Dallas – September 13, 2026 -- The U.S. Food and Drug Administration has accepted and filed the Biologics License Application for MOGENRY, an optogenetic gene therapy from Nanoscope Therapeutics designed to restore vision in retinitis pigmentosa patients with severe vision loss. If approved, MOGENRY would become the first gene-agnostic treatment available for this patient population, which currently has no approved therapy.
RESTORE trial data anchors the regulatory filing
The BLA is supported by the Phase 2b/3 RESTORE trial (NCT04945772), a multicenter, randomized, double-masked, sham-controlled study that met its primary and key secondary endpoints, showing improvements in visual acuity at weeks 52 and 76. No treatment-related serious adverse events were reported. Additional efficacy and safety data come from the earlier Phase 1/2a trial (NCT04919473).
Long-term REMAIN follow-up strengthens durability claims
Most patients dosed in RESTORE have continued into the REMAIN long-term follow-up study, providing multi-year durability data included in the BLA. Nanoscope CEO Sulagna Bhattacharya said the RESTORE data combined with REMAIN's durability findings give the company confidence that MOGENRY could become a new standard of care for patients living with RP with severe vision loss.
Treatment bypasses genetic testing and surgical suites
MOGENRY is a one-time, in-office intravitreal injection built on Nanoscope's multi-characteristic opsin (MCO) platform, which makes surviving bipolar retinal cells directly light-sensitive after photoreceptor loss. It requires no genetic testing, invasive surgery, or repeat dosing, allowing administration within existing community retina practice workflows rather than requiring tertiary academic centers.
Physician advisors cite rare durability in ophthalmic gene therapy
Allen C. Ho, M.D. of Thomas Jefferson University and Wills Eye Hospital, Nanoscope's Chief Medical Advisor, said restoring vision is the hardest metric to achieve in ophthalmic gene therapy and that MOGENRY appears to clear that bar with years of improved vision. SriniVas Sadda, M.D. of UCLA and the Doheny Eye Institute, chair of Nanoscope's Visionary Advisory Committee, noted that sustained effect over years is exactly what physicians need to trust a one-time treatment.
Addressable population exceeds 100,000 patients in the U.S.
Retinitis pigmentosa affects more than 100,000 people in the U.S., with over 25,000 legally blind, making it a leading cause of blindness among the working-age population. Patients lose approximately 0.03 LogMAR per year on average, equivalent to roughly one line of vision every three years, with most legally blind by age 60.