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December 5, 2018

Donald B. Kohn, MD, Orchard Therapeutics Scientific Advisory Board Member, Presents Clinical Proof-of-Concept Data for OTL-102 Gene Therapy for the Treatment of Patients with X-CGD at the Presidential Symposium at the 2018 ASH Annual Meeting

First demonstration of potential gene therapy treatment in this population leading to sustained levels of functioning neutrophils after 12 months

BOSTON and LONDON, Dec. 05, 2018 (GLOBE NEWSWIRE) — Orchard Therapeutics (NASDAQ:ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced that Scientific Advisory Board Member, Donald B. Kohn, MD, presented clinical proof-of-concept data from an ongoing academic clinical trial evaluating OTL-102 for the treatment of X-Linked Chronic Granulomatous Disease (X-CGD) during the Presidential Symposium at the 2018 American Society of Hematology (ASH) Annual Meeting & Exposition on December 4, 2018. X-CGD is a life-threatening inherited immunodeficiency disorder which is caused by a genetic mutation that results in the inability of neutrophils to effectively kill bacterial and fungal pathogens. This immune deficiency leads to repeated chronic and severe infections often requiring hospitalization and resulting in chronic sequelae leading to early mortality. Preclinical and clinical development of OTL-102 had originally been initiated by Genethon (Evry, France) before licensing to Orchard.

“These clinical proof-of-concept data demonstrate, for the first time, 12 months or more of restoration of immunity in X-CGD patients treated with ex-vivo autologous gene therapy,” said Adrian Thrasher, professor of pediatric immunology and Wellcome Trust Principal Research Fellow at UCL Great Ormond Street Institute of Child Health in London. “In six of the seven evaluated patients, we are encouraged to see sustained levels of functioning neutrophils at greater than 10%, which prior publicly available data suggests is the level sufficient to see potential clinical benefit. This important milestone, together with improved clinical outcomes and an emerging safety profile with no signs of genotoxicity, suggest OTL-102 may provide a transformative treatment for X-CGD patients.”

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