Dr. Gonzalez shares 126-week topline results from the REMAIN study, assessing optogenetic therapy MCO-010 (Nanoscope Therapeutics).
Ahead of the Clinical Trials at the Summit (CTS) meeting in Las Vegas, Nevada, Victor Gonzalez, MD, shared an overview of his presentation. Dr. Gonzalez is a trial investigator in the REMAIN follow-up study, assessing 126-week outcomes from MCO-010 (Nanoscope Therapeutics), an optogenetic therapy for vision restoration in patients with retinitis pigmentosa. During the CTS session on inherited retinal diseases, Dr. Gonzalez provided information on the multi-characteristic opsin (MCO) which aims to re-sensitize photoreceptors in the retina. The FDA has granted Fast Track and Orphan Drug designations to the ambient-light activatable MCO optogenetic monotherapy.
"Ophthalmology has had very important improvements and advances in the way we treat diseases...We're great at trying to stabilize; rarely can we recover," Dr. Gonzalez told Modern Retina. "I think the big step forward is going to be restoration of vision in patients. We've taken huge strides forward in slowing down or stopping a disease process, but we have not had the ability to recover what's been lost. The next frontier, the next step forward, is to have a way to restore vision, whether it's genetics, whether it's stem cell gene therapy. We're going to have an incredible armamentarium to be able to do exactly what I just described...We're at an important crossroads."
One particularly promising characteristic of the therapeutic candidate is its applicability in varying pathogeneses of retinitis pigmentosa. "[MCO-010] is agnostic to the genetic cause, so it can be used for any cause of retinitis pigmentosa," he said. This could indicate efficacy in treating other inherited retinal diseases, he added. "[And] it does not require photoreceptors to be present, so for that reason, you can treat very severe, advanced disease."
In this video interview, Dr. Gonzalez details the next big shift in ophthalmology, characterized by vision restoration and structural reactivation.
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