Jerry R. Mendell Center for Gene Therapy
The mission of the Jerry R. Mendell Center for Gene Therapy is to investigate and employ the use of gene and cell based therapeutics for prevention and treatment of human diseases including: neuromuscular and neurodegenerative diseases, lysosomal storage disorders, ischemia and re-perfusion injury, neonatal hypertension, cancer and infectious diseases.
From Discovery to FDA Approval
The first approved gene therapy for Sanfilippo Syndrome Type A originated from research at the Jerry R. Mendell Center for Gene Therapy. Read More
The Abigail Wexner Research Institute is ranked among the top 10 for NIH funding among free-standing children's hospitals.
GMP Facility
The cGMP Clinical Manufacturing Facility, located at Nationwide Children's affiliate company Andelyn Biosciences, operates according to FDA cGMP guidelines to ensure the safety of manufactured biologic products.