Orchard Therapeutics announces first patient dosed with OTL-103 gene therapy
Orchard Therapeutics announced that the first patient with Wiskott-Aldrich Syndrome, or WAS, has been dosed in a open label study designed to evaluate engraftment of the cryopreserved formulation of OTL-103, its ex vivo autologous hematopoietic stem cell, or HSC, gene therapy. While the registrational trial for OTL-103 for WAS used a fresh cell formulation, Orchard plans to commercialize this and other HSC gene therapies, if approved, using a cryopreserved formulation. In total, nearly 40 patients have been treated with a cryopreserved product across the company's portfolio. The WAS cryopreserved formulation trial will enroll up to six patients, with the number of patients with successful engraftment measured at six months as the primary endpoint. This patient data will be used to supplement the company's in vitro CMC comparability work between the fresh and cryopreserved formulation.