Restore Vision Announces First Patient Dosed in Phase I/II Clinical Trial of RV-001 for Gene-Agnostic Retinitis Pigmentosa

• The first optogenetic "Chimeric Rhodopsin" gene therapy tested in humans.
• RV-001 starting dose was generally well tolerated in this open-label trial.
• RV-001 a gene-agnostic approach for RP patients.

TOKYO, Feb. 13, 2025 /PRNewswire/ -- Restore Vision Inc., a clinical-stage biotechnology company advancing gene therapies for retinal disorders, today announced that the first patient in its Phase I/II clinical trial with RV-001 has been dosed at Keio University Hospital. RV-001 utilizes adeno-associated virus (AAV) vectors to deliver a unique functional gene encoding "Chimeric Rhodopsin", a light-driven G-protein activation mechanism to re-establish light activation in retinal interneurons. The goal is to expand light sensitivity to retinal interneurons via an intravitreal injection of RV-001. This milestone marks the initiation of the world's first clinical trial of optogenetic gene therapy, which leverages a proprietary "Chimeric Rhodopsin" for patients with retinitis pigmentosa (RP). "Over 2 million people worldwide suffer photoreceptor loss in retinitis pigmentosa, resulting in severe vision loss and eventual blindness" said, CEO and Ophthalmologist Yusaku Katada, MD, PhD. "Our "chimeric rhodopsin" approach has broad potential to alter light sensitivity across multiple subtypes of RP patients.^[1] We are continuing to advance the Phase I/II trial to assess safety, light-activation and light-sensitivity in these patients, post-administration of our lead gene therapy product RV-001."

Phase I/II Safety, Tolerability, and Exploratory Efficacy Trial Highlights

• Patient Criteria: Participants with advanced RP, agnostic to genotype, and characterized by severe vision loss, no- to limited vision.
• Safety and Efficacy: In addition to safety, exploratory efficacy endpoints include improvements in light sensitivity and visual function.

Connecting Innovation, Academia, and Investment 

Restore Vision, Inc., a spin-out startup from Keio University, bridges cutting-edge academic innovation with entrepreneurial application to drive progress in retinal therapies. Developed through collaborations with Keio University School of Medicine and Nagoya Institute of Technology, RV-001's optogenetic technology addresses the limitations of traditional approaches by combining high light sensitivity with a unique G-protein activation mechanism.^[2],[3] This groundbreaking technology has the potential to be expanded to other retinal diseases, paving the way for broader applications in vision restoration. The Japan Agency for Medical Research and Development (AMED) and other academic grants, with key contributions from Keio University and Nagoya Institute of Technology, supported RV-001 development.

About Restore Vision

Restore Vision Inc., a clinical-stage gene therapy company, specializes in developing transformative gene therapies for inherited retinal disorders. By integrating academic excellence with entrepreneurial vision, the company is committed to pioneering treatments that address critical unmet medical needs and improve patient outcomes worldwide.

Media Contact

Hikaru Miyazaki
COO, Restore Vision Inc.
Email: [email protected] 

SOURCE Restore Vision Inc.