Nanoscope Therapeutics Announces FDA Clearance of IND for MCO-010 Gene Therapy in Stargardt Macular Degeneration Patients

Nanoscope Therapeutics Announces FDA Clearance of IND for MCO-010 Gene Therapy in Stargardt Macular Degeneration Patients

BEDFORD, TX (January 25, 2022) — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced it received IND clearance from the FDA to begin a Phase 2 trial of its Multi-Characteristic Opsin (MCO-010) ambient-light activatable optogenetic monotherapy to restore vision in Stargardt patients.

“This is another important milestone for Nanoscope, giving us the opportunity of conducting a second clinical trial in the U.S. of our proprietary MCO gene therapy platform, designed to restore vision for patients suffering from retinal degenerative diseases,” said Sulagna Bhattacharya, CEO of Nanoscope. “Currently there are no approved treatments for these conditions.”

This Phase 2 clinical trial for Stargardt is expected to begin in H1-2022. Nanoscope is currently conducting a Phase 2b multicenter, randomized, sham-controlled and double-masked study of MCO-010 for RP patients. MCO-010 has received orphan drug designations from the FDA for RP and Stargardt. Stargardt is a macular degeneration (MD) affecting children and adults. Progress on Nanoscope’s Stargardt program trial has implications for MCO as a platform and its impact beyond rare diseases.

“Presently all existing trials attempt to slow down the progression of vision loss in patients with Stargardt disease, Optogenetic approach is to restore vision. This can be a groundbreaking attempt to evaluate optogenetic gene therapy to improve vision in Stargardt patients. I’m excited by the potential MCO-010 has to restore vision for many patients with sight loss caused by outer retinal dystrophies including dry age-related macular degeneration”, said David S. Boyer, M.D., Retina-Vitreous Associates Medical Group and adjunct clinical professor of ophthalmology with Keck School of Medicine at the University of Southern California.

Stargardt is an inherited retinal disease involving dysfunction/degeneration of photoreceptors. Nanoscope’s MCO-010 gene therapy reprograms healthy retinal cells to make them photosensitive. It uses proprietary AAV2 vectors to deliver the MCO genes into the cells where they express polychromatic opsins enabling vision in different color environments. The therapy, involving a single intravitreal injection given in a medical office setting, is applicable for RP and Stargardt regardless of underlying gene mutations.

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 regeneration therapy for patients with RP, Stargardt, and dry age-related MD.


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.