Positive Data of Optogenetic Gene Therapy for Patients Blinded by Retinitis Pigmentosa to be presented at Retina Society's Annual Conference


- 52 weeks follow-up from MCO-010 Vision Restoration in Retinal Degeneration patients to be presented
- Results of the Nanoscope's on-going Phase 2b US Trial (RESTORE) of its optogenetic gene therapy to restore vision for retinitis pigmentosa patients expected in Q4 2022


BEDFORD, Texas, Sept. 29, 2021 /PRNewswire/ -- Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for treatment of retinal degeneration diseases, today announced findings from its clinical study of its optogenetic gene therapy for people blinded by ABCA4 mutation will be presented at The Retina Society's 54th Annual Scientific Meeting in Chicago September 29October 2, 2021.

The presentation by Dr. Sai Chavala, M.D. titled "Optogenetic Vision Restoration in ABCA4 Mutation Retinal Degeneration Patients," is scheduled for Friday, October 1, 2021, at 11:03 am in the Grand Ballroom of the Ritz Carlton Hotel in Chicago. The Retina Society was founded in 1968 for educational and scientific purposes concerning the diagnosis, care and treatment of diseases and injuries to the retina.

Dr. Chavala, a professor at TCU-UNTHSC Medical School in Fort Worth, TX, will present findings from retinitis pigmentosa (RP) patients dosed intravitreally with Nanoscope's Multi-Characteristic Opsin (MCO) gene therapy. The RP patients with ABCA4 gene mutation received a single intravitreal injection of the gene therapy, were followed for 52 weeks, and all patients experienced improved visual acuity and function with no serious adverse events.

Nanoscope's optogenetics therapy uses a proprietary AAV2 vector to deliver MCO genes into retinal cells where they express polychromatic opsins sensitive to ambient light for vision restoration in different color environments. The therapy, which can be administered in a medical office, focuses on disease phenotype, enabling treatment of retinal degenerative diseases, such as RP, regardless of genetic mutations.

MCO-010 currently is in a randomized, double-masked Phase 2b multi-center trial (NCT04945772) in the US. Results are expected in Q4 2022. MCO-010 has orphan drug designations from the US Food and Drug Administration for RP and Stargardt disease, which are known to be associated with ABCA4 mutation.

About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing optogenetic therapies for giving sight to the millions of blind individuals suffering from retinal degenerative diseases, for which no cure exists. The company's pipeline includes optogenetics-based retinal gene therapies for patients with RP, Stargardt disease, and dry age-related macular degeneration.

Dan Eramian
Opus Biotech Communications


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SOURCE Nanoscope Therapeutics


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