Liver-directed gene therapy shows preclinical efficacy for severe inherited metabolic disorder

Article excerpt

Genespire, in collaboration with researchers at the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), announced the publication of preclinical data supporting the potential of its liver-directed immune-shielded lentiviral gene therapy approach to treat methylmalonic acidemia (MMA), a severe inherited metabolic disorder.