Drugs / Urbagen
last change Nov 2025 re-read 2 minutes ago

Urbagen

Gene therapy (AAV / viral vector) targets CTNNB1 via restoration

Developed for
CTNNB1-related neurodevelopmental disorder and/or vitreoretinopathy
Investigated by
CTNNB1 Foundation · University Medical Centre Ljubljana

Trials 1 · a red edge is where a trial was stopped

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PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/2 NCT07270549 Nov 2025 → Dec 2032 expected CTNNB1-related neurodevelopmental disorder and/or vitreoretinopathy CTNNB1 Foundation Recruiting No outcome recorded

Evidence & citations 4 cited values

Every value below carries the sentence it was read from. 1 source stands behind the page.

FieldValueCited text
Known as Urbagen ClinicalTrials.gov intervention name — accepted as the source's own label NCT07270549
Action Restore “designed to restore functional CTNNB1 expression” NCT07270549
Modality Gene therapy (AAV / viral vector) “AAV9 mediated gene replacement therapy (Urbagen)” NCT07270549
Target CTNNB1 “Urbagen is an investigational gene addition therapy designed to restore functional CTNNB1 expression” NCT07270549