We’re thrilled to share groundbreaking news: the Slovenian government has awarded €1 million in public funding to the CTNNB1 Foundation to support the development of URBAGEN — the world’s first gene therapy for CTNNB1 Syndrome.
This is the first time a government has publicly backed a nonprofit-led gene therapy at such an early stage. The support will fund the final steps toward clinical trials, which are expected to begin in late summer 2025.
URBAGEN, developed for children with CTNNB1 Syndrome, offers a one-time gene therapy using an AAV9 vector to target the root cause of this devastating condition — bringing hope to families worldwide.
“This funding marks a turning point,” said Špela Miroševič, CEO and founder of the Foundation. “It’s a signal that governments can—and should—step up to support equitable access to life-changing treatments.”
This is more than a scientific milestone — it’s a model for how rare disease therapies can be developed equitably and collaboratively, ensuring access for all.
