News and Trends 3 May 2018
FDA Backs Gene Therapy for Fatal Neurodegenerative Childhood Disease
Orchard Therapeutics has received a fourth Rare Pediatric Disease Designation from the FDA for its gene therapy candidate for metachromatic leukodystrophy. Orchard Therapeutics, based in London, has received a Rare Pediatric Disease Designation form the FDA for its gene therapy OTL-200. The treatment is for patients with metachromatic leukodystrophy (MLD), a fatal genetic disease that causes […]
