The following transcript has been edited for clarity. 

Hello, my name is Ramiro S. Maldonado, MD. I am at Duke University. I had the pleasure to be at Innovate Retina in New Orleans, presenting results from the RESTORE trial of MCO-010 (Nanoscope Therapeutics). This is an optogenetic therapy that may become an option for patients with retinitis pigmentosa (RP) with very advanced disease.

Let’s remember that for many, many years we have been trying to have treatments for our patients with RP. And there is a lot going on for early RP—gene therapy, nutritional supplements, etc. But the advanced cases—those patients with hand-motion or light-perception vision—those patients are in need of treatments. Here is where optogenetics comes in. It’s a therapy that uses intravitreal injection delivery to rescue the retinas by converting bipolar cells—remember in advanced cases, there are no photoreceptors, so we can use this technology to convert the bipolar cells into light-sensitive cells. These bipolar cells will be able to produce vision perception there.

It’s very important to note that in the trial, MCO-010 met the endpoints, especially gains on visual acuity in mobility and also shape discrimination. The important factor as well is that the efficacy was maintained over 3 years. Safety was encouraging; no significant side effects were noted. This is extremely exciting for our field and especially for those patients with advanced disease. RP