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FT-001
- Development stage
- Phase 2
- Lead developer
- Frontera Therapeutics
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Subretinal
01
Overview
FT-001 is an investigational gene therapy developed by Frontera Therapeutics for the treatment of *Leber Congenital Amaurosis-2* (LCA2), a severe inherited retinal disease caused by mutations in the *RPE65* gene. FT-001 utilizes an **adeno-associated virus (AAV) vector** to deliver a functional copy of the human RPE65 gene directly into the subretinal space of the eye. The therapy aims to restore visual function by enabling retinal cells to produce the missing or dysfunctional RPE65 protein. Administration is a **one-time subretinal injection**. As of 2023, FT-001 is in Phase 1 clinical trials with IND clearance from both the U.S. FDA and China CDE[1][2][4][5][7].
02
Targets
RPE65 (Retinal pigment epithelium-specific 65 kDa protein)
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