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Orchard Therapeutics Enrolls First Mucopolysaccharidosis Type I Patient in Gene Therapy Trial

NEW YORK – Orchard Therapeutics on Monday said it has randomized the first patient in a registrational Phase III trial of OTL-203, an experimental hematopoietic stem cell gene therapy it is developing as a treatment for those with the Hurler subtype of mucopolysaccharidosis type I (MPS-IH).

Orchard, which was acquired by Tokyo-based Kyowa Kirin in January, plans to enroll 40 patients with this rare and inherited neurometabolic disease in the US and Europe into the HURCULES study and compare the safety and efficacy of OTL-203 against the standard care, an allogeneic hematopoietic stem cell transplant. Treatment with OTL-203 involves inserting a functional copy of the IDUA gene, which produces the enzyme deficient in MPS-IH, into a patient's autologous hematopoietic stem cells.

The first patient was enrolled at M Health Fairview University of Minnesota Masonic Children's Hospital in Minneapolis. So far, the University of Minnesota is the only clinical site actively recruiting patients for the trial. However, Orchard is planning to activate six additional sites in the US and Europe.   Orchard has received fast-track and rare pediatric disease designations from the US Food and Drug Administration and priority medicines status from the European Medicines Agency for this therapy.