Nanoscope Advances Optogenetic Therapy for Retinal Diseases

Dallas, Texas, September 23, 2026

Nanoscope to Present Macular Degeneration Data

At the 26th Euretina Congress, Jordi Mones, MD, PhD, will present data titled Vision Improvement in Macular Degeneration with Sonpiretigene Isteparvovec Optogenetic Therapy: First Direct-to-Phase 2 Randomized, Controlled Trial. The presentation is scheduled for October 4, 2026, during the AMD/CSC session at the VIECON – Vienna Congress & Convention Center. The company said the presentation will provide clinical findings from its program evaluating sonpiretigene isteparvovec, an investigational optogenetic therapy designed to address vision impairment associated with retinal degeneration. Nanoscope’s technology is based on its proprietary Multi-Characteristic Opsin (MCO) platform. The approach uses a genetically engineered synthetic opsin intended to make surviving retinal cells sensitive to light following photoreceptor loss. According to the company, the therapy delivers the MCO gene to retinal bipolar cells through a one-time intravitreal injection, with the objective of enabling remaining visual circuitry to respond to ambient light. The investigational therapy does not depend on identifying a specific disease-causing genetic mutation, reflecting Nanoscope’s disease-agnostic approach to retinal degeneration.

Four-Year Retinitis Pigmentosa Data

Nanoscope will also present four-year REMAIN extension data from the RESTORE Phase 2b/3 trial evaluating MCO-010 in patients with retinitis pigmentosa. Benjamin Bakall, MD, PhD, will present the data as a poster during the Inherited Retinal Diseases ePoster session on October 1, 2026. The company said the analysis is intended to provide longer-term information regarding the durability of vision improvements observed in the clinical development program. The RESTORE program is a multicenter, randomized, double-masked, sham-controlled Phase 2b/3 clinical trial evaluating MCO-010 in patients with RP. Nanoscope has reported positive results from the study and is developing MCO-010 as an investigational treatment for severe vision loss associated with retinal degeneration. The company has also submitted a Biologics License Application (BLA) for MOGENRY®, the proposed name for sonpiretigene isteparvovec, to the U.S. FDA.

Optogenetic Platform Expands Retinal Disease Pipeline

The upcoming presentations will also highlight the broader development potential of Nanoscope’s optogenetic gene-therapy platform. The company is studying MCO-010 across multiple retinal conditions, including retinitis pigmentosa, Stargardt disease, and geographic atrophy. Nanoscope has reported that the therapy has received regulatory designations including FDA Fast Track and Orphan Drug designations for RP and Stargardt disease, as well as Regenerative Medicine Advanced Therapy designation for Stargardt disease. These designations facilitate regulatory development but do not establish safety or efficacy or guarantee approval. In addition to the scientific presentations, Nanoscope executives will participate in the Ophthalmology Futures Forums Retina Forum 2026 in Vienna on September 30. Samarendra Mohanty, PhD, Co-Founder, President and Chief Scientific Officer, is scheduled to provide a company presentation, while Chief Medical Officer Samuel Barone, MD, will participate in a panel focused on geographic atrophy and intermediate age-related macular degeneration therapeutics.

The company’s latest disclosures underscore its focus on developing genetic and optogenetic therapies for retinal diseases involving photoreceptor loss. The upcoming Euretina and Retina Forum presentations are expected to provide additional clinical and development information on MCO-010 across different retinal conditions. While the reported findings are part of an ongoing investigational program, longer-term clinical data and regulatory review will be important in determining the therapy’s potential role in treating patients with retinal degeneration.

Source: Nanoscope Therapeutics press release