MMA-101

**MMA-101** is a liver-targeted, recombinant adeno-associated virus serotype 8 (AAV8) gene therapy designed to deliver a functional MMUT gene for the treatment of methylmalonic acidemia (MMA) due to MMUT gene deficiency. The therapy aims to achieve constitutive hepatic expression of the wild-type MMUT enzyme, correcting the underlying metabolic defect in patients and providing long-term benefits. It is administered intravenously and often tested in combination with ImmTOR, a nanoparticle-based immunomodulator containing sirolimus, intended to induce immune tolerance, decrease immunogenicity of the viral vector, and allow for potential redosing. MMA-101 has received orphan drug and rare pediatric disease designations from the FDA. The primary indication is for the isolated MMUT subtype of methylmalonic acidemia in pediatric patients, providing an alternative to liver transplantation.

Other names

MMA-101MMA101MMA 101