CANbridge Pharmaceuticals to Present Fabry Disease Gene Therapy Abstract at ESGCT 30th Annual Congress


Beijing, China; Cambridge, Mass., October 25, 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, today announced that data from the preclinical evaluation of CAN201, a potential gene therapy for Fabry disease, will be presented at the at the the European Society Gene & Cell Therapy (ESGCT) 30th Annual Congress, taking place from October 24-27 in Brussels, Belgium.


The study aimed to utilize the liver as a manufacturing site for the enzyme, α-galactosidase A (α-GAL), by delivering a human GLA gene via an adeno-associated virus (AAV) vector. In preclinical studies involving Fabry mice and a PXB mouse model containing a humanized liver, CAN201 showed a dose-dependent increase in α-GAL enzyme levels across various tissues with a corresponding reduction in disease-causing Gb3 lipid levels. The gene therapy was well tolerated with no significant adverse effects observed in Fabry mice. CANbridge holds an exclusive global license from LogicBio Therapeutics, Inc (“LogicBio”) to develop, manufacture and commercialize gene therapy candidates for the treatment of Fabry and Pompe diseases, based on LogicBio’s AAV sL65 technology.


“We are delighted that our therapeutic approach utilizing a liver-targeting AAV capsid to convert the liver into an efficient producer of α-GAL enzyme demonstrated in vivo proof-of-concept in the Fabry mouse model” said Gerry Cox, MD, PhD, Chief Development Strategist and Interim Chief Medical Officer of CANbridge Pharmaceuticals. “These findings bolster our confidence in the therapeutic potential of CAN201 as an alternative approach to address the limitations of current treatment options, such as enzyme replacement therapy and chaperone therapy, and offer hope for improved outcomes in individuals with Fabry disease.”



Presentation Details:

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

Session Date and Time: October 24-27, 2023

Authors: Jun Yang, Gregory Preston, Amy Bastille, Ryan Thompson, Peter Pechan, John Xiong, Megan Ericson, Anannya Banga, Aida Jameei, Nannan Jia, Shiliang Hu, Beth Thurberg, Yijie Ma, Leila Jalinous, Yunxiang Zhu, Jason West, Gerald Cox



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 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 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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