[{"orgOrder":0,"company":"Apotex Inc","sponsor":"CHIESI USA INC","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Small molecule","year":"2020","type":"Acquisition","leadProduct":"Deferiprone","moa":"Iron","graph1":"Genetic Disease","graph2":"Approved","graph3":"Apotex Inc","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Apotex Inc \/ Chiesi Group","highestDevelopmentStatusID":"12","companyTruncated":"Apotex Inc \/ Chiesi Group"},{"orgOrder":0,"company":"Bayer AG","sponsor":"WuXi Biologics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Large molecule","year":"2020","type":"Acquisition","leadProduct":"Koate DVI","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Bayer AG \/ WuXi Biologics","highestDevelopmentStatusID":"12","companyTruncated":"Bayer AG \/ WuXi Biologics"},{"orgOrder":0,"company":"Genentech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Genentech \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Genentech \/ Not Applicable"},{"orgOrder":0,"company":"Ipsen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Musculoskeletal","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Palovarotene","moa":"RAR-gamma","graph1":"Genetic Disease","graph2":"Approved","graph3":"Ipsen","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Ipsen \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Ipsen \/ Not Applicable"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"General Atlantic","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Large molecule","year":"2020","type":"Series D Financing","leadProduct":"Idursulfase beta","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"CANbridge Pharmaceuticals","amount2":0.10000000000000001,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Intravenous Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ General Atlantic","highestDevelopmentStatusID":"12","companyTruncated":"CANbridge Pharmaceuticals \/ General Atlantic"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Torii Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Agreement","leadProduct":"Berotralstat","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioCryst Pharmaceuticals","amount2":0.040000000000000001,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Oral","sponsorNew":"BioCryst Pharmaceuticals \/ Torii Pharmaceutical","highestDevelopmentStatusID":"12","companyTruncated":"BioCryst Pharmaceuticals \/ Torii Pharmaceutical"},{"orgOrder":0,"company":"Aptevo Therapeutics","sponsor":"Medexus Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Acquisition","leadProduct":"Recombinant coagulation factor IX","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Approved","graph3":"Aptevo Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Intravenous Infusion","sponsorNew":"Aptevo Therapeutics \/ Medexus Pharmaceuticals","highestDevelopmentStatusID":"12","companyTruncated":"Aptevo Therapeutics \/ Medexus Pharmaceuticals"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Pegunigalsidase alfa","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved","graph3":"Protalix BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Protalix BioTherapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Protalix BioTherapeutics \/ Not Applicable"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Berotralstat","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"BioCryst Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioCryst Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Psagot Investment House","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Large molecule","year":"2020","type":"Financing","leadProduct":"Pegunigalsidase alfa","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Protalix BioTherapeutics","amount2":0.040000000000000001,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"","sponsorNew":"Protalix BioTherapeutics \/ Psagot Investment House","highestDevelopmentStatusID":"12","companyTruncated":"Protalix BioTherapeutics \/ Psagot Investment House"},{"orgOrder":0,"company":"Acadia Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Peptide","year":"2020","type":"Not Applicable","leadProduct":"Trofinetide","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Acadia Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Acadia Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Acadia Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Acadia Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Peptide","year":"2020","type":"Not Applicable","leadProduct":"Trofinetide","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Acadia Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Acadia Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Acadia Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Berotralstat","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioCryst Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"LFB","sponsor":"HEMA Biologics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Eptacog Alfa","moa":"Factor X","graph1":"Genetic Disease","graph2":"Approved","graph3":"LFB","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"LFB \/ HEMA Biologics","highestDevelopmentStatusID":"12","companyTruncated":"LFB \/ HEMA Biologics"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Lumasiran","moa":"Glycolate oxidase","graph1":"Genetic Disease","graph2":"Approved","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Alnylam Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Genentech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Genentech \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Genentech \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"FSIO","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Agreement","leadProduct":"Lumacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Vertex Pharmaceuticals \/ FSIO","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ FSIO"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Lonafarnib","moa":"Ftase","graph1":"Genetic Disease","graph2":"Approved","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Eiger BioPharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Eiger BioPharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Berotralstat","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioCryst Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Pegunigalsidase alfa","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved","graph3":"Protalix BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Protalix BioTherapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Protalix BioTherapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Aptevo Therapeutics","sponsor":"Medexus Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Acquisition","leadProduct":"Recombinant coagulation factor IX","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Approved","graph3":"Aptevo Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Aptevo Therapeutics \/ Medexus","highestDevelopmentStatusID":"12","companyTruncated":"Aptevo Therapeutics \/ Medexus"},{"orgOrder":0,"company":"Vanda Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Tasimelteon","moa":"Melatonin receptor","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vanda Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Vanda Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vanda Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Lonafarnib","moa":"Ftase","graph1":"Genetic Disease","graph2":"Approved","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Eiger BioPharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Eiger BioPharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Deferiprone","moa":"Iron","graph1":"Genetic Disease","graph2":"Approved","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Chiesi Group \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Chiesi Group \/ Not Applicable"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Berotralstat","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioCryst Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Lumasiran","moa":"Glycolate oxidase","graph1":"Genetic Disease","graph2":"Approved","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Alnylam Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Pre-Filled Syringe","sponsorNew":"Takeda Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Takeda Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Pegunigalsidase alfa","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved","graph3":"Protalix BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Protalix BioTherapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Protalix BioTherapeutics \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Blood coagulation factor","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Onasemnogene abeparvovec-xioi","moa":"SMN1 gene","graph1":"Genetic Disease","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Blood coagulation factor","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Lupin Ltd","sponsor":"Exeltis Healthcare","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Small molecule","year":"2020","type":"Agreement","leadProduct":"Mexiletine","moa":"Sodium channel alpha subunits","graph1":"Genetic Disease","graph2":"Approved","graph3":"Lupin Ltd","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Lupin Ltd \/ Exeltis Healthcare","highestDevelopmentStatusID":"12","companyTruncated":"Lupin Ltd \/ Exeltis Healthcare"},{"orgOrder":0,"company":"American Porphyria Foundation","sponsor":"Alnylam Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Funding","leadProduct":"Patisiran","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"American Porphyria Foundation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"American Porphyria Foundation \/ Alnylam Pharmaceuticals","highestDevelopmentStatusID":"12","companyTruncated":"American Porphyria Foundation \/ Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Lumasiran","moa":"Glycolate oxidase","graph1":"Genetic Disease","graph2":"Approved","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Alnylam Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Pre-Filled Syringe","sponsorNew":"Takeda Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Takeda Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Ipsen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Palovarotene","moa":"RAR-gamma","graph1":"Genetic Disease","graph2":"Approved","graph3":"Ipsen","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Ipsen \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Ipsen \/ Not Applicable"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Berotralstat","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioCryst Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Betibeglogene autotemcel","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Bluebird Bio \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Bluebird Bio \/ Not Applicable"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Antihemophilic factor recombinant PEGylated-aucl","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Approved","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Lyophilized Powder for Solution for Intravenous","sponsorNew":"Bayer AG \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Bayer AG \/ Not Applicable"},{"orgOrder":0,"company":"Genentech","sponsor":"PTC Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Genentech \/ PTC Therapeutics","highestDevelopmentStatusID":"12","companyTruncated":"Genentech \/ PTC Therapeutics"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Blood coagulation factor","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"National Health Service (NHS)","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Large molecule","year":"2020","type":"Agreement","leadProduct":"Efmoroctocog alfa","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Swedish Orphan Biovitrum AB \/ National Health Service (NHS)","highestDevelopmentStatusID":"12","companyTruncated":"Swedish Orphan Biovitrum AB \/ National Health Service (NHS)"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Ataluren","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Granules for Oral Suspension","sponsorNew":"PTC Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"PTC Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Taiba Healthcare","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Agreement","leadProduct":"Patisiran","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Alnylam Pharmaceuticals \/ Taiba","highestDevelopmentStatusID":"12","companyTruncated":"Alnylam Pharmaceuticals \/ Taiba"},{"orgOrder":0,"company":"Orsini Specialty Pharmacy","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Partnership","leadProduct":"Triheptanoin","moa":"Triglyceride","graph1":"Genetic Disease","graph2":"Approved","graph3":"Orsini Specialty Pharmacy","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Orsini Specialty Pharmacy \/ Ultragenyx Pharmaceutical","highestDevelopmentStatusID":"12","companyTruncated":"Orsini Specialty Pharmacy \/ Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Lumasiran","moa":"Glycolate oxidase","graph1":"Genetic Disease","graph2":"Approved","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Alnylam Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Genentech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Emicizumab","moa":"Factor IX\/Factor X","graph1":"Genetic Disease","graph2":"Approved","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Genentech \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Genentech \/ Not Applicable"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Albutrepenonacog Alfa","moa":"Factor X","graph1":"Genetic Disease","graph2":"Approved","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"CSL Behring \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"CSL Behring \/ Not Applicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Royalty Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Acquisition","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved","graph3":"PTC Therapeutics","amount2":0.65000000000000002,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0.65000000000000002,"dosageForm":"Oral","sponsorNew":"PTC Therapeutics \/ Royalty Pharma","highestDevelopmentStatusID":"12","companyTruncated":"PTC Therapeutics \/ Royalty Pharma"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Odevixibat","moa":"IBAT","graph1":"Genetic Disease","graph2":"Approved","graph3":"Albireo Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Albireo Pharma \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Albireo Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Emicizumab","moa":"Factor IX\/Factor X","graph1":"Genetic Disease","graph2":"Approved","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Chugai Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Chugai Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Burosumab","moa":"FGF23","graph1":"Genetic Disease","graph2":"Approved","graph3":"Kyowa Kirin","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Kyowa Kirin \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Kyowa Kirin \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2020","type":"Not Applicable","leadProduct":"Vosoritide","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Crizanlizumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Vanda Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Tasimelteon","moa":"Melatonin receptor","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vanda Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Vanda Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vanda Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Genentech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"Genentech \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Genentech \/ Not Applicable"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Chiesi Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Pegunigalsidase alfa","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved","graph3":"Protalix BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Protalix BioTherapeutics \/ Chiesi Global Rare Diseases","highestDevelopmentStatusID":"12","companyTruncated":"Protalix BioTherapeutics \/ Chiesi Global Rare Diseases"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Viltolarsen","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"NS Pharma \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"NS Pharma \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2020","type":"Not Applicable","leadProduct":"Vosoritide","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Elivaldogene autotemcel","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Bluebird Bio \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Bluebird Bio \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2020","type":"Not Applicable","leadProduct":"Vosoritide","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Blood coagulation factor","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Pegunigalsidase alfa","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved","graph3":"Protalix BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Protalix BioTherapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Protalix BioTherapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA positive","graph1":"Genetic Disease","graph2":"Approved","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Biogen \/ Not Applicable"},{"orgOrder":0,"company":"Ipsen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Palovarotene","moa":"RAR-gamma","graph1":"Genetic Disease","graph2":"Approved","graph3":"Ipsen","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Ipsen \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Ipsen \/ Not Applicable"},{"orgOrder":0,"company":"Recordati Rare Diseases","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Cysteamine Hydrochloride","moa":"Cystine reducing agent","graph1":"Genetic Disease","graph2":"Approved","graph3":"Recordati Rare Diseases","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Ophthalmic Solution","sponsorNew":"Recordati Rare Diseases \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Recordati Rare Diseases \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Elexacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Elivaldogene autotemcel","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Bluebird Bio \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Bluebird Bio \/ Not Applicable"},{"orgOrder":0,"company":"Option Care Health","sponsor":"NS Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Agreement","leadProduct":"Viltolarsen","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Option Care Health","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Option Care Health \/ NS Pharma","highestDevelopmentStatusID":"12","companyTruncated":"Option Care Health \/ NS Pharma"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Omaveloxolone","moa":"Nrf2","graph1":"Genetic Disease","graph2":"Approved","graph3":"Reata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Reata Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Omaveloxolone","moa":"Nrf2","graph1":"Genetic Disease","graph2":"Approved","graph3":"Reata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Reata Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Odevixibat","moa":"IBAT","graph1":"Genetic Disease","graph2":"Approved","graph3":"Albireo Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Albireo Pharma \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Albireo Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Biopharma","sponsor":"Global Blood Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.A.E","productType":"Small molecule","year":"2020","type":"Agreement","leadProduct":"Voxelotor","moa":"HbA positive","graph1":"Genetic Disease","graph2":"Approved","graph3":"Biopharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Biopharma \/ Global Blood Therapeutics","highestDevelopmentStatusID":"12","companyTruncated":"Biopharma \/ Global Blood Therapeutics"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2020","type":"Not Applicable","leadProduct":"Vosoritide","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Odevixibat","moa":"IBAT","graph1":"Genetic Disease","graph2":"Approved","graph3":"Albireo Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Albireo Pharma \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Albireo Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Santhera Pharmaceuticals","sponsor":"ReveraGen BioPharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Vamorolone","moa":"Mineralocorticoid receptor","graph1":"Genetic Disease","graph2":"Approved","graph3":"Santhera Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Santhera Pharmaceuticals \/ ReveraGen BioPharma","highestDevelopmentStatusID":"12","companyTruncated":"Santhera Pharmaceuticals \/ ReveraGen BioPharma"},{"orgOrder":0,"company":"Ipsen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Palovarotene","moa":"RAR-gamma","graph1":"Genetic Disease","graph2":"Approved","graph3":"Ipsen","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Ipsen \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Ipsen \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Daratumumab","moa":"CD38","graph1":"Genetic Disease","graph2":"Approved","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Not Applicable"},{"orgOrder":0,"company":"Cycle Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Nitisinone","moa":"HPDD","graph1":"Genetic Disease","graph2":"Approved","graph3":"Cycle Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Cycle Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Cycle Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Maralixibat","moa":"IBAT","graph1":"Genetic Disease","graph2":"Approved","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Mirum Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Mirum Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"SWITZERLAND","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Onasemnogene abeparvovec","moa":"SMN1 gene","graph1":"Genetic Disease","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Human C1 Esterase Inhibitor","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Approved","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"CSL Behring \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"CSL Behring \/ Not Applicable"},{"orgOrder":0,"company":"Taro Pharmaceutical Industries","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Deferiprone","moa":"Iron","graph1":"Genetic Disease","graph2":"Approved","graph3":"Taro Pharmaceutical Industries","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Taro Pharmaceutical Industries \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Taro Pharmaceutical Industries \/ Not Applicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"HLS Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Large molecule","year":"2020","type":"Acquisition","leadProduct":"Porcine recombinant Factor VIII","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Takeda Pharmaceutical","amount2":0.050000000000000003,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"","sponsorNew":"Takeda Pharmaceutical \/ HLS Therapeutics","highestDevelopmentStatusID":"12","companyTruncated":"Takeda Pharmaceutical \/ HLS Therapeutics"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Onasemnogene Abeparvovec","moa":"SMN1 gene","graph1":"Genetic Disease","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Lumasiran","moa":"Glycolate oxidase","graph1":"Genetic Disease","graph2":"Approved","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Alnylam Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Kamada","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Large molecule","year":"2020","type":"Agreement","leadProduct":"Alpha1-Proteinase Inhibitor (Human)","moa":"Alpha-1 antitrypsin protein","graph1":"Genetic Disease","graph2":"Approved","graph3":"Kamada","amount2":0.029999999999999999,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Solution For Infusion","sponsorNew":"Kamada \/ Takeda","highestDevelopmentStatusID":"12","companyTruncated":"Kamada \/ Takeda"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Elivaldogene autotemcel","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Bluebird Bio \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Bluebird Bio \/ Not Applicable"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Burosumab","moa":"FGF23","graph1":"Genetic Disease","graph2":"Approved","graph3":"Kyowa Kirin","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Kyowa Kirin \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Kyowa Kirin \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Libmeldy","moa":"ARSA gene","graph1":"Genetic Disease","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Orchard Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Agreement","leadProduct":"NAV AAV9 vector","moa":"SMN1 gene","graph1":"Genetic Disease","graph2":"Approved","graph3":"Regenxbio","amount2":0.080000000000000002,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Intravenous Infusion","sponsorNew":"Regenxbio \/ Novartis","highestDevelopmentStatusID":"12","companyTruncated":"Regenxbio \/ Novartis"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Lumasiran","moa":"Glycolate oxidase","graph1":"Genetic Disease","graph2":"Approved","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Alnylam Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Berotralstat","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioCryst Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Odevixibat","moa":"IBAT","graph1":"Genetic Disease","graph2":"Approved","graph3":"Albireo Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Albireo Pharma \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Albireo Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Maralixibat","moa":"IBAT","graph1":"Genetic Disease","graph2":"Approved","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Mirum Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Mirum Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Omaveloxolone","moa":"Nrf2","graph1":"Genetic Disease","graph2":"Approved","graph3":"Reata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Reata Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Reata Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Crizanlizumab","moa":"P-selectin","graph1":"Genetic Disease","graph2":"Approved","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Not Applicable"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Berotralstat","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioCryst Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Cystic Fibrosis Foundation","sponsor":"Royalty Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Acquisition","leadProduct":"Ivacaftor","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Cystic Fibrosis Foundation","amount2":0.65000000000000002,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0.65000000000000002,"dosageForm":"","sponsorNew":"Cystic Fibrosis Foundation \/ Royalty Pharma","highestDevelopmentStatusID":"12","companyTruncated":"Cystic Fibrosis Foundation \/ Royalty Pharma"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Maralixibat","moa":"IBAT","graph1":"Genetic Disease","graph2":"Approved","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Mirum Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Mirum Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Libmeldy","moa":"ARSA gene","graph1":"Genetic Disease","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"intravenous infusion","sponsorNew":"Orchard Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2020","type":"Not Applicable","leadProduct":"Vosoritide","moa":"FGFR3","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Film-coated Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Lanadelumab","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"pre-filled syringe","sponsorNew":"Takeda Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Takeda Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Maralixibat","moa":"IBAT","graph1":"Genetic Disease","graph2":"Approved","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Mirum Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Mirum Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Odevixibat","moa":"IBAT","graph1":"Genetic Disease","graph2":"Approved","graph3":"Albireo Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"capsule","sponsorNew":"Albireo Pharma \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Albireo Pharma \/ Not Applicable"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Berotralstat","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioCryst Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Lumasiran","moa":"Glycolate oxidase","graph1":"Genetic Disease","graph2":"Approved","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Alnylam Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Lonafarnib","moa":"Ftase","graph1":"Genetic Disease","graph2":"Approved","graph3":"Eiger BioPharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Eiger BioPharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Eiger BioPharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Eiger BioPharmaceuticals","sponsor":"The Progeria Research Foundation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Divestment","leadProduct":"Lonafarnib","moa":"Ftase","graph1":"Genetic Disease","graph2":"Approved","graph3":"Eiger BioPharmaceuticals","amount2":0.10000000000000001,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Oral","sponsorNew":"Eiger BioPharmaceuticals \/ The Progeria Research Foundation","highestDevelopmentStatusID":"12","companyTruncated":"Eiger BioPharmaceuticals \/ The Progeria Research Foundation"},{"orgOrder":0,"company":"Reata Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Omaveloxolone","moa":"Nrf2","graph1":"Genetic Disease","graph2":"Approved","graph3":"Reata Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Reata Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Reata Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Berotralstat","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioCryst Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"capsule","sponsorNew":"BioCryst Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioCryst Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"3W Fund Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Large molecule","year":"2020","type":"Series E Financing","leadProduct":"Idursulfase beta","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"CANbridge Pharmaceuticals","amount2":0.040000000000000001,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Intravenous Infusion","sponsorNew":"CANbridge Pharmaceuticals \/ 3W Fund Management","highestDevelopmentStatusID":"12","companyTruncated":"CANbridge Pharmaceuticals \/ 3W Fund Management"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Royalty Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Funding","leadProduct":"Berotralstat","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioCryst Pharmaceuticals","amount2":0.33000000000000002,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0.33000000000000002,"dosageForm":"","sponsorNew":"BioCryst Pharmaceuticals \/ Royalty Pharma","highestDevelopmentStatusID":"12","companyTruncated":"BioCryst Pharmaceuticals \/ Royalty Pharma"},{"orgOrder":0,"company":"PANTHERx Rare Pharmacy","sponsor":"Alnylam Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Partnership","leadProduct":"Lumasiran","moa":"Glycolate oxidase","graph1":"Genetic Disease","graph2":"Approved","graph3":"PANTHERx Rare Pharmacy","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"PANTHERx Rare Pharmacy \/ Alnylam Pharmaceuticals","highestDevelopmentStatusID":"12","companyTruncated":"PANTHERx Rare Pharmacy \/ Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"Lupin Ltd","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Penicillamine","moa":"Copper","graph1":"Genetic Disease","graph2":"Approved","graph3":"Lupin Ltd","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Lupin Ltd \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Lupin Ltd \/ Not Applicable"},{"orgOrder":0,"company":"Amicus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Cipaglucosidase alfa","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved","graph3":"Amicus Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Amicus Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Amicus Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Lumasiran","moa":"Glycolate oxidase","graph1":"Genetic Disease","graph2":"Approved","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Alnylam Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Fresenius Kabi AG","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Icatibant Acetate","moa":"Bradykinin B2 receptor","graph1":"Genetic Disease","graph2":"Approved","graph3":"Fresenius Kabi AG","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Fresenius Kabi AG \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Fresenius Kabi AG \/ Not Applicable"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Emicizumab","moa":"Factor IX\/Factor X","graph1":"Genetic Disease","graph2":"Approved","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"F. Hoffmann-La Roche \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"F. Hoffmann-La Roche \/ Not Applicable"},{"orgOrder":0,"company":"Nektar Therapeutics","sponsor":"Healthcare Royalty","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Agreement","leadProduct":"Antihemophilic Factor (Recombinant), PEGylated","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Nektar Therapeutics","amount2":0.14999999999999999,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0.14999999999999999,"dosageForm":"Injection","sponsorNew":"Nektar Therapeutics \/ Healthcare Royalty","highestDevelopmentStatusID":"12","companyTruncated":"Nektar Therapeutics \/ Healthcare Royalty"},{"orgOrder":0,"company":"Bluebird Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Betibeglogene autotemcel","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Bluebird Bio","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Bluebird Bio \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Bluebird Bio \/ Not Applicable"},{"orgOrder":0,"company":"Albireo Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Hepatology (Liver, Pancreatic, Gall Bladder)","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Odevixibat","moa":"IBAT","graph1":"Genetic Disease","graph2":"Approved","graph3":"Albireo Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"capsule","sponsorNew":"Albireo Pharma \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Albireo Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Orsini Specialty Pharmacy","sponsor":"Alnylam Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Partnership","leadProduct":"Lumasiran","moa":"Glycolate oxidase","graph1":"Genetic Disease","graph2":"Approved","graph3":"Orsini Specialty Pharmacy","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Orsini Specialty Pharmacy \/ Alnylam Pharmaceuticals","highestDevelopmentStatusID":"12","companyTruncated":"Orsini Specialty Pharmacy \/ Alnylam Pharmaceuticals"},{"orgOrder":0,"company":"Prime Therapeutics","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2020","type":"Agreement","leadProduct":"Antihemophilic factor recombinant","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Approved","graph3":"Prime Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Prime Therapeutics \/ Takeda","highestDevelopmentStatusID":"12","companyTruncated":"Prime Therapeutics \/ Takeda"},{"orgOrder":0,"company":"Alnylam Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Vutrisiran","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Alnylam Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Alnylam Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Alnylam Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"Valoctocogene Roxaparvovec","moa":"Blood coagulation factor","graph1":"Genetic Disease","graph2":"Approved","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"BioMarin Pharmaceutical \/ Not Applicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA positive","graph1":"Genetic Disease","graph2":"Approved","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Biogen \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Biogen \/ Not Applicable"},{"orgOrder":0,"company":"Diurnal Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Hydrocortisone","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Diurnal Group","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Diurnal Group \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Diurnal Group \/ Not Applicable"},{"orgOrder":0,"company":"Johnson & Johnson Innovative Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Daratumumab","moa":"CD38","graph1":"Genetic Disease","graph2":"Approved","graph3":"Johnson & Johnson Innovative Medicine","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Johnson & Johnson Innovative Medicine \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Johnson & Johnson Innovative Medicine \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Genpharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"Autologous CD34+ cell enriched HSPC","moa":"ARSA gene","graph1":"Genetic Disease","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Dispersion for infusion","sponsorNew":"Orchard Therapeutics \/ GenPharm","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ GenPharm"},{"orgOrder":0,"company":"Diurnal Group","sponsor":"Citrine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Small molecule","year":"2021","type":"Licensing Agreement","leadProduct":"Hydrocortisone","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Diurnal Group","amount2":0.01,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"Capsule","sponsorNew":"Diurnal Group \/ Citrine","highestDevelopmentStatusID":"12","companyTruncated":"Diurnal Group \/ Citrine"},{"orgOrder":0,"company":"Slayback Pharma","sponsor":"Sandoz B2B","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2021","type":"Licensing Agreement","leadProduct":"Icatibant Acetate","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Slayback Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Slayback Pharma \/ Sandoz","highestDevelopmentStatusID":"12","companyTruncated":"Slayback Pharma \/ Sandoz"},{"orgOrder":0,"company":"GC Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Idursulfase beta","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"GC Pharma","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebroventricular Infusion","sponsorNew":"GC Pharma \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"GC Pharma \/ Not Applicable"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Voxelotor","moa":"HbA positive","graph1":"Genetic Disease","graph2":"Approved","graph3":"Global Blood Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Global Blood Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Global Blood Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2021","type":"Public Offering","leadProduct":"Autologous CD34+ cell enriched HSPC","moa":"ARSA gene","graph1":"Genetic Disease","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Dispersion for infusion","sponsorNew":"Orchard Therapeutics \/ RA Capital Management","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ RA Capital Management"},{"orgOrder":0,"company":"Diurnal Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Hydrocortisone","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Diurnal Group","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Diurnal Group \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Diurnal Group \/ Not Applicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Ataluren","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral suspension","sponsorNew":"PTC Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"PTC Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Protalix BioTherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ISRAEL","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Pegunigalsidase alfa","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Approved","graph3":"Protalix BioTherapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Protalix BioTherapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Protalix BioTherapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"Libmeldy","moa":"ARSA gene","graph1":"Genetic Disease","graph2":"Approved","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"intravenous infusion","sponsorNew":"Orchard Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Orchard Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Anakinra","moa":"IL-1 receptor","graph1":"Genetic Disease","graph2":"Approved","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Swedish Orphan Biovitrum AB \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Swedish Orphan Biovitrum AB \/ Not Applicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Sanofi","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Efanesoctocog alfa","moa":"VWF","graph1":"Genetic Disease","graph2":"Approved","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Sanofi \/ Bioverativ","highestDevelopmentStatusID":"12","companyTruncated":"Sanofi \/ Bioverativ"},{"orgOrder":0,"company":"Genentech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Risdiplam","moa":"SMN2 splicing","graph1":"Genetic Disease","graph2":"Approved","graph3":"Genentech","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral solution","sponsorNew":"Genentech \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Genentech \/ Not Applicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Casimersen","moa":"Exon 45","graph1":"Genetic Disease","graph2":"Approved","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Sarepta Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Sarepta Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2021","type":"Not Applicable",
