FDA Clears NIH-Supported Gene Therapy MMA-101 for First Human Trial in Rare Metabolic Disorder

The FDA has cleared an Investigational New Drug (IND) application for MMA-101, an AAV-based gene therapy candidate developed with NIH support for methylmalonyl-CoA mutase (MMUT) methylmalonic acidemia (MMA), a rare pediatric metabolic disorder with no approved treatments. This cl