Nanoscope Therapeutics Announces Presentations at the World Orphan Drug Congress
DALLAS, May 22, 2023 /PRNewswire/ -- Nanoscope Therapeutics Inc., a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, today announced that its Multi-Characteristic Opsin (MCO) gene therapy platform will be featured in multiple presentations at the World Orphan Drug Congress, taking place May 23-25, 2023 at Gaylord National Resort & Convention center in Washington, DC.
Details for the presentations are as follows:
Title: Pioneering mutation-agnostic gene therapy for retinal disorders
Dr. Mohanty will discuss Nanoscope's mutation-agnostic optogenetic therapy for treatment of various retinal degenerative diseases. Current gene therapies are intended to treat patients with specific gene mutations in outer retinal cells, while ambient-light activatable MCO optogenetic monotherapy targeting abundant inner retinal neurons has the potential to restore vision lost due to advanced retinitis pigmentosa (RP) and Stargardt macular degeneration, with degenerated outer retinal cells. MCO-010 (sonpiretigene isteparvovec, suspension for intravitreal injection) is the only broadband, fast, and most-light sensitive opsin currently in clinical trials. Efficacy and safety data from the RESTORE and STARLIGHT trials are consistent with those observed in the earlier Phase 1/2 trial (NCT04919473), and thus provide compelling evidence on the benefit/risk profile of MCO-010.
Title: Multi-Characteristic Opsins - A new wave of optogenetic therapeutics for vision restoration
Ms. Bhattacharya will provide high level update on Nanoscope's corporate and development path to commercialize MCO-010 for advanced RP, for which there is presently no approved treatment available. A significant proportion of patients treated with MCO-010 in randomized, double-masked, multicenter RESTORE trial exhibited improvements in functional vision assessed by vision-guided mobility, shape discrimination and visual acuity, along with a favorable safety profile. An observational study (NCT05820100) is ongoing to provide supportive evidence to validate the novel functional vision endpoints.
About Nanoscope Therapeutics Inc.
Nanoscope Therapeutics is developing gene-agnostic, sight restoring optogenetic therapies for the millions of patients blinded by retinal degenerative 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 fully enrolled a Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt patients (NCT05417126). MCO-010 has received FDA Fast Track designations and FDA orphan drug designations for both RP and Stargardt. Preclinical assets include non-viral laser delivered MCO-020 gene therapy for geographic atrophy.
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SOURCE Nanoscope Therapeutics