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RV-001
- Development stage
- Unknown
- Lead developer
- Restore Vision
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Ophthalmic
01
Overview
RV-001 is an optogenetic gene therapy developed by Restore Vision Inc., a spin-out from Keio University, for the treatment of retinitis pigmentosa (RP). The therapy utilizes an adeno-associated virus (AAV) vector to deliver a gene encoding 'Chimeric Rhodopsin' into retinal interneurons. This chimeric protein employs a light-driven G-protein activation mechanism to restore light sensitivity in the remaining retinal cells of patients with advanced RP. RV-001 is designed as a gene-agnostic treatment, meaning it is intended to be effective regardless of the specific genetic mutation causing the disease. It is administered via intravitreal injection and entered Phase I/II clinical trials in early 2025.
- Other names
- chimeric rhodopsin gene therapyAAV-based visual restoration therapy
02
Targets
GHSR (Growth hormone secretagogue receptor)
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