New OTL-203 proof-of-concept data demonstrate extensive metabolic correction in the skeletal system of patients with MPS-IH including normal growth rates, improvement in joint function and progressive acquisition of motor skills
Updated OTL-201 data from ongoing proof-of-concept study in MPS-IIIA patients show additional favorable neurocognitive outcomes compared to disease natural history with median follow-up of 2.5 years
First preclinical data for OTL-204 highlight ability of HSC gene therapy to restore microglial function, modulate neuroinflammation, and normalize predictive biomarkers in the progranulin form of frontotemporal dementia (GRN-FTD)
Company to host conference call and webcast Tuesday, May 23 at 8:00 a.m. EDT
BOSTON and LONDON, May 19, 2023 (GLOBE NEWSWIRE) — Orchard Therapeutics (NASDAQ:ORTX), a global gene therapy leader, today announced multiple clinical and preclinical updates from its portfolio of investigational hematopoietic stem cell (HSC) gene therapies in neurometabolic and neurodegenerative disorders. The data are being featured in several oral presentations at the ongoing 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles.
“Positive data from multiple clinical and preclinical studies reinforce the ability of our HSC gene therapy platform to drive the migration of gene-corrected cells into the central nervous system and other tissues and deliver therapeutic enzymes and proteins locally to potentially correct multiple severe genetic diseases,” said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard Therapeutics. “The OTL-203 and OTL-201 programs for MPS disorders are intended to address a significant medical need given the limitations or lack of effective therapies for these conditions, and present development and commercial synergies with our neurometabolic pipeline by building on our experience with Libmeldy for metachromatic leukodystrophy. The ability of HSC gene therapy to impact the CNS is further demonstrated by our research program in a genetic subtype of FTD and highlights the potential of our platform to address larger indications.”
