drugset / Trial / NCT06860672
Clinical Trial of the Dual Vector Base Editor for the Treatment of the CHD3-R1025W Mutation
NaSingle-groupOpen-labelTreatment
Summary
To evaluate the 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
Timeline
- Start
- 2025-02-19
- Primary completion
- 2025-04
- Completion
- 2025-09
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | AAV-CHD3-R1025W Base Editor | Gene therapy (AAV / viral vector) | — | Intrathecal |