FDA Grants Orphan Drug Status to M6P Gene Therapy

FDA Grants Orphan Drug Status to M6P Gene Therapy

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FDA Grants Orphan Drug Status to M6P Gene Therapy

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to two gene therapies that M6P Therapeutics is developing — one intended for Gaucher disease, the other aiming to treat the inherited metabolic disorder mucolipidosis. The regulatory agency also awarded six rare pediatric disease designations to various investigational therapies that M6P Therapeutics is developing to treat lysosomal storage disorders, which include Gaucher and mucolipidosis. “We are grateful to the FDA for these multiple…

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