Orchard Therapeutics Announces 2021 Corporate Priorities Supporting the Build-out of its Commercial Business in Hematopoietic Stem Cell (HSC) Gene Therapy and Expansion of its Clinical Applications

Preparations on Track for First Half 2021 Commercial Launch of Libmeldy (OTL-200), the First Approved Product for Metachromatic Leukodystrophy (MLD) in the EU

Filing Strategy for OTL-200 Biologics License Application (BLA) in MLD in the U.S. to be Communicated by Mid-2021 Following Additional Regulatory Interactions

Marketing Authorization Application (MAA) Filing for OTL-103 in Wiskott-Aldrich Syndrome (WAS) on Track for Year End 2021 in the EU; Followed by BLA Filing in 2022 in the U.S.

New Clinical Data for OTL-203 (for MPS-I) and OTL-201 (for MPS-IIIA) Accepted for Oral Presentation at February 2021 WORLD Symposium; Preclinical Data from Research Programs in Larger Indications Expected in 2021

$192M in Cash and Investments to Support Strategic Execution into the First Half of 2022

BOSTON and LONDON, Jan. 11, 2021 (GLOBE NEWSWIRE) --  Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today outlined the company’s 2021 strategic priorities in advance of its attendance at the virtual 39^th Annual J.P. Morgan Healthcare Conference. These priorities support the company’s plan of building a successful commercial business in HSC gene therapy and advancing its portfolio of investigational medicines for high-value, high-need indications.

“In a year that challenged how we live and work, I’m extremely proud of Orchard’s achievements in 2020,” said Bobby Gaspar, M.D., Ph.D., chief executive officer, Orchard Therapeutics. “Our accomplishments were a direct result of the drive and innovation that fuels our commitment to bring our potentially life-saving HSC therapies to patients, including Libmeldy, which is the first product approved for the treatment of eligible patients with early-onset MLD in the EU. With the HSC approach to gene therapy as our scientific foundation, we are focused on the capabilities that can deliver our therapies on a global commercial scale and support our ability to also treat larger indications over time. It has been a privilege to be a pioneer in changing the way medicine is practiced in these conditions, and we look forward to another year of continued execution and scientific progress.”