CANbridge Pharmaceuticals to Present Fabry Disease Gene Therapy Abstract at ESGCT 30th Annual Congress.
ENPNewswire-October 26, 2023--CANbridge Pharmaceuticals to Present Fabry Disease Gene Therapy Abstract at ESGCT 30th Annual Congress
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Release date- 25102023 - Beijing - 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, -galactosidase A (-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 -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 -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...
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