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GAIN-CTNNB1: Gene Therapy for CTNNB1 Syndrome
NCT07270549Phase 2Recruiting
- Sponsor
- CTNNB1 Foundation
- Start
- Nov 2025
- Primary completion
- Dec 2032
- Modality
- Gene Editing, Gene Silencing, Gene Addition/Replacement
- Countries
- 1
01
Overview
A Phase 2 trial of Urbagen in CTNNB1-related neurodevelopmental disorder (CTNNB1-NDD), sponsored by CTNNB1 Foundation. Started Nov 2025; status: recruiting.
02
Drugs
Urbagen
03
Conditions
CTNNB1-related neurodevelopmental disorder (CTNNB1-NDD)
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