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Orchard Therapeutics Announces New England Journal of Medicine Publication of Interim Proof-of-concept Study Results of OTL-203 for Hurler Syndrome

100 percent overall survival with median follow-up of two years post-treatment with HSC gene therapy

Preliminary findings show promising metabolic and early clinical outcomes

Results warrant further evaluation in global registrational study expected to be initiated in 2022 following recent meeting to discuss trial design with U.S. and European regulators

Excerpt from the Press Release:

BOSTON and LONDON, Nov. 18, 2021 (GLOBE NEWSWIRE) — Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced data published in the New England Journal of Medicine (NEJM) evaluating the safety and efficacy of OTL-203 for the treatment of the Hurler subtype of Mucopolysaccharidosis type I (MPS-IH). OTL-203 is an investigational autologous hematopoietic stem cell (HSC) gene therapy comprising an individual’s own CD34+ HSCs transduced ex vivo with a lentiviral vector encoding the alpha-L-iduronidase (IDUA) gene.

Mucopolysaccharidosis type I (MPS-I) is a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme. It is estimated to occur globally in approximately 1 in 100,000 live births. Approximately 60 percent of children born with MPS-I have the most severe subtype, MPS-IH, also called Hurler syndrome, and rarely live past the age of 10 when untreated.

“MPS-IH places an enormous burden on affected children, their families and society,” said Maria Ester Bernardo, Ph.D. head of the pediatric bone marrow transplantation unit at San Raffaele Hospital in Milan and a senior author of the NEJM manuscript. “Current treatment options are associated with significant limitations and do not adequately address some of the more severe manifestations of disease. Based on these preliminary first-in-human data, administration of a one-time HSC gene therapy designed to overexpress IDUA represents a potential step forward in the treatment landscape.”

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