New Path for a Gene Therapy Trial at NIH for a Rare Metabolic Disease
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.
Program Areas
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)