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

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  — 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

Nanoscope Therapeutics, Inc.
Trinity Towers
2777 N. Stemmons Fwy.
Dallas, TX 75207
(817) 857-1186
  • Dr. Samarendra Mohanty, PhD |  Co-Founder & President

    Samarendra Mohanty (Co-Founder/President) is an inventor & serial entrepreneur with 20+ years experience in Biomedical Sciences. He is Co-Founder of several Biotechs and Biomedical device /diagnostic companies (developed & commercialized $100K+ biomedical instruments.)

    Samar obtained M.Tech in Applied Optics from the Indian Institute of Technology-Delhi and a PhD in (Bio)Physics from the Indian Institute of Sciences-Bangalore.

    His extensive Biomedical Technologies experience includes serving as Professor/Senior Scientist at the University of Texas;  University of California, Irvine; Center for Adv. Tech (India); Int. Mol. Biotech (Germany); Univ. Pavia (Italy); NUS (Singapore); and University of St. Andrews (UK). He has authored over 200 international patents and publications in leading journals including Nature and Nature Photonics.

    He is the Principal Investigator for major grants from the National Eye Institute including Audacious Goal Initiative and Bioengineering Research grants. He serves on the editorial board of journals and chairs an international conference on optogenetics. He is the winner of a 2019 Healthcare Heroes award (Fort Worth Business Press), Retinal Organoid Challenge Award, Audacious Goal Initiative Award (NIH), Finalist of Tech Titan, and NIH-Director’s Innovator Award.