Nanoscope Therapeutics Announces Presentation of Positive Results from Optogenetic Gene Therapy for Retinal Degenerative Diseases at the EURETINA 2021 Virtual Conference
Company’s lead product, an optogenetic gene therapy to restore vision for patients blinded by retinitis pigmentosa, is currently in late-stage Phase 2b trials in US
BEDFORD, TX (September 8, 2021) — Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for treatment of retinal degeneration diseases, today announced Dr. Santosh Mahapatra, principal investigator of the company’s clinical study on optogenetic gene therapy for vision restoration in people blinded by different gene mutations including ABCA4, will present findings from the study at the EURETINA 2021 Virtual Conference September 9 – 12. The conference is conducted by the European Society of Retina Specialists.
Dr. Mahapatra’s presentation, “52 Weeks Safety and Efficacy of Multi-Characteristic Opsin Enabled Vision Restoration in Patients with ABCA4 Mutation”, is Sunday, September 12, in Prize Papers 13 session on New Drug Treatments & Technologies.
ABCA4 mutations are associated with retinal degeneration diseases, including retinitis pigmentosa and Stargardt disease. Patients in the study received a single intravitreal injection of Nanoscope’s Multi-Characteristic Opsin (MCO) gene therapy and were followed for 52 weeks. All patients experienced improvement in visual acuity and function with no serious adverse events.
Nanoscope’s optogenetics therapy delivers MCO genes in proprietary AAV2 vectors into retinal cells leading to expression of ambient light-sensitive polychromatic opsins 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 diseases regardless of genetic mutations.
Nanoscope researchers also developed a Low-Vision Multi-Parameter Test (LVMPT) for assessing different attributes of functional vision. Michael Carlson’s presentation of the “Low-Vision Multi-Parameter Test for Monitoring Visual Function of Patients with Advanced Retinal Diseases” is Thursday, September 9, in the New Drug Treatment and Technology session.
The MCO-010 gene therapy and LVMPT device are being evaluated in a Phase 2b trial (NCT04945772) in the US. MCO-010 has received orphan drug designations from the US Food and Drug Administration for RP and Stargardt disease.
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 ocular gene therapies for patients with RP, Stargardt disease, and geographic atrophies.
Opus Biotech Communications