Stargardt Disease Gene Therapy IND Cleared to Proceed


The investigational therapy has received orphan drug designation from the FDA for Stargardt disease as well as retinitis pigmentosa.

This article previously appeared on our sister site, HCPLive.

Nanoscope Therapeutics has received approval from the FDA for its investigational new drug (IND) application for MCO-010, a gene therapy for the potential treatment of Stargardt disease, a type of macular degeneration.

The IND approval gives way to a phase 2 trial of the therapy set to launch in the first half of 2022. The therapy, which is a multi-characteristic opsin ambient-light activatable optogenetic monotherapy, has shown potential to restore vision in patients with the degnerative disorder.

Notably, MCO-010 is designated as an orphan drug by the FDA for Stargardt disease and retinitis pigmentosa (RP).

The clincal-stage biotechnology company is currently conducting a Phase 2b multicenter, randomized, sham-controlled, double-masked study of MCO-010 for patients with retinitis pigmentosa.

Retinitis pigmentosa is a group of rare, genetic disorders associated with difficulty seeing at night and the inability to see peripherally due to the breakdown and loss of cells in the retina.

Stargardt is an inherited rare disease that affects children and adults. As a result of this retinal disease, photoreceptors in the eye degenerate. MCO-010 gene therapy makes them photosensitive by reprogramming the healthy retinal cells.

MCO-010 is a single intravitreal injection administered in a medical office setting. Proprietary AAV2 vectors deliver the MCO genes to the cells where they express polychromatic opsins and enable vision.

Patients with Stargardt and retinitis pigmentosa can utilize this therapy regardless of underlying gene mutations.

"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," David Boyer, MD, Retina-Vitreous Associates Medical Group, adjunct clinical professor of ophthalmology, Keck School of Medicine, University of Southern California said in a statement.

Nanoscope Therapeutics Announces FDA Clearance of IND for MCO-010 Gene Therapy in Stargardt Macular Degeneration Patients. News release. Nanoscope Therapeutics. January 25, 2022.
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