New Path for a Gene Therapy Trial at NIH for a Rare Metabolic Disease

November 19, 2024 - News Type NCATS News

• Bespoke Gene Therapy Consortium (BGTC)
• Our Impact on Clinical Trials
• Our Impact on Drug Discovery and Development
• Our Impact on Rare Diseases
• Platform Vector Gene Therapy (PaVe-GT)

Scientists at NCATS and other NIH institutes and centers are teaming up to advance a gene therapy treatment for MMA, a rare and potentially life-threatening genetic disorder.