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Lexeo Therapeutics Granted FDA Fast Track Designation and Orphan Drug Designation for LX2020, an AAV-Based Gene Therapy Candidate for PKP2 Arrhythmogenic Cardiomyopathy (ACM)

NEW YORK, Dec. 18, 2023 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (LXEO), a clinical stage genetic medicine company dedicated to pioneering treatments for genetically defined cardiovascular ...

Solid Biosciences Announces Receipt of European Commission Orphan Drug Designation for SGT-003 for the Treatment of Duchenne Muscular Dystrophy

Orphan drug designation from the European Commission underscores Solid’s commitment to advance SGT-003 through a global development effort for ...
BioWorld

Precision Biosciences’ PBGENE-DMD gains US orphan drug designation

The U.S. FDA has granted Precision Biosciences Inc.’s PBGENE-DMD orphan drug designation for the treatment of Duchenne muscular dystrophy (DMD). PBGENE-DMD uses two complementary Arcus nucleases ...