GBA1 Canada initiative (G-can), a pre-competitive, open science platform for GBA1 research

GBA1 Canada (G-Can) is a pre-competitive, open-science platform serving as a central hub for basic, translational, and clinical research on GBA1, which encodes the lysosomal enzyme acid beta-glucocerebrosidase (GCase), a key component of the glycosphingolipid metabolism pathway. G-Can unites basic scientists, clinicians, and industry partners in Canada and internationally to minimize duplication of effort and maximize research impact. G-Can has established a comprehensive and growing suite of biological, clinical, and analytical resources for the GBA1 research community. Through the C-BIG Repository and partner infrastructures, these resources are made broadly accessible to academic and industry investigators under non-exclusive agreements, in accordance with G-Can's open-science mandate. G-Can's primary objectives are to deepen understanding of GCase biology and its genetic modifiers, validate disease-relevant biomarkers, and establish trial-ready cohorts exceeding 5000 genotyped participants. Therapeutic programs under development include gene therapy, RNA-based approaches, and combination strategies, with a Phase 0/1 clinical trial targeted within five years. By integrating open-science infrastructure with coordinated therapeutic development, G-Can aims to accelerate the translation of discovery into clinical application for patients with GBA1-associated neurodegeneration.