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Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"YolTech Therapeutics \/ Northwestern University","highestDevelopmentStatusID":"6","companyTruncated":"YolTech Therapeutics \/ Northwestern University"},{"orgOrder":0,"company":"PRG S&Tech","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"SOUTH KOREA","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Progerinin","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"PRG S&Tech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"PRG S&Tech \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"PRG S&Tech \/ Inapplicable"},{"orgOrder":0,"company":"Longboard 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Hoffmann-La Roche","sponsor":"Oak Hill Bio","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"SWITZERLAND","productType":"Oligonucleotide","year":"2025","type":"Licensing Agreement","leadProduct":"Rugonersen","moa":"UBE3A-ATS","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"F. Hoffmann-La Roche","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"F. Hoffmann-La Roche \/ Oak Hill Bio","highestDevelopmentStatusID":"6","companyTruncated":"F. Hoffmann-La Roche \/ Oak Hill Bio"}]
Find Drugs for Rare Diseases and Disorders in Phase I Clinical Development in UNITED STATES
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