Quotient Sciences Translational Pharmaceuticals Quotient Sciences Translational Pharmaceuticals

X
menu

    Market Place

    Market Place

    Post Enquiry

    Post Enquiry
    menu
    [{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Initiates Rolling Submission of New Drug Application (NDA) to U.S. Food and Drug Administration (FDA) for Lumasiran for the Treatment of Primary Hyperoxaluria Type 1 (PH1)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioCryst to Provide Berotralstat and BCX9930 Program Updates at 38th Annual J.P. Morgan Healthcare Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Abeona Therapeutics Initiates Pivotal Phase 3 Clinical Trial Evaluating EB-101 Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Abeona Therapeutics"},{"orgOrder":0,"company":"Fortress Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fortress Biotech Announces Rare Pediatric Disease Designation for CUTX-101 for the Treatment of Menkes Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Fortress Biotech"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Albireo Achieves Significant Milestones for Odevixibat Across Multiple Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Albireo Pharma"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"General Atlantic","pharmaFlowCategory":"D","amount":"$98.0 million","upfrontCash":"Undisclosed","newsHeadline":"CANbridge Pharmaceuticals Completes US$98 Million Series D Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Torii Pharmaceutical","pharmaFlowCategory":"D","amount":"$42.0 million","upfrontCash":"$22.0 million","newsHeadline":"BioCryst Submits Japanese NDA for Oral, Once Daily Berotralstat","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protalix Presents Key Clinical Data of Pegunigalsidase Alfa","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ISRAEL","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Protalix BioTherapeutics"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Priority Review for Avalglucosidase Alfa, a Potential New Therapy for Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Completes Enrollment of HELIOS-A Phase 3 Study of Vutrisiran for the Treatment of hATTR Amyloidosis with Polyneuropathy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Accepts BioCryst\u2019s NDA for Oral, Once Daily Berotralstat (BCX7353) to Prevent HAE Attacks","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Chugai\u2019s Huntington\\'s disease drug, RG6042 gets Japanese orphan drug designation","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Chugai Pharmaceutical"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Catabasis Pharmaceuticals to Present Edasalonexent at the XVIII International Conference on Duchenne and Becker Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Catabasis Pharmaceuticals"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Marinus Pharmaceuticals Completes Targeted Enrollment in Pivotal Phase 3 Study for CDKL5 Deficiency Disorder","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Marinus Pharmaceuticals"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Psagot Investment House","pharmaFlowCategory":"D","amount":"$43.7 million","upfrontCash":"Undisclosed","newsHeadline":"Protalix BioTherapeutics Announces $43.7 Million in Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ISRAEL","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Protalix BioTherapeutics"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Rare Pediatric Disease Designation to Stealth BioTherapeutics for Elamipretide for the Treatment of Barth Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Peptide","graph2":"Stealth Biotherapeutics"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Roche Trial of Ionis Huntington\u2019s Disease Drug Halted After Side Effects","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"F. Hoffmann-La Roche"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable ","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Berotralstat (BCX7353) Significantly Reduced Use of Acute On-Demand Medicine in HAE Patients in APeX-2","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rhythm Pharmaceuticals Receives Orphan Drug Designation from U.S. FDA for Setmelanotide for the Treatment of Alstr\u00f6m Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Peptide","graph2":"Rhythm Pharmaceuticals"},{"orgOrder":0,"company":"Pharnext","sponsor":"Not Applicable ","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pharnext Receives Promising Innovative Medicine Designation for PXT3003 ","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Small molecule","productStatus":"Approved","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Pharnext"},{"orgOrder":0,"company":"Castle Creek Biosciences","sponsor":"Fidelity Management & Research Company","pharmaFlowCategory":"D","amount":"$55.0 million","upfrontCash":"Undisclosed","newsHeadline":"Castle Creek Biosciences Announces $75 Million Investment","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Castle Creek Biosciences"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Receives Positive Opinion on Orphan Drug Designation from the European Medicines Agency for Mitapivat","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Agios Pharmaceuticals"},{"orgOrder":0,"company":"OxThera AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"OxThera Announces Completion of Recruitment in Phase 3 ePHex Study With Oxabact\u00ae in Patients With Primary Hyperoxaluria","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWEDEN","productType":"Large molecule","productStatus":"Undisclosed","date":"April 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"OxThera AB"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Completes Rolling Submission of New Drug Application to the U.S.FDA and Submits Marketing Authorization Application","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Receives Fast Track Designation for Vutrisiran for the Treatment of the Polyneuropathy of hATTR Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Catabasis Pharmaceuticals to Present at 2020 Muscular Dystrophy Association Virtual Poster Session","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Catabasis Pharmaceuticals"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ionis and Partner Announce Enrollment Completion of Global Phase 3 GENERATION HD1 Study for Huntington's Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Ionis Pharmaceuticals"},{"orgOrder":0,"company":"Aeglea BioTherapeutics","sponsor":"J.P. Morgan Securities","pharmaFlowCategory":"D","amount":"$100.0 million ","upfrontCash":"Undisclosed","newsHeadline":"Aeglea BioTherapeutics Announces Proposed Public Offering","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Aeglea BioTherapeutics"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Soleno Therapeutics Announces Publication Evaluating Role of Activating ATP-Sensitive Potassium Channel in Treatment of Hyperphagic Obesity","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Soleno Therapeutics"},{"orgOrder":0,"company":"Italfarmaco S.p.A","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Italfarmaco Provides Update on Advanced-Stage Clinical Development Program with Givinostat in Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ITALY","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Italfarmaco S.p.A"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"New Composition of Matter Patent Allowance Extends Protection for Berotralstat in U.S. Market by Four Years to 2039","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protalix BioTherapeutics Announces Topline Results from the BRIDGE Phase III Open-Label Trial of Pegunigalsidase Alfa in Fabry Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ISRAEL","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Protalix BioTherapeutics"},{"orgOrder":0,"company":"Vanda Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vanda Pharmaceuticals Inks Agreement with FDA on Resubmission of the Application for HETLIOZ to Treat Patients with Smith-Magenis Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Vanda Pharmaceuticals"},{"orgOrder":0,"company":"Aeglea BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aeglea BioTherapeutics to Present Late-Breaking 1-Year Data for Pegzilarginase in Patients with Arginase 1 Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Aeglea BioTherapeutics"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Santhera Completes Enrollment of Phase 3 SIDEROS Study with Puldysa\u00ae (Idebenone) in Duchenne Muscular Dystrophy (DMD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Santhera Pharmaceuticals"},{"orgOrder":0,"company":"Bristol Myers Squibb","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Bristol Myers Squibb Research at EHA 2020 Demonstrates Continued Advances Across Multiple Blood Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Bristol Myers Squibb"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Catabasis Pharmaceuticals Presents Edasalonexent, A Potential Foundational Therapy For Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Catabasis Pharmaceuticals"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Cowen","pharmaFlowCategory":"D","amount":"$40.0 million","upfrontCash":"Undisclosed","newsHeadline":"Marinus Pharmaceuticals Announces Proposed Public Offering of Common Stock","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Marinus Pharmaceuticals"},{"orgOrder":0,"company":"Aeglea BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aeglea BioTherapeutics Announces 1-Year Data for Pegzilarginase in Patients with Arginase 1 Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Aeglea BioTherapeutics"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioCryst to Present New Berotralstat Data at European Academy of Allergy and Clinical Immunology Digital Congress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Announces FDA Has Granted Priority Review of the Lumasiran New Drug Application for the Treatment of Primary Hyperoxaluria Type 1","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"Genmab","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Genmab Announces Positive Topline Results in Phase III ANDROMEDA Study of Daratumumab in Light-chain (AL) Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"DENMARK","productType":"Large molecule","productStatus":"Approved","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Genmab"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protalix BioTherapeutics and Chiesi Global Rare Diseases Submits BLA to U.S. Food and Drug Administration for Pegunigalsidase Alfa","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ISRAEL","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Protalix BioTherapeutics"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioMarin's Biologics License Application for Valoctocogene Roxaparvovec Accepted for Priority Review by FDA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"BioMarin Pharmaceutical"},{"orgOrder":0,"company":"GenSight Biologics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GenSight Biologics Announces Presentation of Bilateral Visual Recovery From GS010 Phase III Trials","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"GenSight Biologics"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"uniQure Announces Achievement of Target Patient Dosing in HOPE-B Pivotal Trial of AMT-061 in Hemophilia B","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Uniqure"},{"orgOrder":0,"company":"Dicerna Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Dicerna Provides Initial Observations From PHYOX 3 Trial of Nedosiran","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Dicerna Pharmaceuticals"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioMarin Provides 4 Years of Clinical Data from Ongoing Phase 1\/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"BioMarin Pharmaceutical"},{"orgOrder":0,"company":"Prilenia therapeutics","sponsor":"Morningside Venture","pharmaFlowCategory":"D","amount":"$62.5 million","upfrontCash":"Undisclosed","newsHeadline":"Prilenia Therapeutics Raises $62.5M to Fund Late Stage Trials In HD and ALS","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ISRAEL","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Prilenia therapeutics"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Highbridge Capital Management","pharmaFlowCategory":"D","amount":"$21.1 million","upfrontCash":"Undisclosed","newsHeadline":"Santhera Receives Financing Commitment of up to CHF 20 Million from Fund Managed by Highbridge Capital Management","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Santhera Pharmaceuticals"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam to Webcast Conference Call Discussing Full Results from ILLUMINATE-A Phase 3 Study of Lumasiran at ERA-EDTA International Congress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum\u2019s Maralixibat Demonstrates Transplant-Free Survival for Pediatric Patients with Progressive Familial Intrahepatic Cholestasis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Mirum Pharmaceuticals"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Oppenheimer & Co.","pharmaFlowCategory":"D","amount":"$23.0 million","upfrontCash":"Undisclosed","newsHeadline":"Catabasis Pharmaceuticals Announces Pricing of $23 Million Underwritten Public Offering","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Catabasis Pharmaceuticals"},{"orgOrder":0,"company":"Ipsen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ipsen Demonstrates Continued Commitment to Rare Diseases with Eight Abstracts Accepted at ENDO 2020","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Ipsen"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Soleno's Drug Misses Key Goal in Prader-Willi Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Soleno Therapeutics"},{"orgOrder":0,"company":"Ovid Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ovid Therapeutics Announces Multiple Presentations on the 2020 American Academy of Neurology Science Highlights Platform","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Ovid Therapeutics"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioCryst Announces Berotralstat Expanded Access Program for Patients with Hereditary Angioedema in United States","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"Halozyme Therapeutics","sponsor":"Johnson & Johnson Innovative Medicine","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Halozyme Reports Findings From Janssen's Phase 3 Andromeda Study Evaluating Daratumumab Utilizing ENHANZE In Light-Chain Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Halozyme Therapeutics"},{"orgOrder":0,"company":"Nippon Shinyaku","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"EC Grants Orphan Drug Designation to Nippon Shinyaku's viltolarsen (NS-065\/NCNP-01)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Nippon Shinyaku"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sanofi To Present Phase 3 Results of Avalglucosidase Alfa in Patients with Late-Onset Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Fortress Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fortress Biotech Announces Publication of Study on Estimated Birth Prevalence of Menkes Disease in\nMolecular Genetics and Metabolism Reports","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Fortress Biotech"},{"orgOrder":0,"company":"GlycoMimetics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"New Efficacy and Biomarker Data From Rivipansel Phase 3 RESET Trial to Be Presented at Sickle Cell Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"GlycoMimetics"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sanofi\u2019s Investigational Enzyme Replacement Therapy Improves Critical Manifestations of Late-Onset Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioMarin Provides Additional Data from Ongoing Phase 1\/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"BioMarin Pharmaceutical"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Sangamo Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pfizer and Sangamo Announce Updated Phase 1\/2 Results Showing Sustained Factor VIII Activity Levels and No Bleeding Events in 3e13 Vg\/Kg Cohort After Sb-525 Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Pfizer Inc"},{"orgOrder":0,"company":"Dicerna Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Dicerna Receives Rare Pediatric Disease Designation From U.S.FDA for Nedosiran for Treatment of Primary Hyperoxaluria","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Dicerna Pharmaceuticals"},{"orgOrder":0,"company":"Cancer Prevention Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cancer Prevention Pharmaceuticals Submits EU Marketing Authorization Application for CPP-1X\/sul to Treat Familial Adenomatous Polyposis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Cancer Prevention Pharmaceuticals"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sanofi Announces Long-Term Efficacy and Safety Data for Fitusiran from Interim Analysis of Phase 2 Extension Study in Hemophilia A and B","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Ovid Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ovid Therapeutics Receives FDA Rare Pediatric Disease Designation for OV101 for the Treatment of Angelman Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Ovid Therapeutics"},{"orgOrder":0,"company":"Uniqure","sponsor":"CSL Behring","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$450.0 million","newsHeadline":"CSL Behring Agrees to Acquire Novel Late-Stage Gene Therapy Candidate for Hemophilia B Patients from uniQure","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Uniqure"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"UK\u2019s MHRA renews EAMS Scientific Opinion for Santhera\u2019s Idebenone in Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Santhera Pharmaceuticals"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rhythm Pharmaceuticals Announces Positive Results from Phase 2 Study of Once-weekly Formulation of Setmelanotide in Healthy Obese Volunteers","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Peptide","graph2":"Rhythm Pharmaceuticals"},{"orgOrder":0,"company":"Cancer Prevention Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cancer Prevention Pharmaceuticals Submits New Drug Application to the FDA for CPP-1X\/sul for Treatment of Familial Adenomatous Polyposis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Cancer Prevention Pharmaceuticals"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rhythm Pharmaceuticals Submitted Marketing Authorization Application to the EMA for Setmelanotide in POMC and LEPR Deficiency Obesities","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Peptide","graph2":"Rhythm Pharmaceuticals"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Rhythm Rare Pediatric Disease Designation for Rare Obesities Drug","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Peptide","graph2":"Rhythm Pharmaceuticals"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics Completes Submission of NDA Seeking Approval of Casimersen for Patients with Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sarepta Therapeutics"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Data presented at ISTH Virtual Congress highlights Sanofi\u2019s commitment to advancing treatments for people with rare blood disorders","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Guggenheim Securities","pharmaFlowCategory":"D","amount":"$50.0 million","upfrontCash":"Undisclosed","newsHeadline":"Soleno Therapeutics Announces Pricing of $50 Million Public Offering of Common Stock","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Soleno Therapeutics"},{"orgOrder":0,"company":"GenSight Biologics","sponsor":"Kreos Capital","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"GenSight Biologics Obtains a \u20ac7m Non-Dilutive Loan and Renegotiates the Bond Agreement With Kreos Capital Extending Runway to Mid-2021","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"GenSight Biologics"},{"orgOrder":0,"company":"MolMed","sponsor":"Orchard Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Orchard Therapeutics and MolMed Announce Extension of Gene Therapy Manufacturing Collaboration","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ITALY","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"MolMed"},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Abeona Therapeutics Reinitiates Enrollment in EB-101 Pivotal Phase 3 VIITAL\u2122 Study in RDEB after COVID-19 Related Pause","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Abeona Therapeutics"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Data to be Presented at ISTH Virtual Congress Highlights Sobi's Commitment to Advancing Rare Haematology Treatments","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWEDEN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Swedish Orphan Biovitrum AB"},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Abeona Therapeutics Announces Two Presentations Related to Its RDEB Clinical Program at the Society for Pediatric Dermatology Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Abeona Therapeutics"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Announces that the United Kingdom\u2019s MHRA Grants Early Access to Lumasiran","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Catabasis Pharmaceuticals to Present at Parent Project Muscular Dystrophy Virtual Annual Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Catabasis Pharmaceuticals"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Albireo Launches Expanded Access Program for Odevixibat for Patients in the U.S., Canada, Australia and Europe","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Albireo Pharma"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Acer Therapeutics Administer ACER-001 in a Fasted State Increased Systemic Exposure of Phenylbutyrate in Healthy Volunteer Food Effect Study","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"July 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Acer Therapeutics"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioMarin Submits Marketing Authorization Application to European Medicines Agency for Vosoritide to Treat Children with Achondroplasia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Peptide","graph2":"BioMarin Pharmaceutical"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","amount":"$400.0 million","upfrontCash":"Undisclosed","newsHeadline":"Amicus Therapeutics Secures $400 Million Non-Dilutive Debt Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Amicus Therapeutics"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rhythm Pharmaceuticals Publishes Data from Phase 2 Study of Setmelanotide in Bardet-Biedl Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Peptide","graph2":"Rhythm Pharmaceuticals"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Krystal Biotech Announces Initiation of Pivotal Phase 3 Study of Beremagene Geperpavec in Patients with Dystrophic Epidermolysis Bullosa","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Krystal Biotech"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Marinus Pharmaceuticals Receives Rare Pediatric Disease Designation from FDA for Ganaxolone for the Treatment of CDKL5 Deficiency Disorder","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Marinus Pharmaceuticals"},{"orgOrder":0,"company":"Vanda Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Accepts and Grants Priority Review of Vanda's Applications for HETLIOZ\u00ae (tasimelteon) in the Treatment of Smith-Magenis Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"August 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Vanda Pharmaceuticals"},{"orgOrder":0,"company":"Fortress Biotech","sponsor":"Cyprium Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fortress Biotech Gets Positive Opinion on Orphan Drug Designation Received from the EMA for CUTX-101","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Fortress Biotech"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Blackstone Life Sciences","pharmaFlowCategory":"D","amount":"$350.0 million","upfrontCash":"Undisclosed","newsHeadline":"Reata Pharmaceuticals, Inc. and Blackstone Life Sciences Announce $350 Million Strategic Investment","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Reata Pharmaceuticals"},{"orgOrder":0,"company":"Levo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Levo Therapeutics Announces Top-line Results from Phase 3 CARE-PWS Study of LV-101","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Peptide","productStatus":"Approved","date":"August 2020","url1":"","url2":"","graph1":"Peptide","graph2":"Levo Therapeutics"},{"orgOrder":0,"company":"Eidos Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Eidos Therapeutics Appoints Industry Leaders With Significant Development and Commercial Expertise to Board of Directors","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Eidos Therapeutics"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pivotal Phase 3 Trial Testing Topical Gene Therapy B-VEC Now Enrolling DEB Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Krystal Biotech"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Reata Announces Positive Results From Year 2 of the Pivotal Phase 3 CARDINAL Study of Bardoxolone Methyl in Patients with Alport Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Reata Pharmaceuticals"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Chiesi Group","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protalix BioTherapeutics and Chiesi Global Rare Diseases Announce U.S. FDA Acceptance of BLA for Pegunigalsidase Alfa","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ISRAEL","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Protalix BioTherapeutics"},{"orgOrder":0,"company":"FibroGen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FibroGen Announces First Patient Enrolled in Pamrevlumab Phase 3 Clinical Trial in Patients with Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"FibroGen"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"European Medicines Agency Validates BioMarin's Marketing Authorization Application for Vosoritide to Treat Children with Achondroplasia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Peptide","graph2":"BioMarin Pharmaceutical"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Bluebird Bio to Present Data from Elivaldogene Autotemcel Gene Therapy Trial for Cerebral Adrenoleukodystrophy EBMT Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Bluebird Bio"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioMarin Submits New Drug Application to U.S. Food and Drug Administration for Vosoritide to Treat Children with Achondroplasia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Peptide","graph2":"BioMarin Pharmaceutical"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioMarin Receives Complete Response Letter (CRL) from FDA for Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"BioMarin Pharmaceutical"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protalix BioTherapeutics Completes Treatment Period for its Phase III BRIGHT Clinical Trial of Pegunigalsidase Alfa in Fabry Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ISRAEL","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Protalix BioTherapeutics"},{"orgOrder":0,"company":"Novo Nordisk","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Novo Nordisk Resumes the Phase 3 Clinical Trials Investigating Concizumab (Anti-Tfpi Mab) In Haemophilia A and B With or without Inhibitors","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"DENMARK","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Novo Nordisk"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Blackstone Life Sciences","pharmaFlowCategory":"D","amount":"$150.0 million","upfrontCash":"Undisclosed","newsHeadline":"Blackstone and Alnylam Close $150 Million R&D Financing to Advance RNAi Therapeutics for Cardiovascular Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"Ipsen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ipsen to Present Results from Move, the First Global Phase III Trial in Fibrodysplasia Ossificans Progressiva (fop), at Asbmr 2020","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Ipsen"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics Announces FDA Acceptance of Casimersen (SRP-4045) NDA for Duchenne Muscular Dystrophy Amenable to Skipping Exon 45","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sarepta Therapeutics"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mirum Pharmaceuticals Presents Maralixibat Five-Year Transplant-Free Survival Data for Patients With PFIC2 at Digital ILC 2020","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Mirum Pharmaceuticals"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Bluebird Bio Gene Therapy Shows Sustained Benefit in Rare Metabolic Disorder","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Bluebird Bio"},{"orgOrder":0,"company":"Charcot Marie Tooth Association","sponsor":"Pharnext","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"CMTA and Pharnext Enter Biomarker Research Collaboration","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Charcot Marie Tooth Association"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Highlights Prospects for Building an Industry-Leading ATTR Amyloidosis Franchise at RNAi Roundtable","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Albireo to Announce Topline Results from PEDFIC 1 Phase 3 Trial of Odevixibat on September 8, 2020","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Albireo Pharma"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioMarin Announces The Lancet Publishes Detailed Vosoritide Phase 3 Data in Children with Achondroplasia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Peptide","graph2":"BioMarin Pharmaceutical"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Albireo Phase 3 Trial Meets Both Primary Endpoints for Odevixibat in PFIC","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Albireo Pharma"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Liminal BioSciences Announces Resubmission of Biologics License Application to USFDA for Ryplazim\u00ae (plasminogen)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"CANADA","productType":"Large molecule","productStatus":"Approved","date":"September 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Liminal BioSciences"},{"orgOrder":0,"company":"Sanofi","sponsor":"Swedish Orphan Biovitrum AB","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"NEJM Publishes Positive Final Results from Phase 1\/2a Study of BIVV001 in People with Severe Hemophilia A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Ipsen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ipsen To Present New Insights at ASBMR For Potential Treatment of Ultra-Rare Disease Fibrodysplasia Ossificans Progressiva, Including Global Phase III MOVE Trial Results","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Ipsen"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Janssen Submits Application Seeking USFDA Approval of DARZALEX FASPRO\u2122 for the Treatment of Patients with Light Chain Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"September 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Johnson & Johnson Innovative Medicine"},{"orgOrder":0,"company":"Cancer Prevention Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cancer Prevention Pharmaceuticals Publishes Landmark Phase 3 Clinical Trial Data for Treatment of Familial Adenomatous Polyposis in NEJM","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Cancer Prevention Pharmaceuticals"},{"orgOrder":0,"company":"Ovid Therapeutics","sponsor":"Angelini Pharma","pharmaFlowCategory":"D","amount":"$232.0 million","upfrontCash":"$20.0 million","newsHeadline":"Ovid Therapeutics and Angelini Pharma Enter into Exclusive License Agreement to Develop, Manufacture and Commercialize OV101 for the Treatment of Angelman Syndrome in Europe","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Ovid Therapeutics"},{"orgOrder":0,"company":"Huntington Study Group","sponsor":"Prilenia Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Huntington Study Group Announces a Partnership with Prilenia Therapeutics to Conduct a Global Phase 3 Clinical Study of Pridopidine","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Huntington Study Group"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orphazyme Announces U.S. FDA Acceptance and Priority Review of New Drug Application for Arimoclomol for Niemann-Pick Disease Type C","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Orphazyme"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sobi's Orfadin\u00ae (nitisinone) Receives Positive Opinion from CHMP for Treatment of AKU","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWEDEN","productType":"Small molecule","productStatus":"Approved","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Swedish Orphan Biovitrum AB"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Liminal BioSciences Provides Update on Progress on BLA for Ryplazim\u00ae(plasminogen)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"CANADA","productType":"Large molecule","productStatus":"Approved","date":"September 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Liminal BioSciences"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Bluebird Bio\u2019s Lenti Globin\u2122 for Sickle Cell Disease Gene Therapy (bb1111) Granted PRIME Designation by European Medicines Agency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Bluebird Bio"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Catabasis Pharmaceuticals Presents Information on Edasalonexent at the Virtual 25th International Congress of the World Muscle Society","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Catabasis Pharmaceuticals"},{"orgOrder":0,"company":"Orphazyme","sponsor":"CytRx Corporation","pharmaFlowCategory":"D","amount":"$83.7 million","upfrontCash":"Undisclosed","newsHeadline":"CytRx Issues Statement Regarding Orphazyme\u2019s Global Offering","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Orphazyme"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma and Origin Biosciences Gets FDA Acceptance Of Its New Drug Application For Fosdenopterin For The Treatment Of MoCD Type A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"BridgeBio Pharma"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"EMA Accepts Sanofi's Regulatory Submission for Avalglucosidase Alfa for Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Reports Positive Topline Results from ILLUMINATE-B Phase 3 Study of Lumasiran for Primary Hyperoxaluria Type 1 in Children Aged 6","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Soleno Therapeutics Announces Updated Top-line Results from Phase III Trial of DCCR for Treatment of Prader-Willi Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Soleno Therapeutics"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"EMA Accepts Regulatory Submission for Avalglucosidase Alfa, a Potentially new Standard of Care Enzyme Replacement Therapy for Pompe disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Palladio Zannini Industrie","sponsor":"Samsara BioCapital","pharmaFlowCategory":"D","amount":"$20.0 million","upfrontCash":"Undisclosed","newsHeadline":"Palladio Completes $20 Million Series B Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ITALY","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Palladio Zannini Industrie"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pfizer Receives FDA Fast Track Designation For Duchenne Muscular Dystrophy Investigational Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Pfizer Inc"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Bluebird Bio Announces EMA Acceptance of Marketing Authorization Application for Elivaldogene Autotemcel Gene Therapy for Cerebral Adrenoleukodystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Bluebird Bio"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Chiesi Global Rare Diseases and Protalix BioTherapeutics Announce Launch of Expanded Access Program in the U.S for Pegunigalsidase Alfa for the Proposed Treatment of Fabry Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ISRAEL","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Protalix BioTherapeutics"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Santhera to Discontinue Phase 3 SIDEROS Study and Development of Puldysa\u00ae in Duchenne Muscular Dystrophy (DMD) and Focus on Vamorolone","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Santhera Pharmaceuticals"},{"orgOrder":0,"company":"GlycoMimetics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants GlycoMimetics Rare Pediatric Disease Designation for Rivipansel for Treatment of Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"GlycoMimetics"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Sangamo Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pfizer and Sangamo Dose First Participant in Phase 3 Study Evaluating Hemophilia a Gene Therapy Treatment","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Pfizer Inc"},{"orgOrder":0,"company":"The Italfarmaco Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Italfarmaco Gets FDA Rare Pediatric Disease Designation for Givinostat in Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ITALY","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"The Italfarmaco Group"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Santhera to Discontinue Phase 3 SIDEROS Study and Development of\n Puldysa\u00ae in Duchenne Muscular Dystrophy (DMD) and Focus on Vamorolone","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Santhera Pharmaceuticals"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Receives Positive CHMP Opinion for Libmeldy\u2122 for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Inventiva Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inventiva Receives FDA Fast Track Designation in MPS VI for its Clinical-Stage Asset Odiparci","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Inventiva Pharma"},{"orgOrder":0,"company":"Palladio Biosciences","sponsor":"Centessa Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Palladio Biosciences Announces Presentation of Data from a Study of Lixivaptan Use in an ADPKD Patient who Previously Experienced Liver Toxicity with Tolvaptan During the American Society of Nephrology Kidney Week 2020 Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Palladio Biosciences"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Presents Positive Results from ILLUMINATE-B Phase 3 Study in Pediatric Patients with Primary Hyperoxaluria Type 1","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Journal of Allergy and Clinical Immunology Publishes Results from BioCryst\u2019s APeX-2 Pivotal Trial of Oral, Once-Daily Berotralstat for the Prevention of HAE Attacks","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"Dicerna Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Dicerna Presents New Interim Data From PHYOX\u21223 Long-Term, Open-Label Extension Study of Nedosiran for Treatment of Primary Hyperoxaluria","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Dicerna Pharmaceuticals"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Albireo Announces Presentations at NASPGHAN 2020 Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Albireo Pharma"},{"orgOrder":0,"company":"Catabasis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Catabasis Pharmaceuticals Announces Top-Line Results for the Phase 3 PolarisDMD Trial of Edasalonexent in Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Catabasis Pharmaceuticals"},{"orgOrder":0,"company":"GlycoMimetics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"New Pediatric and Secondary Endpoint Data from Rivipansel Phase 3 RESET Trial Presented at ASCAT Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"GlycoMimetics"},{"orgOrder":0,"company":"Castle Creek Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Castle Creek Biosciences Announces First Patient Dosed in DeFi-RDEB Phase 3 Clinical Trial of Debcoemagene Autoficel (D-Fi) Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Castle Creek Biosciences"},{"orgOrder":0,"company":"Prilenia therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Prilenia Enrolls First Patients Into its PROOF-HD Phase 3 Clinical Trial for Huntington\u2019s Disease in the United States","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ISRAEL","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Prilenia therapeutics"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Soleno Therapeutics Announces Late-Breaking Oral Presentation with Body Composition Results from Phase III DESTINY PWS Trial of DCCR in Prader-Willi Syndrome at ObesityWeek\u00ae 2020 Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Soleno Therapeutics"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"MHRA Grants HAE Patients Early Access to BioCryst\u2019s Berotralstat in United Kingdom","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rhythm Pharmaceuticals Announces Publication of Results from Phase 3 Clinical Trials of Setmelanotide in The Lancet Diabetes & Endocrinology","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Peptide","graph2":"Rhythm Pharmaceuticals"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Albireo\u2019s Odevixibat PFIC Phase 3 Results Accepted for AASLD Late-Breakers","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Albireo Pharma"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Reviews Recent Portfolio Progress and Reports Third Quarter 2020 Financial Results","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Soleno Therapeutics Announces Presentation of Body Composition Results from DESTINY PWS, a Phase III Trial of DCCR in Prader-Willi Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Soleno Therapeutics"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Food and Drug Administration Accepts BioMarin's New Drug Application for Vosoritide to Treat Children with Achondroplasia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Peptide","graph2":"BioMarin Pharmaceutical"},{"orgOrder":0,"company":"Caelum Biosciences","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Caelum and Alexion Announce Upcoming Data Presentations at the 62nd American Society of Hematology Annual Meeting and Exposition","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Caelum Biosciences"},{"orgOrder":0,"company":"Fortress Biotech","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fortress Biotech Announces Oral and Poster Data Presentations at the 62nd American Society of Hematology (ASH) Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Fortress Biotech"},{"orgOrder":0,"company":"GlycoMimetics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GlycoMimetics Program Data to be Highlighted Via Three Oral Presentations and Two Posters at 62nd ASH Annual Meeting and Exposition","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"GlycoMimetics"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rhythm Pharmaceuticals Announces New Clinical Data on Setmelanotide at ObesityWeek\u00ae 2020","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Peptide","graph2":"Rhythm Pharmaceuticals"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Liminal BioSciences Announces Upcoming Presentations for Ryplazim\u00ae (plasminogen) at the 62nd ASH Annual Meeting and Exposition","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"CANADA","productType":"Large molecule","productStatus":"Approved","date":"November 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Liminal BioSciences"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Bluebird Bio Confirms Its Agreement with the U.S. FDA Regarding BLA Submission for LentiGlobin\u2122","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Bluebird Bio"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Catalyst Biosciences Announces Poster Presentation at the 62nd Annual American Society of Hematology Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Catalyst Biosciences"},{"orgOrder":0,"company":"Genmab","sponsor":"Johnson & Johnson Innovative Medicine","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Janssen Submitts a Type II Variation Application to the EMA for Use Of Subcutaneous DARZALEX\u00ae in Patients with Light-Chain Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"DENMARK","productType":"Large molecule","productStatus":"Approved","date":"November 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Genmab"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Global Dosing Hold in Fitusiran Trials Initiated by Sanofi Genzyme to Investigate New Adverse Events","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Liminal BioSciences Announces FDA Extension of Review Period for Ryplazim\u00ae(Plasminogen) BLA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"CANADA","productType":"Large molecule","productStatus":"Approved","date":"November 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Liminal BioSciences"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"uniQure Announces Clinical Data Presentations at the 62nd American Society of Hematology (ASH) Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Uniqure"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"MyoKardia Presents Mavacamten Clinical and Non-Clinical Data at the American Heart Association\u2019s Scientific Sessions 2020","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Albireo Pharma"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioCryst Presents Data Showing Sustained Attack Rate Reductions and Quality of Life for HAE Patients Taking Berotralstat in APeX-2 Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"Palladio Biosciences","sponsor":"Centessa Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Palladio Biosciences Doses First Patient With Lixivaptan in The ALERT Study","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Palladio Biosciences"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Announces FDA Clearance of IND Application for OTL-200 for Metachromatic Leukodystrophy (MLD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Sangamo Therapeutics","sponsor":"Biogen","pharmaFlowCategory":"D","amount":"$2,720.0 million","upfrontCash":"$125.0 million","newsHeadline":"Sangamo Announces Closing of Biogen Collaboration Agreement","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Sangamo Therapeutics"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Allergy Publishes Results from BioCryst\u2019s APeX-J Trial of Oral, Once-Daily Berotralstat for the Prevention of HAE Attacks","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amicus Therapeutics Starts Rolling Biologic License Application to the U.S. FDA for AT-GAA in Late-Onset Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Amicus Therapeutics"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Catalyst Biosciences Gets FDA Fast Track Designation for Subcutaneous MarzAA to Treat Episodic Bleeding in Hemophilia A or B with Inhibitors","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Catalyst Biosciences"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ionis Announces Initiation of the Global Phase 3 BALANCE study for AKCEA-APOCIII-LRX in Patients with Familial Chylomicronemia Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Ionis Pharmaceuticals"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Bluebird Bio's Investigational LentiGlobin\u2122 Gene Therapy Results in Complete Elimination of SCD-Related Severe Vaso-Occlusive Events","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Bluebird Bio"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Janssen's ANDROMEDA Study Show Hematologic Response for DARZALEX FASPRO\u00ae in Newly Diagnosed Light Chain (AL) Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"December 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Johnson & Johnson Innovative Medicine"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Liminal BioSciences Presents Two Case Histories on Investigational Intravenous Plasminogen (Human) Replacement Therapy at ASH Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"CANADA","productType":"Large molecule","productStatus":"Approved","date":"December 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Liminal BioSciences"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Catalyst Biosciences Presents Crimson 1 Phase 3 Study Design at the 62nd Annual American Society of Hematology Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Catalyst Biosciences"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Marinus Launches Expanded Access Program for Ganaxolone Treatment in CDKL5 Deficiency Disorder","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Marinus Pharmaceuticals"},{"orgOrder":0,"company":"Seaside Therapeutics","sponsor":"Allos Pharma","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Allos Pharma Secures Exclusive License on Arbaclofen in Fragile X Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Seaside Therapeutics"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pfizer Doses First Participant in Phase 3 Marstacimab Study for People with Severe Hemophilia A and B with or Without Inhibitors","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Pfizer Inc"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"uniQure Presents Late-Breaking Data from the HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Uniqure"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Data for Bluebird Bio\u2019s beti-cel Gene Therapy Show Patients Across Ages and \u03b2-thalassemia Genotypes Achieve Transfusion Independence","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Approved","date":"December 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Bluebird Bio"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sanofi to Resume Dosing in Fitusiran Clinical Studies in the U.S","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Sangamo Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pfizer and Sangamo Announce Updated Phase 1\/2 Results of Hemophilia A Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Pfizer Inc"},{"orgOrder":0,"company":"Soleno Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Soleno Therapeutics Provides Regulatory Update on DCCR for the Treatment of Prader-Willi Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Soleno Therapeutics"},{"orgOrder":0,"company":"Ovid Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ovid Therapeutics Announces Phase 3 NEPTUNE Clinical Trial of OV101 for the Treatment of Angelman Syndrome Did Not Meet Primary Endpoint","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Ovid Therapeutics"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Albireo Initiates Global Phase 3 Clinical Trial of Odevixibat in Alagille Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Albireo Pharma"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rhythm Pharmaceuticals Announces Positive Results from Phase 3 Clinical Trial Evaluating Setmelanotide in Bardet-Biedl and Alstr\u00f6m Syndromes","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Rhythm Pharmaceuticals"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Reports Positive Topline Results from HELIOS-A Phase 3 Study of Vutrisiran in Patients with hATTR Amyloidosis with Polyneuropathy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pfizer Doses First Participant in Phase 3 Study for Duchenne Muscular Dystrophy Investigational Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Pfizer Inc"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioMarin Announces Positive Phase 3 Gene Therapy Trial Results in Adults with Severe Hemophilia A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"BioMarin Pharmaceutical"},{"orgOrder":0,"company":"Leo Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GenSight Biologics Announces Publication of Results from LUMEVOQ\u00ae RESCUE Pivotal Phase III Trial in AAO journal Ophthalmology\u00ae","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"DENMARK","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Leo Pharma"},{"orgOrder":0,"company":"Diurnal Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Diurnal Seeks UK MHRA Marketing Approval for Chronocort to Treat Patients with Rare Condition Congenital Adrenal Hyperplasia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"January 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Diurnal Group"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Relief Therapeutics","pharmaFlowCategory":"D","amount":"$1.0 million","upfrontCash":"$1.0 million","newsHeadline":"Relief Therapeutics and Acer Therapeutics Ink Option Agreement for Exclusivity to Negotiate a Collaboration and License Deal for ACER-001","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"January 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Acer Therapeutics"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Seelos Therapeutics Receives a Notice of Allowance in Japan for SLS-005 (Trehalose)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"January 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Seelos Therapeutics"},{"orgOrder":0,"company":"Prilenia therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Prilenia Enrolls First Subject in Europe in its PROOF-HD Phase 3 Clinical Trial for Huntington\u2019s Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ISRAEL","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Prilenia therapeutics"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Goldman Sachs & Co. LLC","pharmaFlowCategory":"D","amount":"$125.0 million","upfrontCash":"Undisclosed","newsHeadline":"Krystal Biotech Announces Pricing of Upsized $125 Million Public Offering of Common Stock","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Krystal Biotech"},{"orgOrder":0,"company":"Diurnal Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Diurnal Group plc - Chronocort\u00ae Phase 3 and Safety Extension Study Results Published in JCEM","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"February 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Diurnal Group"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protalix BioTherapeutics and Chiesi Global Rare Diseases Present Key Clinical Data of Pegunigalsidase Alfa for Fabry Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ISRAEL","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Protalix BioTherapeutics"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amicus\u2019 AT-GAA Shows Significant Improvements in Both Musculoskeletal and Respiratory Measures in Late-Onset Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Amicus Therapeutics"},{"orgOrder":0,"company":"Dicerna Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Dicerna Initiates PHYOX\u21224 Trial of Nedosiran for Primary Hyperoxaluria Type 3","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Dicerna Pharmaceuticals"},{"orgOrder":0,"company":"University of Cambridge","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Gene Therapy Study for Monogenic Blindness Fails to Meet Endpoint","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"University of Cambridge"},{"orgOrder":0,"company":"Centessa Pharmaceuticals","sponsor":"General Atlantic","pharmaFlowCategory":"D","amount":"$250.0 million","upfrontCash":"Undisclosed","newsHeadline":"Centessa Pharmaceuticals Launches with $250 Million Series A Financing and Unveils a New Kind of Pharmaceutical R&D Model","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Centessa Pharmaceuticals"},{"orgOrder":0,"company":"Cyprium Therapeutics","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$20.0 million","newsHeadline":"Cyprium Therapeutics and Sentynl Therapeutics Sign Development and Asset Purchase Agreement","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Cyprium Therapeutics"},{"orgOrder":0,"company":"Sanofi","sponsor":"Sanofi","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sanofi's Efanesoctocog Alfa Granted FDA Fast Track Designation for treatment of hemophilia A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"Approved","date":"February 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Italfarmaco S.p.A","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Italfarmaco Provides Update on Ongoing Clinical Programs with Givinostat in Oral Presentation at XVIII International Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ITALY","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Italfarmaco S.p.A"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Reata Pharmaceuticals, Inc. Submits NDA for Company\u2019s Lead Program: Bardoxolone in Alport Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Reata Pharmaceuticals"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Bluebird Bio Provides Updated Findings from Reported Case of AML in LentiGlobin for Sickle Cell Disease (SCD) Gene Therapy Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Bluebird Bio"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Relief Therapeutics","pharmaFlowCategory":"D","amount":"$36.0 million","upfrontCash":"$10.0 million","newsHeadline":"Relief and Acer Therapeutics Sign Collaboration for Worldwide Development and Commercialization of ACER-001","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"March 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Acer Therapeutics"},{"orgOrder":0,"company":"Neurocrine Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Neurocrine Biosciences to Present New Data Analyses for Crinecerfont in Adults with Classic Congenital Adrenal Hyperplasia at ENDO 2021","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Neurocrine Biosciences"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Ionis Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Roche Provides Update on Tominersen Programme in Manifest Huntington\u2019s Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cyclo Therapeutics Meets Primary Efficacy Endpoint in Phase 1\/2 Trial from Intravenous Trappsol\u00ae Cyclo\u2122 in Rare Disease Niemann-Pick Type C1","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"March 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Cyclo Therapeutics"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"First Patient Dosed in Phase 3 XTEND-Kids Study with Efanesoctocog Alfa in Children with Haemophilia A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWEDEN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Swedish Orphan Biovitrum AB"},{"orgOrder":0,"company":"FibroGen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FibroGen Receives Fast Track Designation from the U.S. FDA for Pamrevlumab for the Treatment of Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"FibroGen"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rhythm Pharmaceuticals Announces Positive Data with Setmelanotide in Additional MC4R Pathway Deficiency-related Obesities","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Peptide","productStatus":"Approved","date":"January 2021","url1":"","url2":"","graph1":"Peptide","graph2":"Rhythm Pharmaceuticals"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rhythm Pharmaceuticals Presents Data from Phase 2 Basket Study Showing Continued Weight Loss at Up to 9 Months HET Obesity on Setmelanotide","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Peptide","productStatus":"Approved","date":"March 2021","url1":"","url2":"","graph1":"Peptide","graph2":"Rhythm Pharmaceuticals"},{"orgOrder":0,"company":"FibroGen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FibroGen Receives Rare Pediatric Disease Designation from the U.S. FDA for Pamrevlumab for the Treatment of Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"FibroGen"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alnylam Presents Positive Results from HELIOS-A Phase 3 Study of Investigational Vutrisiran","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Novartis to Initiate SMART Phase 3b Global Study of Zolgensma in Children up to 21 kg, Building on Real-World Experience","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Cell and Gene therapy","productStatus":"Approved","date":"April 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Novartis Pharmaceuticals Corporation"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"uniQure Announces FDA Removes Clinical Hold on Hemophilia B Gene Therapy Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Uniqure"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Krystal Biotech Provides Update on Pivotal GEM-3 Study of B-VEC for DEB","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Krystal Biotech"},{"orgOrder":0,"company":"Pharnext","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Interim Analysis from Phase III Extension Study Shows Sustained Benefits of PXT3003 for Patients with Charcot-Marie-Tooth Disease Type 1A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Small molecule","productStatus":"Approved","date":"April 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Pharnext"},{"orgOrder":0,"company":"Uniqure","sponsor":"CSL Behring","pharmaFlowCategory":"D","amount":"$2,050.0 million","upfrontCash":"$450.0 million","newsHeadline":"UniQure Announces Closing of Commercialization and License Agreement With CSL Behring","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Uniqure"},{"orgOrder":0,"company":"Aeglea BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aeglea Completes Patient Randomization for its Pivotal Phase 3 Clinical Trial of Pegzilarginase for the Treatment of Arginase 1 Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Aeglea BioTherapeutics"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios to Present Updated Data from Clinical Trials of Mitapivat in Pyruvate Kinase Deficiency and Thalassemia at EHA 2021","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Agios Pharmaceuticals"},{"orgOrder":0,"company":"Centessa Pharmaceuticals","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","amount":"$330.0 million","upfrontCash":"Undisclosed","newsHeadline":"Centessa Announces Pricing of Initial Public Offering","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Centessa Pharmaceuticals"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Kedrion","pharmaFlowCategory":"D","amount":"$22.0 million","upfrontCash":"Undisclosed","newsHeadline":"Liminal BioSciences Announces Closing of Divestiture of Plasma Collection Centers and Enters into Option Agreement with Kedrion","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"CANADA","productType":"Large molecule","productStatus":"Approved","date":"May 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Liminal BioSciences"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioMarin Provides Highlights of 5 Years of Clinical Data from Ongoing Phase 1\/2 Study of Valoctocogene Roxaparvovec","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"BioMarin Pharmaceutical"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Albireo Receives Positive CHMP Opinion for Bylvay\u2122 (odevixibat) for Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Albireo Pharma"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Acer Therapeutics Plans NDA Submission for ACER-001 in Q3 2021 Following Pre-NDA Meeting with FDA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"May 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Acer Therapeutics"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Seelos Therapeutics Receives European Orphan Drug Designation for SLS-005 (Trehalose) in Amyotrophic Lateral Sclerosis (ALS)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"May 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Seelos Therapeutics"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pfizer Enrols First UK Participant in Phase 3 Investigational Gene Therapy Study for Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Pfizer Inc"},{"orgOrder":0,"company":"Pharnext","sponsor":"Alpha Blue Ocean","pharmaFlowCategory":"D","amount":"$96.0 million","upfrontCash":"Undisclosed","newsHeadline":"Alpha Blue Ocean & Pharnext Announce the Signing of a Financing Agreement for up to 81 Million Euros","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Small molecule","productStatus":"Approved","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Pharnext"},{"orgOrder":0,"company":"Ipsen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Accepts Ipsen\u2019s Nda for Ultra-Rare Genetic Disorder Drug","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Ipsen"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amryt Announces FDA Acceptance of New Drug Application for Oleogel-S10 for the Treatment of Epidermolysis Bullosa","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Amryt Pharma"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protalix and Chiesi Global Rare Diseases Provide Update Regarding Clinical Development of PRX-102 for Fabry Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ISRAEL","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Protalix BioTherapeutics"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"New Phase 3 Data at WCPGHAN Show Long-Term Safety, Tolerability, and Treatment Benefits of Bylvay (odevixibat) in PFIC","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Albireo Pharma"},{"orgOrder":0,"company":"Caelum Biosciences","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Caelum and Alexion Present Additional Phase 2 Data Reinforcing Safety and Tolerability of CAEL-101 in AL Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Caelum Biosciences"},{"orgOrder":0,"company":"OxThera AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"OxThera reports Results from Phase 3 ePHex Study with Oxabact in Primary Hyperoxaluria Patients with Maintained Kidney Function","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"SWEDEN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"OxThera AB"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Acer Therapeutics Plans Clinical Trial for EDSIVO (celiprolol) Following Type B FDA Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Acer Therapeutics"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Presents Positive Results from Phase 2 Study of Mitapivat in Non-transfusion-dependent \u03b1- and \u03b2-Thalassemia at EHA2021","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Agios Pharmaceuticals"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Bluebird Bio Announces the Lifting of FDA Clinical Hold for Sickle Cell Disease and \u03b2-Thalassemia Studies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Approved","date":"June 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Bluebird Bio"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cyclo Therapeutics Commences Patient Enrollment in TransportNPC","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"June 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Cyclo Therapeutics"},{"orgOrder":0,"company":"Biogen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Biogen Announces Topline Results from Phase 3 Gene Therapy Study in Choroideremia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Biogen"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioMarin Gets Positive CHMP Opinion in Europe for Vosoritide for Children with Achondroplasia from Age 2 Until Growth Plates Close","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Peptide","graph2":"BioMarin Pharmaceutical"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsH