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EXG001-307 in Spinal Muscular Atrophy
NCT06888661Phase 1Enrolling by invitation
- Sponsor
- Hangzhou Jiayin Biotech
- Start
- Mar 2025
- Primary completion
- Mar 2026
- 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 (SMA), sponsored by Hangzhou Jiayin Biotech. Primary endpoint: Safety and tolerability of EXG001-307 at 52 weeks. Started Mar 2025; status: enrolling by invitation.
02
Drugs
EXG001-307
03
Conditions
spinal muscular atrophy (SMA)
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