The dual vector AAV-CHD3-R1025W base editor is an investigational in vivo DNA base-editing therapy being evaluated for Snijders Blok-Campeau syndrome, a neurodevelopmental disorder caused by the heterozygous CHD3 p.Arg1025Trp (R1025W) mutation. The therapy uses a dual-AAV delivery system to introduce base-editing components into the central nervous system via a single intrathecal injection, with the goal of correcting the pathogenic nucleotide change without inducing double-strand DNA breaks.
Disease: Snijders Blok-Campeau syndrome, (NCT06860672)
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Candidate summary:
Disease/Disease category:
- Neurodevelopmental disorders
Official title:
Safety, Tolerability and Preliminary Efficacy Study of a Single Intrathecal Injection of the Dual Vector AAV-CHD3-R1025W Base Editor for the Treatment of Developmental Disorders Caused by the R1025W Mutation in the CHD3 Gene
Phases:
- Phase 1
Status:
Active recruiting
Country:
- China
