ORTX - Orchard Therapeutics Announces Multiple Presentations at the 20th Annual WORLDSymposium | Benzinga
BOSTON and LONDON, Feb. 02, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a global gene therapy leader recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced 10 presentations from across its neurometabolic portfolio will be featured at the 20th Annual WORLDSymposium™ taking place February 4-9, 2024, in San Diego, California.
Featured presentations include updated neurocognitive and biochemical results from an ongoing proof-of-concept study of investigational OTL-201 in mucopolysaccharidosis type IIIA (MPS-IIIA), also known as Sanfilippo syndrome type A, with median follow-up now extending to 2.75 years (ranging from 24 to 39 months). Additional data highlights include several accepted abstracts detailing newborn screening efforts to support the timely and accurate diagnosis of metachromatic leukodystrophy (MLD), as well as an encore presentation showcasing long-term results from an updated integrated analysis of 39 patients with MLD treated with investigational OTL-200, which comprises key components of the clinical data package of the Biologics License Application (BLA) currently under Priority Review by the U.S. Food and Drug Administration.
In addition to the data presentations outlined below, the company will also host a sponsored symposium on Monday, February 5, at 11:45 a.m., titled "Momentum, Leadership & Diagnosis: Changing the paradigm for MLD," featuring expert speakers from different regions sharing their knowledge and experience to establish new standards and advance newborn screening through interdisciplinary collaboration.
Oral presentation details are as follows (all times in PST; * denotes corresponding poster):
- Title: Clinical outcomes and sustained biochemical engraftment following ex-vivo autologous stem ...