Orchard Therapeutics announces first patient randomized in trial of OTL-203

Orchard Therapeutics (ORTX), recently acquired by Kyowa Kirin (KYKOF) with the goal of accelerating the delivery of new gene therapies to patients around the globe, announced the first patient has been randomized at the M Health Fairview Masonic Children’s Hospital in a registrational trial evaluating the efficacy and safety of OTL-203, an investigational hematopoietic stem cell gene therapy, in patients with the Hurler subtype of mucopolysaccharidosis type I, MPS-IH. The trial, referred to as HURCULES, compares treatment with OTL-203 to standard of care with allogeneic hematopoietic stem cell transplant, and is expected to enroll 40 MPS-IH patients at sites across the U.S. and Europe. “This is a substantial milestone in the development of OTL-203 as we work toward bringing an important new treatment option to children with MPS-IH and their families,” said Leslie Meltzer, Ph.D., chief medical officer of Orchard Therapeutics. “Previously reported results from an earlier proof-of-concept study in patients showed robust metabolic correction, continued cognitive, motor, and physical development, as well as early improvements in skeletal, ocular and auditory health demonstrating that one-time treatment with OTL-203 has the potential to positively impact a broad range of clinical manifestations not fully addressed by the current standard of care.”