GENE202

GENE202 is an investigational, off-the-shelf gene therapy developed by Genespire for the treatment of methylmalonic acidemia (MMA), a rare inherited metabolic disorder. The therapy uses Genespire’s proprietary Immune Shielded Lentiviral Vector (ISLV) platform to deliver a functional copy of the human methylmalonyl-CoA mutase (MUT) gene directly to liver cells. MMA is caused by deficiency or absence of the MUT enzyme, leading to toxic metabolite accumulation and severe complications in children. By enabling lifelong production of the enzyme in hepatocytes after a single intravenous administration, GENE202 aims to address the underlying cause of MMA and provide a one-time curative option for pediatric patients. Preclinical studies have demonstrated dose-dependent efficacy and support progression into clinical trials[4][5][7][8].