Genespire Reports Durable Preclinical Success in MMA Gene Therapy

The research, conducted by Genespire in partnership with the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), demonstrates that delivering the MMUT gene can overcome the metabolic deficiencies characteristic of MMA. In treated mice, researchers observed gene transfer efficiency exceeding 80% of liver cells. The study indicates that genetically corrected cells can progressively replace diseased ones, potentially allowing for therapeutic efficacy to improve over time even with lower initial doses.

MMA remains a severe inherited disorder where the absence of methylmalonyl-CoA mutase causes toxic metabolite accumulation, leading to multi-organ damage and neurological impairment. Current treatment options are limited, leaving patients with high morbidity and reduced life expectancy. Genespire’s platform utilizes immune-shielded lentiviral vectors (ISLVs) designed for intravenous administration, aiming to establish life-long enzyme production directly within the liver.

Dr. Alessio Cantore, senior author and SR-TIGET group leader, noted that this data package provides the necessary foundation to initiate clinical testing. Lucia Faccio, CEO of Genespire, confirmed the company is now focused on advancing GENE202 toward human trials, viewing the single-administration approach as a potential long-term solution for patients suffering from metabolic liver-based diseases.