Categories: Wire Stories

CANbridge Pharmaceuticals Spinal Muscular Atrophy Gene Therapy Abstract Accepted for Presentation at the American Society for Gene and Cell Therapy annual Meeting

BEIJING & CAMBRIDGE, Mass.–(BUSINESS WIRE)–CANbridge Pharmaceuticals, Inc. (1228.HK), a China and U.S.-based global biopharmaceutical company committed to the research, development and commercialization of transformative therapies to treat rare diseases and oncology, announced that the American Society for Gene and Cell Therapy (ASGCT) has accepted the following abstract for presentation at the ASCGT Annual Meeting, May 16-20, in Los Angeles, CA.

Title:

Low-dose intracerebroventricular delivery of a second-generation AAV gene therapy for spinal muscular atrophy achieves efficient and toxicity-free motor function rescue in mice

About CANbridge Pharmaceuticals Inc.

CANbridge Pharmaceuticals Inc. (HKEX:1228) global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare disease and rare oncology. CANbridge has a differentiated drug portfolio, with three approved drugs and a pipeline of 10 assets, targeting prevalent rare disease and rare oncology indications that have unmet needs and significant market potential. These include Hunter syndrome and other lysosomal storage disorders, complement-mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases, as well as glioblastoma multiforme. The CANbridge Next-Generation Innovation and Process Development Facility is developing novel, potentially curative, gene therapies for rare genetic diseases, including Pompe disease, Fabry disease, spinal muscular atrophy (SMA) and other neuromuscular conditions, and collaborates with world-leading researchers and biotech companies. Animal data from the SMA gene therapy was presented in 2022 at the American Society for Gene and Cell Therapy (ASGCT), the European Society for Gene and Cell Therapy (ESGCT) and the World Muscle Congress. CANbridge global partners include: Apogenix, GC Pharma, Mirum, Wuxi Biologics, Privus, UMass Chan Medical School, the University of Washington School of Medicine and Scriptr Global.

For more on CANbridge Pharmaceuticals Inc., please go to: www.canbridgepharma.com.

Forward-Looking Statements

The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the data on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development.

Contacts

U.S. Investor Relations:
Chris Brinzey

ICR Westwicke

Chris.brinzey@westwicke.com

China Investor Relations
CANbridge Pharmaceuticals Inc.

ir@canbridgepharma.com

Media:
Deanne Eagle

Planet Communications

deanne@planetcommunications.nyc
917.837.5866

Alex

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