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EXG001-307 in Type I Spinal Muscular Atrophy
NCT06576388Phase 1Completed
- Sponsor
- Hangzhou Jiayin Biotech
- Start
- Jan 2022
- Primary completion
- Sep 2023
- Modality
- Gene Editing, Gene Silencing, AAV Vectors
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of EXG001-307 in spinal muscular atrophy type 1 (SMA1), sponsored by Hangzhou Jiayin Biotech. Primary endpoint: Safety and tolerability of EXG001-307 at 18 months. Started Jan 2022; status: completed.
02
Drugs
EXG001-307
03
Conditions
spinal muscular atrophy type 1 (SMA1)
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