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ATA-100 gene therapy in LGMDR9
NCT05224505Phase 1Active, not recruiting
- Sponsor
- Atamyo Therapeutics
- Start
- Sep 2022
- Primary completion
- Feb 2025
- Modality
- Gene Editing, Gene Silencing, Gene Addition/Replacement
- Countries
- 3
01
Overview
Phase 1, open-label trial of AAV gene therapy for LGMD 2I/R9 in Limb-girdle muscular dystrophy type R9 (LGMDR9) (2 cohorts), sponsored by Atamyo Therapeutics. Primary endpoint: Safety and Tolerability at 12 months. Started Sep 2022; status: active, not recruiting.
02
Drugs
AAV gene therapy for LGMD 2I/R9
03
Conditions
Limb-girdle muscular dystrophy type R9 (LGMDR9)
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