Genespire and SR-TIGET Show Durable Preclinical Efficacy of Liver-Directed Gene Therapy for Methylmalonic Acidemia
The study shows that ISLV-directed delivery of the MMUT gene produced durable therapeutic benefits in mouse models. Gene transfer efficiency exceeded 80% of the liver. Genespire is progressing its lead asset for MMA, GENE202, towards the clinic. MILAN, July 8, 2026 /PRNewswire/ --...
The study shows that ISLV-directed delivery of the MMUT gene produced durable therapeutic benefits in mouse models. Gene transfer efficiency exceeded 80% of the liver. Genespire is progressing its lead asset for MMA, GENE202, towards the clinic. MILAN, July 8, 2026 /PRNewswire/ --...
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