irvine","highestDevelopmentStatusID":"1","companyTruncated":"AgeX Therapeutics \/ University of California, Irvine"},{"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","year":"2020","type":"Collaboration","leadProduct":"Bispecific antibodies","moa":"Factor VIII","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Light Chain Bioscience","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Discovery","highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Light Chain Bioscience \/ Takeda","highestDevelopmentStatusID":"2","companyTruncated":"Light Chain Bioscience \/ Takeda"},{"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","year":"2020","type":"Acquisition","leadProduct":"Ciprofloxacin","moa":"DNA gyrase","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aradigm","amount2":0.01,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"Antibiotic","amount2New":0.01,"dosageForm":"Inhalation","sponsorNew":"Aradigm \/ Grifols","highestDevelopmentStatusID":"8","companyTruncated":"Aradigm \/ Grifols"},{"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","year":"2020","type":"Acquisition","leadProduct":"Recombinant coagulation factor IX","moa":"Factor IX","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Aptevo Therapeutics","amount2":0.029999999999999999,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Approved","highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.029999999999999999,"dosageForm":"Intravenous Infusion","sponsorNew":"Aptevo Therapeutics \/ Medexus Pharmaceuticals","highestDevelopmentStatusID":"12","companyTruncated":"Aptevo Therapeutics \/ Medexus Pharmaceuticals"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Odiparcil","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inventiva Pharma","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"Small molecule","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Inventiva Pharma \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Inventiva Pharma \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Cannabidiol","moa":"CB1 receptor","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","productSubType":"Controlled Substance","amount2New":0,"dosageForm":"Transdermal Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Zynerba Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Scholar Rock Presents Data for SRK-015","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2020","year":"2020","type":"Not Applicable","leadProduct":"SRK-015","moa":"Myostatin activation","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Scholar Rock \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Pegunigalsidase alfa","moa":"Alpha-galactosidase A","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Protalix BioTherapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Approved","highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Protalix BioTherapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Protalix BioTherapeutics \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"mRNA-3704","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Moderna Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase I\/ Phase II","highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Moderna Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Moderna Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Rezolute","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rezolute Initiates Phase 2b Clinical Trial of Lead Candidate RZ358","therapeuticArea":"Endocrinology","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2020","year":"2020","type":"Not Applicable","leadProduct":"RZ358","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rezolute","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Rezolute \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Rezolute \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"RGLS4326","moa":"miR-17","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Regulus Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase I","highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Regulus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Regulus Therapeutics \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"JR-141","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"JCR Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II\/ Phase III","highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"JCR Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"JCR Pharmaceuticals \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"GTX-102","moa":"UBE3A-AS expression","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase I\/ Phase II","highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"GeneTx Biotherapeutics \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Sodium Fusidate","moa":"Bacterial protein synthesis","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Arrevus","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"Antibiotic","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Arrevus \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Arrevus \/ Not Applicable"},{"orgOrder":0,"company":"First Wave BioPharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AzurRx BioPharma Announces Presentation of Phase II MS1819 Data in Cystic Fibrosis at the 2020 Digestive Disease Week Conference","therapeuticArea":"Endocrinology","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2020","year":"2020","type":"Not Applicable","leadProduct":"MS1819","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"First Wave BioPharma","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Capsule","sponsorNew":"First Wave BioPharma \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"First Wave BioPharma \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Undisclosed","moa":"SNP3","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Wave Life Sciences","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Preclinical","highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Wave Life Sciences \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Wave Life Sciences \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Berotralstat","moa":"Plasma kallikrein","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioCryst Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Approved","highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Oral","sponsorNew":"BioCryst Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioCryst Pharmaceuticals \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Tominersen","moa":"mRNA","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chugai Pharmaceutical","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Chugai Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Chugai Pharmaceutical \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"FT-4202","moa":"Pyruvate kinase","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Forma Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II\/ Phase III","highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Forma Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Forma Therapeutics \/ Not Applicable"},{"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":"SWITZERLAND","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","year":"2020","type":"Not Applicable","leadProduct":"MGTA-456","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Magenta Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Magenta Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Magenta Therapeutics \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Cyclo Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Edasalonexent","moa":"NFkB","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Catabasis Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","productSubType":"Small molecule","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Catabasis Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Catabasis Pharmaceuticals \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Sodium Phenylbutyrate","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Acer Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","productSubType":"Small molecule","amount2New":0,"dosageForm":"Immediate Release Tablet","sponsorNew":"Acer Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Acer Therapeutics \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Cyclo Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"ELX-02","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Not Applicable"},{"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":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2020","year":"2020","type":"Not Applicable","leadProduct":"MRT5005","moa":"CFTR","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sanofi","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase I\/ Phase II","highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Nebulizer","sponsorNew":"Sanofi \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Sanofi \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"ZYN002","moa":"CB1 receptor","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Zynerba Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II\/ Phase III","highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Transdermal Gel","sponsorNew":"Zynerba Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Zynerba Pharmaceuticals \/ Not Applicable"},{"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","year":"2020","type":"Financing","leadProduct":"Pegunigalsidase alfa","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Protalix BioTherapeutics","amount2":0.040000000000000001,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Approved","highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.040000000000000001,"dosageForm":"","sponsorNew":"Protalix BioTherapeutics \/ Psagot Investment House","highestDevelopmentStatusID":"12","companyTruncated":"Protalix BioTherapeutics \/ Psagot Investment House"},{"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","year":"2020","type":"Public Offering","leadProduct":"Tovinontrine","moa":"PDE9","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Imara","amount2":0.080000000000000002,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.080000000000000002,"dosageForm":"Oral","sponsorNew":"Imara \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Imara \/ Undisclosed"},{"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","year":"2020","type":"Series B Financing","leadProduct":"SIG-001","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Sigilon Therapeutics","amount2":0.080000000000000002,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"IND Enabling","highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.080000000000000002,"dosageForm":"","sponsorNew":"Sigilon Therapeutics \/ Canada Pension Plan Investment Board ","highestDevelopmentStatusID":"5","companyTruncated":"Sigilon Therapeutics \/ Canada Pension Plan Investment Board "},{"orgOrder":0,"company":"Armata Pharmaceuticals","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","amount":"$5.0 million ","upfrontCash":"Undisclosed","newsHeadline":"Armata Pharmaceuticals Announces up to $5 Million Therapeutics Development Award from the Cystic Fibrosis Foundation","therapeuticArea":"Infections and Infectious Diseases","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2020","year":"2020","type":"Funding","leadProduct":"AP-PA02","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Armata Pharmaceuticals","amount2":0.01,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase I\/ Phase II","highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.01,"dosageForm":"","sponsorNew":"Armata Pharmaceuticals \/ Cystic Fibrosis Foundation","highestDevelopmentStatusID":"7","companyTruncated":"Armata Pharmaceuticals \/ Cystic Fibrosis Foundation"},{"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","year":"2020","type":"Divestment","leadProduct":"FT-4202","moa":"RBC pyruvate kinase","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Forma Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II\/ Phase III","highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Forma Therapeutics \/ NewCo","highestDevelopmentStatusID":"9","companyTruncated":"Forma Therapeutics \/ NewCo"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Cyclo Therapeutics \/ Not Applicable ","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ Not Applicable "},{"orgOrder":0,"company":"Acadia Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ACADIA Pharmaceuticals and Neuren Pharmaceuticals Announce Rare Pediatric Disease Designation","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase III","country":"","productType":"Peptide","productStatus":"New Molecular Entity","date":"March 2020","year":"2020","type":"Not Applicable","leadProduct":"Trofinetide","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Acadia Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Approved","highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Solution","sponsorNew":"Acadia Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Acadia Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Dynacure","sponsor":"Not Applicable ","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Dynacure Announces First Patient Dosed in Phase 1 \/ 2 'UNITE-CNM' Study of DYN101","therapeuticArea":"Musculoskeletal","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2020","year":"2020","type":"Not Applicable","leadProduct":"DYN101","moa":"Dynamin 2 expression","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dynacure","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase I\/ Phase II","highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Dynacure \/ Not Applicable ","highestDevelopmentStatusID":"7","companyTruncated":"Dynacure \/ Not Applicable "},{"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","year":"2020","type":"Not Applicable","leadProduct":"Tominersen","moa":"Huntingtin mRNA antisense","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"F. Hoffmann-La Roche","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"F. Hoffmann-La Roche \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"F. Hoffmann-La Roche \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"ACE-083","moa":"GDF8","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Acceleron Pharma","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intramuscular Injection","sponsorNew":"Acceleron Pharma \/ Not Applicable ","highestDevelopmentStatusID":"8","companyTruncated":"Acceleron Pharma \/ Not Applicable "},{"orgOrder":0,"company":"Acadia Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ACADIA announce Rare Pediatric Disease Designation for Trofinetide","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase III","country":"","productType":"Peptide","productStatus":"New Molecular Entity","date":"March 2020","year":"2020","type":"Not Applicable","leadProduct":"Trofinetide","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Acadia Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Approved","highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Solution","sponsorNew":"Acadia Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Acadia Pharmaceuticals \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Berotralstat","moa":"Plasma kallikrein","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioCryst Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Approved","highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Not Applicable ","highestDevelopmentStatusID":"12","companyTruncated":"BioCryst Pharmaceuticals \/ Not Applicable "},{"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","year":"2020","type":"Not Applicable","leadProduct":"GTX-102","moa":"UBE3A-AS expression","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"GeneTx Biotherapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase I\/ Phase II","highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"GeneTx Biotherapeutics \/ Not Applicable ","highestDevelopmentStatusID":"7","companyTruncated":"GeneTx Biotherapeutics \/ Not Applicable "},{"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":"","productType":"Peptide","productStatus":"New Molecular Entity","date":"March 2020","year":"2020","type":"Not Applicable","leadProduct":"Setmelanotide Acetate","moa":"Melanocortin receptor 4","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Rhythm Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Rhythm Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Rhythm Pharmaceuticals \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Baclofen","moa":"GABA B receptor","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Pharnext","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","productSubType":"Small molecule","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Pharnext \/ Not Applicable ","highestDevelopmentStatusID":"10","companyTruncated":"Pharnext \/ Not Applicable "},{"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","year":"2020","type":"Not Applicable","leadProduct":"Lonafarnib","moa":"Ftase","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eiger BioPharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"Small molecule","amount2New":0,"dosageForm":"Oral","sponsorNew":"Eiger BioPharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Eiger BioPharmaceuticals \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Hydroxypropyl-Beta Cyclodextrin","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Cyclo Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Cyclo Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Cyclo Therapeutics \/ Not Applicable"},{"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","year":"2020","type":"Funding","leadProduct":"Dabocemagene Autoficel","moa":"COL7A1","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Castle Creek Biosciences","amount2":0.059999999999999998,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase III","highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.059999999999999998,"dosageForm":"Intradermal Injection","sponsorNew":"Castle Creek Biosciences \/ Fidelity Management & Research Company","highestDevelopmentStatusID":"10","companyTruncated":"Castle Creek Biosciences \/ Fidelity Management & Research Company"},{"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","year":"2020","type":"Series A Financing","leadProduct":"mRNA-mediated protein replacement therapies","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"ReCode Therapeutics","amount2":0.080000000000000002,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Undisclosed","highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.080000000000000002,"dosageForm":"","sponsorNew":"ReCode Therapeutics \/ MPM Capital","highestDevelopmentStatusID":"1","companyTruncated":"ReCode Therapeutics \/ MPM Capital"},{"orgOrder":0,"company":"Armata Pharmaceuticals","sponsor":"Innoviva","pharmaFlowCategory":"D","amount":"$25.0 million","upfrontCash":"Undisclosed","newsHeadline":"Armata Pharmaceuticals Announces Closing of Second Tranche of $25 Million Securities Purchase Agreement with Innoviva, Inc.","therapeuticArea":"Infections and Infectious Diseases","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2020","year":"2020","type":"Financing","leadProduct":"AP-PA02","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Armata Pharmaceuticals","amount2":0.029999999999999999,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase I\/ Phase II","highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.029999999999999999,"dosageForm":"","sponsorNew":"Armata Pharmaceuticals \/ Innoviva","highestDevelopmentStatusID":"7","companyTruncated":"Armata Pharmaceuticals \/ Innoviva"},{"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","year":"2020","type":"Financing","leadProduct":"Oligonucleotide linked antibody","moa":"Dystrophin","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Dyne Therapeutics","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Undisclosed","highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"","sponsorNew":"Dyne Therapeutics \/ CureDuchenne Ventures","highestDevelopmentStatusID":"1","companyTruncated":"Dyne Therapeutics \/ CureDuchenne Ventures"},{"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","year":"2020","type":"Financing","leadProduct":"Pepinemab","moa":"SEMA4D","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vaccinex","amount2":0.02,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0.02,"dosageForm":"Intravenous Infusion","sponsorNew":"Vaccinex \/ Keystone Capital Partners","highestDevelopmentStatusID":"8","companyTruncated":"Vaccinex \/ Keystone Capital Partners"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Scholar Rock Presents Data for SRK-015 at the Muscular Dystrophy Association Clinical and Scientific Conference","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2020","year":"2020","type":"Not Applicable","leadProduct":"SRK-015","moa":"Myostatin activation","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Scholar Rock \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"ELX-02","moa":"Ribosome activity","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase II","highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"OT-58","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orphan Technologies","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase I\/ Phase II","highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Orphan Technologies \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Orphan Technologies \/ Not Applicable"},{"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","year":"2020","type":"Not Applicable","leadProduct":"Inzomelid","moa":"","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Inflazome","amount2":0,"therapeuticAreaNew":"Genetic Disease","highestDevelopmentStatusNew":"Phase I","highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","productSubType":"","amount2New":0,"dosageForm":"Oral","sponsorNew":"Inflazome \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Inflazome \/ Not Applicable"},{"orgOrder":0,"company":"AP