We are pleased to share that the CTNNB1 Foundation has received approval to initiate a Phase I/II clinical trial evaluating URBAGEN™, the first investigational gene-replacement therapy developed for CTNNB1 syndrome. This represents a significant step forward for the CTNNB1 community, as the first therapeutic program to reach human clinical testing for this rare neurodevelopmental condition.

The CTNNB1 Foundation is a non-profit organization established with the mission to accelerate the development of treatments for CTNNB1 syndrome and improve the lives of those affected. The project was inspired by the personal promise of Špela Miroševič, founder and president of the Foundation, whose dedication began with her own child’s CTNNB1 syndrome diagnosis.

URBAGEN™ will be assessed for safety, tolerability, and early signs of efficacy, bringing the field one step closer to a potential treatment that targets the underlying genetic cause of the disorder.

3D-PharmXchange is proud to have supported the CTNNB1 Foundation throughout this journey, including contributions to nonclinical development and regulatory preparation, helping advance the program toward clinical evaluation. Our experience supporting gene therapy programs has helped ensure the seamless transition of URBAGEN™ from preclinical development toward its first-in-human study.

We celebrate this milestone with CTNNB1 Foundation and are eager to see continued progress as the program moves forward.

Read the full announcement from CTNNB1 Foundation at this link.