Restore Vision Announces First-in-Human Clinical Interim Results for RV-001, a GPCR-Based Optogenetic Gene Therapy

• Phase I/II clinical trial interim results, out to 168 days, from the two RV-001 dosing cohorts with advanced Retinitis Pigmentosa are presented at key May 2026 meetings: Eyecelerator and Retinal Therapeutics Innovation Summit.
• No dose-limiting toxicities (DLTs) or drug-related serious adverse events (SAEs) have been reported within the scope of this interim analysis, to date in either low or high dose cohorts (n=3/cohort) post the single intravitreal injection.
• The high-dose cohort started at no light perception at baseline and all individuals recorded light perception or better within one month of the single intravitreal injection
• Dose-dependent results across multiple endpoints: Consistent trends observed across visual acuity, full-field stimulus testing (FST), and functional vision assessments including mobility and object recognition tasks
• Measurable logMAR visual acuity reported: One high-dose patient reported chart-based visual acuity measurable by the Berkeley Rudimentary Vision Test (BRVT)
• Collection of additional safety and efficacy data across broader patient populations is planned

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."

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]

[Important Notice Regarding This Release]
This press release is intended solely to provide information regarding the Company's activities and research and development and does not constitute or imply any advertising, promotion, or recommendation of the efficacy or effect of any specific pharmaceutical product, regenerative medicine product, medical device, or other product. This release does not provide medical advice and is not intended for the purpose of recruiting or soliciting any patients to participate in any clinical trial.

The products in development or research stages mentioned in this release, including RV-001, have not been approved under the Pharmaceuticals and Medical Devices Act of Japan or by any regulatory authority in any other jurisdiction, and their safety and efficacy have not been established. Development plans, findings, conclusions, and other information described in this release may change based on future study results, regulatory review, or additional data.

The interim trial results described in this release are based on a limited number of patients in an ongoing study and may not be predictive of final results or results in future clinical trials with larger patient populations.

[Forward-Looking Statements]
This press release contains forward-looking statements based on current expectations, targets, and projections. Forward-looking statements in this press release include statements regarding the Company's clinical development plans, the potential benefits of RV-001, planned dose escalation, planned expansion into broader patient populations, global clinical development, and the anticipated timing of future milestones.

Actual results may differ materially from those expressed or implied in this release due to various risks, uncertainties, and other factors, including unexpected results in clinical trials, changes in regulatory requirements or regulatory authorities' judgments, the emergence of competing technologies, and other factors. The Company undertakes no obligation to update or revise any forward-looking statements contained in this release, whether as a result of new information, future events, or otherwise.

SOURCE Restore Vision Inc.