Exbio Βιολογια και Κοινωνια
FDA Clears IND for Gene Therapy Candidate to Treat Rare Metabolic Disorder
First-in-human clinical trial aims to address unmet need for patients with methylmalonic acidemia On May 8, 2026, the U.S. Food and Drug Administration (FDA) cleared an Investigational New Drug (IND) application for MMA-101, an adeno-associated virus (AAV)-based gene therapy candidate for patients with methylmalonyl-CoA mutase (MMUT) methylmalonic acidemia (MMA), a rare pediatric metabolic disease with no approved treatments. The IND ...
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