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SAR446597
- Development stage
- Phase 2
- Lead developer
- Sanofi
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Ophthalmic, Intravitreal
01
Overview
SAR446597 is an investigational adeno-associated virus (AAV) gene therapy developed by Sanofi for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Administered as a one-time intravitreal injection, the therapy delivers a transgene encoding a modified version of human Complement Factor I (CFI), a key regulatory protease that downregulates the alternative complement pathway. By increasing local expression of CFI, SAR446597 aims to inhibit the overactive complement cascade that drives retinal pigment epithelium (RPE) cell death and lesion expansion in GA. The therapy is currently being evaluated in Phase 1/2 clinical trials to assess its safety, tolerability, and efficacy in slowing the progression of retinal atrophy.
- Other names
- AAV-CFI gene therapy
02
Targets
C1S (Complement component 1s subcomponent serine protease)B. burgdorferi (Borrelia burgdorferi)CFI (Complement factor I)
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