[{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Ganaxolone","moa":"GABAA receptor","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Marinus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Suspension","sponsorNew":"Marinus Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Marinus Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Foundation for Angelman Syndrome Therapeutics","sponsor":"Lixte Biotechnology","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Collaboration","leadProduct":"LB-100","moa":"PP2A","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Foundation for Angelman Syndrome Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Foundation for Angelman Syndrome Therapeutics \/ Lixte Biotechnology","highestDevelopmentStatusID":"4","companyTruncated":"Foundation for Angelman Syndrome Therapeutics \/ Lixte Biotechnology"},{"orgOrder":0,"company":"Biom Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"BIO017","moa":"TRPV1","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Biom Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biom Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Biom Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"University of North Carolina","sponsor":"AskBio","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"University of North Carolina","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of North Carolina \/ AskBio","highestDevelopmentStatusID":"4","companyTruncated":"University of North Carolina \/ AskBio"},{"orgOrder":0,"company":"Zylka Lab","sponsor":"University of North Carolina","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Zylka Lab","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Zylka Lab \/ University of North Carolina","highestDevelopmentStatusID":"4","companyTruncated":"Zylka Lab \/ University of North Carolina"},{"orgOrder":0,"company":"University of North Carolina","sponsor":"AskBio","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"University of North Carolina","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of North Carolina \/ AskBio","highestDevelopmentStatusID":"4","companyTruncated":"University of North Carolina \/ AskBio"},{"orgOrder":0,"company":"Axovia Therapeutics","sponsor":"ALSA Ventures","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2023","type":"Acquisition","leadProduct":"AXV-101","moa":"BBS1","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Axovia Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Axovia Therapeutics \/ ALSA Ventures","highestDevelopmentStatusID":"4","companyTruncated":"Axovia Therapeutics \/ ALSA Ventures"},{"orgOrder":0,"company":"Charles River Laboratories, Inc","sponsor":"Axovia Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Collaboration","leadProduct":"AXV-101","moa":"BBS1","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Charles River Laboratories, Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Charles River Laboratories, Inc \/ Axovia Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Charles River Laboratories, Inc \/ Axovia Therapeutics"},{"orgOrder":0,"company":"Rhythm Pharmaceuticals","sponsor":"Axovia Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Collaboration","leadProduct":"AXV-101","moa":"BBS1","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Rhythm Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Rhythm Pharmaceuticals \/ Axovia Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Rhythm Pharmaceuticals \/ Axovia Therapeutics"},{"orgOrder":0,"company":"Axovia Therapeutics","sponsor":"A Race Against Blindness","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2024","type":"Funding","leadProduct":"AXV-101","moa":"BBS1","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Axovia Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Axovia Therapeutics \/ A Race Against Blindness","highestDevelopmentStatusID":"4","companyTruncated":"Axovia Therapeutics \/ A Race Against Blindness"},{"orgOrder":0,"company":"Neurogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"CLN7","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neurogene \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Neurogene \/ Inapplicable"},{"orgOrder":0,"company":"Graphite Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"GPH102","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Graphite Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Graphite Bio \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Graphite Bio \/ Inapplicable"},{"orgOrder":0,"company":"Adverum Biotechnologies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"AAV.7m8-L-opsin","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Adverum Biotechnologies","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"Adverum Biotechnologies \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Adverum Biotechnologies \/ Inapplicable"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Trehalose","moa":"Amyloid-beta","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Seelos Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Seelos Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Seelos Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Leaps by Bayer","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Aerosol","sponsorNew":"ReCode Therapeutics \/ Leaps by Bayer","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Leaps by Bayer"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"ReCode Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"ReCode Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"ReCode Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"ReCode Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Catalent Pharma Solutions","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Partnership","leadProduct":"TSHA-118","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Taysha Gene Therapies \/ Catalent Pharma Solutions","highestDevelopmentStatusID":"4","companyTruncated":"Taysha Gene Therapies \/ Catalent Pharma Solutions"},{"orgOrder":0,"company":"Neurogene","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Neurogene","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Neurogene \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Neurogene \/ Inapplicable"},{"orgOrder":0,"company":"WuXi Biologics","sponsor":"Toregem BioPharma","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody","year":"2022","type":"Partnership","leadProduct":"TRG035","moa":"USAG1","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"WuXi Biologics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"WuXi Biologics \/ Toregem BioPharma","highestDevelopmentStatusID":"4","companyTruncated":"WuXi Biologics \/ Toregem BioPharma"},{"orgOrder":0,"company":"Modalis Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"MDL-101","moa":"Laminin-alpha-1 protein","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Modalis Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Modalis Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Modalis Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Institute for Life Changing Medicines","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Collaboration","leadProduct":"mRNA-3351","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Moderna Therapeutics \/ Institute for Life Changing Medicines","highestDevelopmentStatusID":"4","companyTruncated":"Moderna Therapeutics \/ Institute for Life Changing Medicines"},{"orgOrder":0,"company":"Tetra BioPharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CANADA","productType":"Controlled Substance","year":"2020","type":"Inapplicable","leadProduct":"Tetrahydrocannabinol","moa":"||CB1\/CB2 receptor","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Tetra BioPharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Cream","sponsorNew":"Tetra BioPharma \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Tetra BioPharma \/ Inapplicable"},{"orgOrder":0,"company":"AlgoTherapeutix","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Amitriptyline","moa":"5-HT transporter","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"AlgoTherapeutix","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"AlgoTherapeutix \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"AlgoTherapeutix \/ Inapplicable"},{"orgOrder":0,"company":"Charles River Laboratories, Inc","sponsor":"FOXG1 Research Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Charles River Laboratories, Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Charles River Laboratories, Inc \/ FOXG1 Research Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Charles River Laboratories, Inc \/ FOXG1 Research Foundation"},{"orgOrder":0,"company":"Papillon Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"PPL-002","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Papillon Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Papillon Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Papillon Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Papillon Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"PPL-002","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Papillon Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Papillon Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Papillon Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"IPS Heart","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"ISX9-CPC","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"IPS Heart","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"IPS Heart \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"IPS Heart \/ Inapplicable"},{"orgOrder":0,"company":"RespireRx Pharmaceuticals","sponsor":"University College London","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Collaboration","leadProduct":"Ampakine","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"RespireRx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"RespireRx Pharmaceuticals \/ University College London","highestDevelopmentStatusID":"4","companyTruncated":"RespireRx Pharmaceuticals \/ University College London"},{"orgOrder":0,"company":"Codexis","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"CDX-6512","moa":"Enzyme Replacement","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Codexis","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Inhalation","sponsorNew":"Codexis \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Codexis \/ Inapplicable"},{"orgOrder":0,"company":"Meta Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"HONG KONG","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"META-001-PH","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Meta Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Meta Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Meta Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"PBKR03","moa":"Galactosylceramidase","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intra-cisterna magna Injection","sponsorNew":"Passage Bio \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Passage Bio \/ Inapplicable"},{"orgOrder":0,"company":"Variant","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"VAR002","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Variant","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"Variant \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Variant \/ Inapplicable"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Massachusetts Eye and Ear and Harvard Medical School","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Agreement","leadProduct":"OPGx-003","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Opus Genetics \/ Massachusetts Eye and Ear and Harvard Medical School","highestDevelopmentStatusID":"4","companyTruncated":"Opus Genetics \/ Massachusetts Eye and Ear and Harvard Medical School"},{"orgOrder":0,"company":"Andelyn Biosciences","sponsor":"Hubble Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Partnership","leadProduct":"HUB-101","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Andelyn Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Andelyn Biosciences \/ Hubble Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Andelyn Biosciences \/ Hubble Therapeutics"},{"orgOrder":0,"company":"Medivir","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SWEDEN","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"MIV-711","moa":"Cathepsin-K","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Medivir","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Medivir \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Medivir \/ Inapplicable"},{"orgOrder":0,"company":"Myto Therapeutics","sponsor":"University of California","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Monomethyl Fumarate Prodrug","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Myto Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Myto Therapeutics \/ University of California","highestDevelopmentStatusID":"4","companyTruncated":"Myto Therapeutics \/ University of California"},{"orgOrder":0,"company":"UAB Institut de Neuroci\u00e8ncies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SPAIN","productType":"Controlled Substance","year":"2023","type":"Inapplicable","leadProduct":"Cannabidiol","moa":"CB1\/2 receptor","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"UAB Institut de Neuroci\u00e8ncies","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"UAB Institut de Neuroci\u00e8ncies \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"UAB Institut de Neuroci\u00e8ncies \/ Inapplicable"},{"orgOrder":0,"company":"London Research & Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CANADA","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Cannabidiol Sulphate","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"London Research & Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"London Research & Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"London Research & Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"HMI-204","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Homology Medicines","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Homology Medicines \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Homology Medicines \/ Inapplicable"},{"orgOrder":0,"company":"Release Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","productType":"Undisclosed","year":"2024","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Release Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Release Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Release Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Affinia Therapeutics","sponsor":"Lonza Group","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Agreement","leadProduct":"Anc80L65","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Affinia Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Affinia Therapeutics \/ Lonza Group","highestDevelopmentStatusID":"4","companyTruncated":"Affinia Therapeutics \/ Lonza Group"},{"orgOrder":0,"company":"Code Bio","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Code Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Code Bio \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"4","companyTruncated":"Code Bio \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"M6P Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"M011","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"M6P Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"M6P Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"M6P Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"M6P Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"M011","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"M6P Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"M6P Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"M6P Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Cyprium Therapeutics","sponsor":"National Institute of Neurological Disorders and Stroke","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Funding","leadProduct":"AAV-ATP7A","moa":"||Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Cyprium Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Cyprium Therapeutics \/ National Institute of Neurological Disorders and Stroke","highestDevelopmentStatusID":"4","companyTruncated":"Cyprium Therapeutics \/ National Institute of Neurological Disorders and Stroke"},{"orgOrder":0,"company":"The Texas A&M University System","sponsor":"Engrail Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Licensing Agreement","leadProduct":"Elesclomol","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"The Texas A&M University System","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"The Texas A&M University System \/ Engrail Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"The Texas A&M University System \/ Engrail Therapeutics"},{"orgOrder":0,"company":"Selecta Biosciences","sponsor":"AskBio","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"ImmTOR","moa":"||Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Selecta Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Selecta Biosciences \/ AskBio","highestDevelopmentStatusID":"4","companyTruncated":"Selecta Biosciences \/ AskBio"},{"orgOrder":0,"company":"Selecta Biosciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"ImmTOR","moa":"||Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Selecta Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Selecta Biosciences \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Selecta Biosciences \/ Inapplicable"},{"orgOrder":0,"company":"AskBio","sponsor":"Selecta Biosciences","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"ImmTOR","moa":"||Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"AskBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AskBio \/ Selecta Biosciences","highestDevelopmentStatusID":"4","companyTruncated":"AskBio \/ Selecta Biosciences"},{"orgOrder":0,"company":"Cellectis","sponsor":"Primera Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Cellectis","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cellectis \/ Primera Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Cellectis \/ Primera Therapeutics"},{"orgOrder":0,"company":"Cyclerion Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Zagociguat","moa":"sGC","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Cyclerion Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cyclerion Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Cyclerion Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Cyclerion Therapeutics","sponsor":"Peter Hecht","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Agreement","leadProduct":"Zagociguat","moa":"sGC","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Cyclerion Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Cyclerion Therapeutics \/ Peter Hecht","highestDevelopmentStatusID":"4","companyTruncated":"Cyclerion Therapeutics \/ Peter Hecht"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Regenxbio \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Regenxbio \/ Inapplicable"},{"orgOrder":0,"company":"UT Southwestern Medical Center","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"UT Southwestern Medical Center","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Injection","sponsorNew":"UT Southwestern Medical Center \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"UT Southwestern Medical Center \/ Inapplicable"},{"orgOrder":0,"company":"Modalis Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"MDL-101","moa":"Laminin-alpha-1 protein","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Modalis Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Modalis Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Modalis Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Modalis Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"MDL-101","moa":"Laminin-alpha-1 protein","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Modalis Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Modalis Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Modalis Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Modalis Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"MDL-101","moa":"Laminin-alpha-1 protein","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Modalis Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Modalis Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Modalis Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Genpharm","sponsor":"Quoin Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.A.E","productType":"Other Small Molecule","year":"2021","type":"Licensing Agreement","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Genpharm","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Lotion","sponsorNew":"Genpharm \/ Quoin Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Genpharm \/ Quoin Pharmaceuticals"},{"orgOrder":0,"company":"AFT Pharmaceuticals","sponsor":"Quoin Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"NEW ZEALAND","productType":"Other Small Molecule","year":"2021","type":"Licensing Agreement","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"AFT Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Lotion","sponsorNew":"AFT Pharmaceuticals \/ Quoin Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"AFT Pharmaceuticals \/ Quoin Pharmaceuticals"},{"orgOrder":0,"company":"Erkim","sponsor":"Quoin Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"TURKEY","productType":"Other Small Molecule","year":"2022","type":"Agreement","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Erkim","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Lotion","sponsorNew":"Erkim \/ Quoin Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Erkim \/ Quoin Pharmaceuticals"},{"orgOrder":0,"company":"Erkim","sponsor":"Quoin Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"TURKEY","productType":"Other Small Molecule","year":"2022","type":"Agreement","leadProduct":"QRX003","moa":"Serine protease","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Erkim","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical Lotion","sponsorNew":"Erkim \/ Quoin Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Erkim \/ Quoin Pharmaceuticals"},{"orgOrder":0,"company":"Aeterna Zentaris","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Large Molecule","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Alpha-synuclein","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Aeterna Zentaris","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Aeterna Zentaris \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Aeterna Zentaris \/ Inapplicable"},{"orgOrder":0,"company":"Aeterna Zentaris","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"AQP-4","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Aeterna Zentaris","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Aeterna Zentaris \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Aeterna Zentaris \/ Inapplicable"},{"orgOrder":0,"company":"Actelion Pharmaceuticals Ltd","sponsor":"Beyond Batten Disease Foundation","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2021","type":"Collaboration","leadProduct":"Miglustat","moa":"||Glucosylceramide synthase","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Actelion Pharmaceuticals Ltd","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Actelion Pharmaceuticals Ltd \/ Beyond Batten Disease Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Actelion Pharmaceuticals Ltd \/ Beyond Batten Disease Foundation"},{"orgOrder":0,"company":"Latus Bio","sponsor":"8VC","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Latus Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Latus Bio \/ 8VC","highestDevelopmentStatusID":"4","companyTruncated":"Latus Bio \/ 8VC"},{"orgOrder":0,"company":"Polaryx Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"PLX-300","moa":"PPA-alpha receptor","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Polaryx Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Polaryx Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Polaryx Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Evox Therapeutics","sponsor":"Redmile Group","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2021","type":"Series C Financing","leadProduct":"HSDB 4295","moa":"5-HT\/norepinephrine reuptake","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Evox Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Evox Therapeutics \/ Redmile Group","highestDevelopmentStatusID":"4","companyTruncated":"Evox Therapeutics \/ Redmile Group"},{"orgOrder":0,"company":"Evox Therapeutics","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"UNITED KINGDOM","productType":"Protein","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Evox Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Evox Therapeutics \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"4","companyTruncated":"Evox Therapeutics \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Rezolute","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2024","type":"Inapplicable","leadProduct":"Ersodetug","moa":"Insulin allosteric site receptor","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Rezolute \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Rezolute \/ Inapplicable"},{"orgOrder":0,"company":"Glycomine","sponsor":"ImmuneID","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Series B Financing","leadProduct":"GLM101","moa":"Mannose-1-phosphate","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Glycomine","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Glycomine \/ ImmuneID","highestDevelopmentStatusID":"4","companyTruncated":"Glycomine \/ ImmuneID"},{"orgOrder":0,"company":"Metagenomi","sponsor":"Moderna Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Termination","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Metagenomi","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Metagenomi \/ Moderna Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Metagenomi \/ Moderna Therapeutics"},{"orgOrder":0,"company":"TargED Biopharmaceuticals","sponsor":"Andera Partners","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Protein","year":"2022","type":"Series A Financing","leadProduct":"Microlyse","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"TargED Biopharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"TargED Biopharmaceuticals \/ Andera Partners","highestDevelopmentStatusID":"4","companyTruncated":"TargED Biopharmaceuticals \/ Andera Partners"},{"orgOrder":0,"company":"GC Biopharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SOUTH KOREA","productType":"Other Large Molecule","year":"2023","type":"Inapplicable","leadProduct":"GC1126A","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"GC Biopharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"GC Biopharma \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"GC Biopharma \/ Inapplicable"},{"orgOrder":0,"company":"Strm.Bio","sponsor":"National Center for Advancing Translational Sciences","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Strm.Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Strm.Bio \/ National Center for Advancing Translational Sciences","highestDevelopmentStatusID":"4","companyTruncated":"Strm.Bio \/ National Center for Advancing Translational Sciences"},{"orgOrder":0,"company":"Resilience","sponsor":"Mayo Clinic","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Resilience","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Resilience \/ Mayo Clinic","highestDevelopmentStatusID":"4","companyTruncated":"Resilience \/ Mayo Clinic"},{"orgOrder":0,"company":"Epigenic Therapeutics","sponsor":"Morningside Ventures","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Undisclosed","year":"2022","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Epigenic Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Epigenic Therapeutics \/ Morningside Ventures","highestDevelopmentStatusID":"4","companyTruncated":"Epigenic Therapeutics \/ Morningside Ventures"},{"orgOrder":0,"company":"Nanoform","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"FINLAND","productType":"Undisclosed","year":"2024","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Nanoform","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Nanoform \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"4","companyTruncated":"Nanoform \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"KBio","sponsor":"ZabBio","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Partnership","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"KBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"KBio \/ ZabBio","highestDevelopmentStatusID":"4","companyTruncated":"KBio \/ ZabBio"},{"orgOrder":0,"company":"KBio","sponsor":"Leaf Expression Systems","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"KBio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"KBio \/ Leaf Expression Systems","highestDevelopmentStatusID":"4","companyTruncated":"KBio \/ Leaf Expression Systems"},{"orgOrder":0,"company":"Affinia Therapeutics","sponsor":"EcoR1 Capital","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Affinia Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Affinia Therapeutics \/ EcoR1 Capital","highestDevelopmentStatusID":"4","companyTruncated":"Affinia Therapeutics \/ EcoR1 Capital"},{"orgOrder":0,"company":"Affinia Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Affinia Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Affinia Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Affinia Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Avilar Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Avilar Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Avilar Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Avilar Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Avilar Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Avilar Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Avilar Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Avilar Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Ensoma","sponsor":"5Am Ventures","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Large Molecule","year":"2021","type":"Series A Financing","leadProduct":"Undisclosed","moa":"||Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Ensoma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Ensoma \/ 5Am Ventures","highestDevelopmentStatusID":"4","companyTruncated":"Ensoma \/ 5Am Ventures"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Pfizer Inc \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Pfizer Inc \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Pfizer Inc \/ Alexion Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Pfizer Inc \/ Alexion Pharmaceuticals"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"University College Cork","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibiotic","year":"2024","type":"Agreement","leadProduct":"Sirolimus","moa":"mTOR","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Quoin Pharmaceuticals \/ University College Cork","highestDevelopmentStatusID":"4","companyTruncated":"Quoin Pharmaceuticals \/ University College Cork"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"ProQR Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"ProQR Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"ProQR Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Everads Therapy","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"ISRAEL","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Everads Therapy","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"Everads Therapy \/ Undisclosed","highestDevelopmentStatusID":"4","companyTruncated":"Everads Therapy \/ Undisclosed"},{"orgOrder":0,"company":"Generation Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Generation Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"Generation Bio \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Generation Bio \/ Inapplicable"},{"orgOrder":0,"company":"Generation Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Generation Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"Generation Bio \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Generation Bio \/ Inapplicable"},{"orgOrder":0,"company":"Surrozen","sponsor":"Boehringer Ingelheim GmbH","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2022","type":"Collaboration","leadProduct":"SZN-413","moa":"Wnt signalling pathway","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Surrozen","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Surrozen \/ Boehringer Ingelheim GmbH","highestDevelopmentStatusID":"4","companyTruncated":"Surrozen \/ Boehringer Ingelheim GmbH"},{"orgOrder":0,"company":"OBiO Technology","sponsor":"Refreshgene Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Cell and Gene therapy","year":"2023","type":"Agreement","leadProduct":"RRG-001","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"OBiO Technology","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"OBiO Technology \/ Refreshgene Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"OBiO Technology \/ Refreshgene Therapeutics"},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Beacon Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Agreement","leadProduct":"AAV204","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Abeona Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Abeona Therapeutics \/ Beacon Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Abeona Therapeutics \/ Beacon Therapeutics"},{"orgOrder":0,"company":"Ocugen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ocugen \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Ocugen \/ Inapplicable"},{"orgOrder":0,"company":"Herophilus","sponsor":"Rett Syndrome Research Trust","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Funding","leadProduct":"HRP-12975","moa":"MECP2","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Herophilus","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Herophilus \/ Rett Syndrome Research Trust","highestDevelopmentStatusID":"4","companyTruncated":"Herophilus \/ Rett Syndrome Research Trust"},{"orgOrder":0,"company":"Marvel Biosciences","sponsor":"iBraiN Institute","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"CANADA","productType":"Other Small Molecule","year":"2024","type":"Collaboration","leadProduct":"Istradefylline","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Marvel Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Marvel Biosciences \/ iBraiN Institute","highestDevelopmentStatusID":"4","companyTruncated":"Marvel Biosciences \/ iBraiN Institute"},{"orgOrder":0,"company":"Alcyone Therapeutics","sponsor":"RTW Investments","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Funding","leadProduct":"ACTX-101","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Alcyone Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alcyone Therapeutics \/ RTW Investments","highestDevelopmentStatusID":"4","companyTruncated":"Alcyone Therapeutics \/ RTW Investments"},{"orgOrder":0,"company":"Unravel Biosciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"RVL001","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Unravel Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Unravel Biosciences \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Unravel Biosciences \/ Inapplicable"},{"orgOrder":0,"company":"Vyant Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Vyant Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vyant Bio \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Vyant Bio \/ Inapplicable"},{"orgOrder":0,"company":"Ideaya Biosciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Darovasertib","moa":"PKC","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Ideaya Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Ideaya Biosciences \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Ideaya Biosciences \/ Inapplicable"},{"orgOrder":0,"company":"AAVantgarde Bio","sponsor":"Atlas Venture","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"ITALY","productType":"Undisclosed","year":"2023","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"AAVantgarde Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"AAVantgarde Bio \/ Atlas Venture","highestDevelopmentStatusID":"4","companyTruncated":"AAVantgarde Bio \/ Atlas Venture"},{"orgOrder":0,"company":"Usher III Initiative","sponsor":"Foundation Fighting Blindness","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Funding","leadProduct":"BF844","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Usher III Initiative","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"Usher III Initiative \/ Foundation Fighting Blindness","highestDevelopmentStatusID":"4","companyTruncated":"Usher III Initiative \/ Foundation Fighting Blindness"},{"orgOrder":0,"company":"Axplora","sponsor":"Sensorion","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"GERMANY","productType":"Cell and Gene therapy","year":"2020","type":"Agreement","leadProduct":"OTOF-GT","moa":"||OTOF","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Axplora","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Axplora \/ Sensorion","highestDevelopmentStatusID":"4","companyTruncated":"Axplora \/ Sensorion"},{"orgOrder":0,"company":"Akouos","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"AAVAnc80-antiVEGF Vector","moa":"VEGF receptor","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Akouos","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intracochlear","sponsorNew":"Akouos \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Akouos \/ Inapplicable"},{"orgOrder":0,"company":"Genflow Biosciences","sponsor":"EXO Biologics","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2024","type":"Collaboration","leadProduct":"GF-3001","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Genflow Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical","sponsorNew":"Genflow Biosciences \/ EXO Biologics","highestDevelopmentStatusID":"4","companyTruncated":"Genflow Biosciences \/ EXO Biologics"},{"orgOrder":0,"company":"ResVita Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"RVB-003","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"ResVita Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Topical","sponsorNew":"ResVita Bio \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"ResVita Bio \/ Inapplicable"},{"orgOrder":0,"company":"NeuroNOS","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"BA-102","moa":"nNOS","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"NeuroNOS","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"NeuroNOS \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"NeuroNOS \/ Inapplicable"},{"orgOrder":0,"company":"Apertura Gene Therapy","sponsor":"RSRT","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Collaboration","leadProduct":"Undisclosed","moa":"TfR1","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Apertura Gene Therapy","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous","sponsorNew":"Apertura Gene Therapy \/ RSRT","highestDevelopmentStatusID":"4","companyTruncated":"Apertura Gene Therapy \/ RSRT"},{"orgOrder":0,"company":"Kamari Pharma","sponsor":"BRM Group","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"ISRAEL","productType":"Other Small Molecule","year":"2025","type":"Series A Financing","leadProduct":"KM023","moa":"TRPV3","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Kamari Pharma","amount2":0.02,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0.02,"dosageForm":"Oral","sponsorNew":"Kamari Pharma \/ BRM Group","highestDevelopmentStatusID":"4","companyTruncated":"Kamari Pharma \/ BRM Group"},{"orgOrder":0,"company":"Actio Biosciences","sponsor":"Regeneron Ventures","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Series B Financing","leadProduct":"ABS-1230","moa":"KCNT1","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Actio Biosciences","amount2":0.070000000000000007,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0.070000000000000007,"dosageForm":"Oral","sponsorNew":"Actio Biosciences \/ Regeneron Ventures","highestDevelopmentStatusID":"4","companyTruncated":"Actio Biosciences \/ Regeneron Ventures"}]
Find Drugs for Rare Diseases and Disorders in Preclinical Development
Details :
Novasep will be in charge of developing and manufacturing the two AAV vectors designed for the Sensorion OTOF-GT project and will supply Drug Product batches to support preclinical and clinical studies.
Details :
The financing aims to advance the Actio’s pipeline, which is led by ABS-1230, a KCNT1 inhibitor for the treatment of KCNT1-related epilepsy and other genetic epilepsies.
Details :
Proceeds will be used to advance Kamari's lead program KM023, a first-in-class oral TRPV3 inhibitor, for three rare genetic skin diseases, Olmsted syndrome, severe keratoderma and ichthyosis.
Details :
The collaboration aims to advance the innovative genetic medicine, which is being evaluated in preclinical clinical trial studies for the treatment of Rett Syndrome.
Details :
BA-102 is an investigational agent that aims to target and regulate nitric oxide (NO) levels in the brain and being developed for the treatment of PMS, a syndrome associated with ASD.
Details :
RVB-003 is based on ResVita Bio's continuous protein therapy platform, which utilizes genetically engineered bacteria applied topically, being developed for Netherton Syndrome.
Details :
Agreement will focus on the development of novel topical rapamycin (sirolimus) formulations as potential treatments for a number of rare and orphan diseases.
Details :
Under the terms of the agreement, Beacon will be responsible for the development and commercialization of AAV204 capsid for potential use for the treatment of retinal diseases.
Details :
The funding will be used to support the company’s lead program, AXV-101, an adeno-associated virus (AAV9)-based gene therapy targeting retinal dystrophy associated with Bardet-Biedl Syndrome.
Details :
MDL-101 is an experimental, epigenetic editing therapy, which is under investigation in the early-stage studies for the treatment of LAMA2-congenital muscular dystrophy.
Reset All
Axplora is your partner of choice for complex APIs.
