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» FDA grants orphan drug designation to liver-targeted gene therapy, BGT-OTCD
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Endocrine/Metabolic
FDA grants orphan drug designation to liver-targeted gene therapy, BGT-OTCD
Aug. 14, 2023
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Bloomsbury Genetic Therapies Ltd. announced that the FDA granted orphan drug designation for its investigational liver-targeted gene therapy, BGT-OTCD, for the treatment of ornithine transcarbamylase deficiency (OTCD).
BioWorld Science
Regulatory
Endocrine/metabolic
Gene therapy
FDA
Orphan drug