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AAVrh.10hFXN Gene Therapy for Cardiomyopathy of Friedreich's Ataxia
NCT05302271Phase 1Recruiting
- Sponsor
- Weill Cornell Medicine
- Start
- Feb 2022
- Primary completion
- Dec 2028
- Modality
- Gene Editing, Gene Silencing, AAV Vectors
- Countries
- 1
01
Overview
Phase 1, open-label trial of LX2006 in Friedreich ataxia (FA) and cardiomyopathy (4 cohorts), sponsored by Weill Cornell Medicine. Primary endpoint: Safety of AAVrh.10hFXN at 5 years. Started Feb 2022; status: recruiting.
02
Drugs
LX2006
03
Conditions
Friedreich ataxia (FA)cardiomyopathy
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