CANbridge Pharmaceuticals Fabry Disease Gene Therapy Abstract Accepted by ESGCT 30th Annual Congress Presentation


Beijing, China; Cambridge, Mass., September 28, 2023 — CANbridge Pharmaceuticals Inc. (“CANbridge”, stock code 1228.HK), a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies for rare diseases and oncology indications, proudly announces the acceptance of the following abstract for presentation at the European Society of Gene & Cell Therapy (ESGCT) 30th Annual Congress, scheduled for October 24-27, 2023 in Brussels, Belgium.


Title: Preclinical Evaluation of CAN201 Containing a Human GLA Transgene Under the Control of a Liver-Specific Promoter for Fabry Disease

Poster: # P020 at ESGCT during October 24-27, 2023


CAN201 is a gene therapy program in the pre-clinical stage, utilizing the unique liver-targeting properties of adeno-associated virus (AAV) sL65 capsid to treat Fabry disease, a rare inherited lysosomal storage disorder (LSD) characterized by glycosphingolipid (fat) metabolic issues due to the absent or markedly deficient lysosomal α-galactosidase A (α-Gal A) enzyme activity. CANbridge holds an exclusive global license from LogicBio Therapeutics, Inc. (“LogicBio”, a wholly owned subsidiary of Alexion, AstraZeneca Rare Disease) to develop, manufacture and commercialize gene therapy candidates for the treatment of Fabry and Pompe diseases, based on LogicBio’s AAV sL65 technology.



About CANbridge Pharmaceuticals, Inc.

CANbridge Pharmaceuticals Inc. (HKEX:1228) is a 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 4 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 Centeris 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  at the American Society for Gene and Cell Therapy (ASGCT) in 2022 and 2023, the European Society for Gene and Cell Therapy (ESGCT) in 2022 and the World Muscle Congress in 2022. 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.


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CANbridge Pharmaceuticals Inc.



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