Restore Vision reports first clinical results for vision gene therapy

TOKYO, May 1, 2026 /PRNewswire/ -- Restore Vision Inc., a clinical-stage biotechnology company advancing gene therapies for retinal disorders, today announced the interim clinical results from its ongoing Phase 1/2 first-in-human trial of RV-001 in Japan. It will present the results at two premier events on May 1, 2026 in Denver, Colorado: Eyecelerator @ ARVO 2026 and the Retinal Therapeutics Innovation Summit, organized by the Foundation Fighting Blindness and the Casey Eye Institute at Oregon Health & Science University.

Presentation Details

About RV-001
RV-001 is a gene therapy designed to restore vision in individuals with advanced retinitis pigmentosa (RP), the leading inherited cause of blindness in young adults, affecting approximately two million people worldwide. RV-001 delivers a chimeric rhodopsin — a novel photosensitive protein that combines the high sensitivity of animal rhodopsin with the self-regenerating properties of microbial rhodopsin — via an adeno-associated virus (AAV) vector through a single intravitreal injection. As a theoretical property when compared with conventional optogenetic approaches that rely on microbial ion channels requiring intense light or external devices, RV-001 activates native G-protein-mediated phototransduction, with the potential to enable high-sensitivity improvement in visual function under natural ambient light conditions without goggles or external devices. RV-001 is a G-protein-coupled receptor (GPCR)-based optogenetic therapy to enter clinical trials for all individuals with RP, regardless of the underlying genetic mutation.

Phase 1/2 Clinical Trial Overview
The ongoing Phase 1/2 first-in-human trial is an open-label, dose-escalation study evaluating the safety and preliminary efficacy of a single intravitreal injection of RV-001 in individuals with advanced retinitis pigmentosa. The trial enrolled patients with no light perception (NLP) in low-dose and high-dose cohorts at a clinical site in Japan. The primary endpoint is safety; exploratory efficacy assessments include visual acuity, full-field stimulus testing (FST), and functional vision testing, including mobility tasks and table-top object recognition. The interim results will be interpreted in the context of a full study which is still in progress.

Key Clinical Findings
In the dose-escalation phase with six patients enrolled across two cohorts, RV-001 was associated with no new findings suggestive of significant safety concerns; specifically, no dose-limiting toxicities (DLTs) or drug-related serious adverse events (SAEs). No safety or tolerability concerns were reported.

Dose-dependent trends across vision endpoints were suggested. In the high-dose cohort, all three enrolled patients progressed from no light perception (NLP) to light perception (LP) or better within one month of treatment, with one patient further reporting chart-based visual acuity measurable by the Berkeley Rudimentary Vision Test (BRVT). In the low-dose cohort, one of three patients progressed to light perception at approximately three months.

These visual acuity gains were corroborated by improvements in full-field stimulus testing (FST) and functional vision assessments, including mobility tasks and table-top object recognition. The convergence of signals across multiple independent measures suggests the possibility of biologically meaningful changes in vision.

"These interim data demonstrate that RV-001 can contribute to improvement of visual function in completely blind patients through a single injection, without any external devices," said Yusaku Katada, M.D., Ph.D., CEO of Restore Vision. "What makes these results particularly encouraging is the convergence of improvement across multiple independent endpoints — visual acuity, retinal sensitivity, and real-world functional tasks — all pointing to changes in visual function that may have clinical significance. We look forward to sharing these findings with the scientific community at ARVO and the Innovation Summit."