Orchard Therapeutics Plans To Seek Strategic Options For Immunodeficiency Programs, Cuts Workforce

Orchard Therapeutics plc ORTX announced its intention to focus its hematopoietic stem cell (HSC) gene therapy platform exclusively on neurometabolic diseases and early research programs. 

• Orchard intends to discontinue its investment and seek alternatives for rare primary immune deficiency programs, including:
• OTL-103 in Wiskott-Aldrich syndrome (WAS).
• OTL-102 for X-linked chronic granulomatous disease (X-CGD).
• Strimvelis, a gammaretroviral vector-based gene therapy approved in Europe for adenosine deaminase severe combined immunodeficiency (ADA-SCID).
• Orchard will continue its investment in Libmeldy (atidarsagene autotemcel) / OTL-200 for metachromatic leukodystrophy (MLD). 
• The Company also will continue to advance the clinical development of OTL-203 for mucopolysaccharidosis type I Hurler's syndrome (MPS-IH) and OTL-201 for mucopolysaccharidosis type IIIA (MPS-IIIA). 
• Regarding the regulatory status of the OTL-103 program, Orchard recently received written feedback from the FDA. It believes the path to a potential BLA filing (late 2022 or early 2023) may require additional time and further investment.
• The Company will reduce its current workforce by approximately 30%, which will result in a restructuring charge in 2022. 
• Cash, cash equivalents, and investments of approximately $220.1 million, with $33.0 million of debt outstanding, are expected to fund Orchard Therapeutics expenditure requirements into 2024.
• Price Action: ORTX shares are trading at $0.78 during the market session on the last check Wednesday.