[{"orgOrder":0,"company":"LifeMax Laboratories","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"LM-030","moa":"KLK7\/ELA2","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"LifeMax Laboratories","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Ointment","sponsorNew":"LifeMax Laboratories \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"LifeMax Laboratories \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Sepofarsen","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"ProQR Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"ProQR Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"TARA-002","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Protara Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Protara Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Protara Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"TARA-002","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Protara Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Protara Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Protara Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Cowen","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Public Offering","leadProduct":"TARA-002","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Protara Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Protara Therapeutics \/ Cowen","highestDevelopmentStatusID":"9","companyTruncated":"Protara Therapeutics \/ Cowen"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Sepofarsen","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"ProQR Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"ProQR Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"Sepofarsen","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"ProQR Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"ProQR Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Large molecule","year":"2021","type":"Not Applicable","leadProduct":"Sepofarsen","moa":"CEP290","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"ProQR Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"ProQR Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Disc medicine","sponsor":"OrbiMed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2021","type":"Series B Financing","leadProduct":"Bitopertin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.089999999999999997,"dosageForm":"Oral","sponsorNew":"Disc medicine \/ OrbiMed","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ OrbiMed"},{"orgOrder":0,"company":"Retrotope","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Linoleic Acid","moa":"Lipid peroxidation","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Retrotope","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Retrotope \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Retrotope \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Orpharm","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2021","type":"Agreement","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Orpharm","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Orpharm"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Sepofarsen","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"ProQR Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"ProQR Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Neopharma","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2022","type":"Agreement","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Neopharm","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Neopharm"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Sepofarsen","moa":"CEP290 gene","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"ProQR Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"ProQR Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"TARA-002","moa":"Th1","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Protara Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Protara Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Protara Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"KemPharm","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","productType":"Small molecule","year":"2022","type":"Acquisition","leadProduct":"Arimoclomol","moa":"HSP","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ KemPharm","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ KemPharm"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Endo Ventures Limited","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2022","type":"Agreement","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Endo Ventures Limited","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Endo Ventures Limited"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Large molecule","year":"2022","type":"Not Applicable","leadProduct":"Sepofarsen","moa":"Splicing","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"ProQR Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"ProQR Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Bitopertin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Disc medicine \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"QRX003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Gemini Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2022","type":"Merger","leadProduct":"Bitopertin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.17999999999999999,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.17999999999999999,"dosageForm":"","sponsorNew":"Disc medicine \/ Gemini Therapeutics","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Gemini Therapeutics"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Bitopertin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Disc medicine \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Not Applicable"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Bain Capital Life Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2023","type":"Private Placement","leadProduct":"Bitopertin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.059999999999999998,"dosageForm":"","sponsorNew":"Disc medicine \/ Bain Capital Life Sciences","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Bain Capital Life Sciences"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"QRX003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"TARA-002","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Protara Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Protara Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Protara Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"QRX003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2023","type":"Public Offering","leadProduct":"Bitopertin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.16,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Morgan Stanley","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Morgan Stanley"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2023","type":"Public Offering","leadProduct":"Bitopertin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.14000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.16,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Morgan Stanley","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Morgan Stanley"},{"orgOrder":0,"company":"Mandos Health","sponsor":"INPDR","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2023","type":"Collaboration","leadProduct":"Adrabetadex","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Mandos Health","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Mandos Health \/ INPDR","highestDevelopmentStatusID":"9","companyTruncated":"Mandos Health \/ INPDR"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2023","type":"Public Offering","leadProduct":"Bitopertin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.16,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.16,"dosageForm":"Tablet","sponsorNew":"Disc medicine \/ Morgan Stanley","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Morgan Stanley"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Laboratoires Thea","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Large molecule","year":"2023","type":"Divestment","leadProduct":"Sepofarsen","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"ProQR Therapeutics","amount2":0.16,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.19,"dosageForm":"Injectable\/Injection","sponsorNew":"ProQR Therapeutics \/ Laboratoires Thea","highestDevelopmentStatusID":"9","companyTruncated":"ProQR Therapeutics \/ Laboratoires Thea"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"QRX003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Oncology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"TARA-002","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Protara Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Protara Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Protara Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Laboratoires Thea","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Large molecule","year":"2023","type":"Divestment","leadProduct":"Sepofarsen","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"ProQR Therapeutics","amount2":0.19,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.19,"dosageForm":"Injection","sponsorNew":"ProQR Therapeutics \/ Laboratoires Thea","highestDevelopmentStatusID":"9","companyTruncated":"ProQR Therapeutics \/ Laboratoires Thea"},{"orgOrder":0,"company":"LadRx","sponsor":"XOMA","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Agreement","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"LadRx","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0.059999999999999998,"dosageForm":"Capsule","sponsorNew":"LadRx \/ XOMA","highestDevelopmentStatusID":"9","companyTruncated":"LadRx \/ XOMA"},{"orgOrder":0,"company":"Avicanna","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"CANADA","productType":"Small molecule","year":"2024","type":"Licensing Agreement","leadProduct":"Cannabidiol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Avicanna","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Cream","sponsorNew":"Avicanna \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Avicanna \/ Undisclosed"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"QRX003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"SKINVISIBLE PHARMACEUTICALS INC","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"QRX003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"SKINVISIBLE PHARMACEUTICALS INC","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"SKINVISIBLE PHARMACEUTICALS INC \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"SKINVISIBLE PHARMACEUTICALS INC \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"QRX003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Galectin Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"GEORGIA","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Belapectin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Galectin Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Galectin Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Galectin Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Peptide","year":"2024","type":"Not Applicable","leadProduct":"Elamipretide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Stealth Biotherapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Stealth Biotherapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Peptide","year":"2024","type":"Not Applicable","leadProduct":"Elamipretide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Stealth Biotherapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Stealth Biotherapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"QRX003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Peptide","year":"2024","type":"Not Applicable","leadProduct":"Elamipretide","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Stealth Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Stealth Biotherapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Stealth Biotherapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"QRX003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"}]
Find Clinical Drug Development Pipelines & Deals | PipelineProspector
Filters
×
FILTER:
Company Name
Year
DEALS // DEV.
Country
Sponsor
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target
Reset All
Sanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.
