FDA Clears NIH-Supported Gene Therapy MMA-101 for First Human Trial in Rare Metabolic Disorder
Summary
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 clearance allows a first-in-human Phase I/II clinical trial to proceed at the NIH Clinical Center, offering a potential treatment path for patients with this serious condition.
Source
Twitter / XFDA Clears NIH-Supported Gene Therapy MMA-101 for First Human Trial in Rare Metabolic Disorderncats.nih.govKey quotes
· 4 pulledOn May 8, 2026, the U.S. Food and Drug Administration (FDA) cleared an Investigational New Drug (IND) application for MMA-101
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 clearance allows a first-in-human Phase I/II clinical trial to move forward at the NIH Clinical Center (CC)
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