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CAN-202
- Development stage
- Preclinical
- Lead developer
- CANbridge Pharmaceuticals
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies
- Administration
- Intravenous
01
Overview
CAN-202 is an investigational gene therapy being developed for the treatment of Fabry disease, a rare lysosomal storage disorder. It utilizes the sL65 adeno-associated virus (AAV) capsid, which is part of the sAAVy platform licensed from LogicBio Therapeutics. The therapy is designed to deliver a functional copy of the GLA gene to the liver (hepatocytes), which then serves as a biofactory to produce and secrete the alpha-galactosidase A (α-Gal A) enzyme into the bloodstream. This systemic distribution of the enzyme is intended to reduce the accumulation of globotriaosylceramide (Gb3) in various tissues, potentially providing a long-term therapeutic effect compared to traditional enzyme replacement therapies.
02
Targets
GLA (Alpha-galactosidase A)
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