Genespire and SR-TIGET Show Durable Preclinical Efficacy of Liver-Directed Gene Therapy for Methylmalonic Acidemia

A recent study revealed that delivering the MMUT gene through ISLV resulted in long-lasting therapeutic effects in mice. The gene transfer was successful in more than 80% of the liver cells. Genespire is moving forward with its primary treatment for MMA, GENE202, aiming to bring it to clinical trials. The development took place in MILAN on July 8, 2026.