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Urbagen
- Development stage
- Phase 2
- Lead developer
- CTNNB1 Foundation
- Modality
- Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
- Administration
- Intracerebroventricular
01
Overview
Urbagen is an investigational gene replacement therapy developed by the CTNNB1 Foundation and The Gene Therapy Research Institute for the treatment of CTNNB1 neurodevelopmental syndrome. The therapy utilizes a non-replicating, single-stranded adeno-associated viral vector serotype 9 (AAV9) to deliver a functional copy of the human CTNNB1 gene directly to brain cells. Administered via a single intracerebroventricular (ICV) infusion, Urbagen is designed to restore levels of the beta-catenin protein, which is deficient or dysfunctional in patients with this genetic disorder. By addressing the underlying genetic cause, the treatment aims to improve developmental, motor, and cognitive functions in pediatric patients. It is currently being evaluated in the Phase I/II GAIN-CTNNB1 clinical trial.
- Brand names
- URBAGEN
- Other names
- Urbagen gene addition therapy
02
Targets
CTNNB1 (Beta-catenin)
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