Orchard Therapeutics Receives EMA PRIME Designation for Investigational Lentiviral Gene Therapy
Orchard Therapeutics, a leading commercial-stage biotech company dedicated to transforming the lives of patients with rare diseases through innovative gene therapies, today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to OTL-300, an investigational autologous ex vivo lentiviral gene therapy for the treatment of transfusion-dependent beta-thalassemia (TDBT), the most severe…
Orchard Therapeutics Receives Rare Pediatric Disease Designation
Orchard Therapeutics announced that the FDA has granted a Rare Pediatric Disease Designation to the company’s gene therapy candidate OTL-200, for the treatment of patients with metachromatic leukodystrophy (MLD). MLD is a rare, fatal, neurodegenerative, inherited metabolic disease caused by mutations in the ARSA gene. In its late infantile and juvenile forms, which represents the majority…