From June 16–20, 2025, our CTNNB1 community came together in Bilbao for an unforgettable week of connection, progress, and hope. The event combined clinical examinations of children (June 16–19) with our 3rd International CTNNB1 Syndrome Conference (June 19–20). We hosted a gathering of families, clinicians, researchers, and industry partners from around the world.

This year marked a major milestone: we have successfully completed preclinical studies and started the manufacturing of our gene replacement therapy. The conference program focused on gene therapy development, safety data, clinical trial readiness, and regulatory pathways, alongside updates on manufacturing, new therapeutic approaches, and the latest research on CTNNB1 syndrome.

We shared scientific advances, discussed the upcoming GAIN clinical trial, and explored complementary strategies such as CRISPR-based editing and small molecule development. Families also had the chance to share their experiences, strengthening the bonds that make our community so resilient.