CANbridge Pharmaceuticals Inc. announced that data from the preclinical evaluation of CAN201, a potential gene therapy for Fabry disease, will be presented at the at 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, a-galactosidase A (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 a-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.