Orchard Therapeutics Announces Last Patient Treated in Registrational Trial of OTL-203 for MPS-I Hurler Syndrome

Completion of enrollment achieved nearly one year ahead of schedule Primary analysis anticipated two years post-treatment TOKYO and LONDON and BOSTON, July 07, 2025 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a Kyowa Kirin company, today announced the last patient has been treated in a registrational trial evaluating the efficacy and safety of OTL-203, an investigational hematopoietic stem cell (HSC) gene therapy, in patients with the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). The trial (NCT0614 ...