Nanoscope Announces Presentation at the 68th Annual Biophysical Society Meeting
DALLAS, February 12, 2024 — Nanoscope Therapeutics, Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that Amir Singh, Ph.D. will present at the 68th Annual Biophysical Society Meeting taking place from February 10-14, 2024, at the Pennsylvania Convention Center in Philadelphia. The meeting provides a platform for researchers to share their latest discoveries and innovations in biophysics.
During his presentation, Dr. Singh will delve into the biophysical characteristics of the MCO molecule (MCO-010). He will reveal the unique mechanism of action of MCO-010 while detailing its distinctive bioinformatics, molecular structure, and photosensitive properties. The presentation underscores the significance of biophysical research and its potential impact on advancing our understanding of vision restoration therapies.
Title: Elucidating the structural and functional uniqueness of multi-characteristic opsin, a highly bioengineered, ambient-light-sensitive molecule, with broadband spectral responsiveness and fast kinetics to modulate neural activities
Date: Wednesday, February 14, 2024
Time: 10:30 a.m. ET
Location: Exhibit Hall AB
Presenter: Dr. Amir Kumar Singh
MCO-010’s distinctive structure enables functions like broadband light sensitivity, high dynamic range, and rapid activation kinetics, making it promising for restoring vision in those with degenerative retinal diseases. Its ability to function in ambient lighting without external stimulation devices opens new possibilities for vision restoration. This novel optogenetic approach is a significant step toward treatments for neurological disorders involving abnormal neural circuitry activity.
Current gene therapies aim to treat patients with specific gene mutations, and are further limited in advanced disease with degenerated outer retina cells. Ambient-light activatable MCO optogenetic monotherapy targets abundant inner retinal neurons and has the potential to restore vision permanently lost due to advanced RP. MCO-010 (sonpiretigene isteparvovec, suspension for intravitreal injection) is the only broadband, fast, and highly light-sensitive opsin currently in clinical trials. With bipolar cell targeting via mGluR6 promoter-enhancer, the MCO-010 expression cassette is designed for restoring high-quality vision in real-world environments. The proprietary AAV2 vector allows robust transduction of MCO-010 in bipolar cells upon intravitreal injection. The Phase 1/2 study of MCO-010 in advanced RP patients demonstrated favorable safety and dose-dependent improvement in visual acuity. Nominally significant visual acuity improvements in MCO-010 treated patients were observed in comparison to control in Phase 2b RESTORE trial. There were also nominally significant functional vision improvements as assessed by multi-luminance vision-guided mobility and shape discrimination, along with a favorable safety profile.
About Nanoscope Therapeutics, Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by inherited retinal diseases for which no cure exists. The company’s lead asset, MCO-010, recently reported topline results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial in the U.S. for retinitis pigmentosa (NCT04945772). The company has also recently completed the Phase 2 STARLIGHT trial of MCO-010 therapy in patients with Stargardt disease (NCT05417126). MCO-010 has received FDA fast track designations and FDA orphan drug designations for both retinitis pigmentosa and Stargardt. Preclinical assets include non-viral laser-delivered MCO-020 gene therapy for geographic atrophy.