CTNNB1 Foundation

CTNNB1 Foundation is a non-governmental organisation based in Ljubljana, Slovenia working on medical research, health.

The CTNNB1 Foundation is a research-driven non-profit based in Ljubljana, Slovenia, focused on developing gene-related treatments for CTNNB1 syndrome, a severe neurodevelopmental disorder affecting walking, speech and independent living. Its main programme is URBAGEN, a gene replacement therapy that has received EMA Orphan Drug Designation and has a clinical trial application submitted to the EMA. The foundation works with research institutes, laboratories and manufacturers, funds its work through donations, campaigns and government support, and runs awareness activities such as international CTNNB1 syndrome conferences and resources for medical teams and families.