FDA grants orphan drug designation to liver-targeted gene therapy, BGT-OTCD

Aug. 14, 2023

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

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BioWorld briefs for April 14, 2025.

Today's news in brief

BioWorld MedTech

BioWorld MedTech briefs for April 14, 2025.

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