The following transcript has been edited for clarity.

Diana V. Do, MD: Hi, I’m Diana V. Do, MD. Welcome to Retinal Physician. I’m joined today by Allen C. Ho, MD, FACS, from the Wills Eye Hospital. Some exciting news for retinitis pigmentosa patients. Tell us about MCO-010 (Mogenry; Nanoscope Therapeutics).

Allen C. Ho, MD, FACS: MCO-010, or Mogenry, is an optogenetic therapy for patients with severe vision loss due to retinitis pigmentosa. The news is that the US Food and Drug Administration (FDA) has announced a biologics license application (BLA) acceptance, which means—to be very clear—not an approval of the intravitreal injection for these patients with severe vision loss, but an acceptance of the application of the clinical package, the manufacturing package. And that’s a big step for any company to get a BLA acceptance. We’ll hear about their assessment in early 2027 or the first half of the year, I hope.

Dr. Do: This is an exciting step in drug development. Tell us about how MCO-010 works.

Dr. Ho: MCO-010 works as a gene therapy. It’s an in-office injected gene therapy. And what’s unique about optogenetic strategies in general and these next-generation strategies, because this type of strategy has been going on for over a decade, is that it is gene agnostic and mutation agnostic. So step back and think about gene therapy, first starting in humans in the subretinal space with Spark Therapeutics’s voretigene neparvovec (Luxturna) pioneered by Jean Bennett, MD, PhD, and Al Maguire, MD. And now we have dozens of approved gene therapies across all of medicine—I think validating, and importantly, opening minds to the potential power of gene therapy techniques.

This particular strategy uses gene therapy strategies in intravitreal injection with an AAV viral vector that encodes for a synthetic opsin, meaning a light-sensitive transmembrane protein. In the eye, we think about rods and cones having rhodopsin. There are actually multiple types of opsins in the body: melanopsin, neuropsin. They’re light sensitive, they’re in hair follicles, they’re in the iris, and they control different functions in the body like circadian rhythms and sleep/wake cycles. Rhodopsin is the native opsin that excites rods and cones to create vision.

This MCO-010/Mogenry is a synthetic transmembrane opsin that then bioactivates residual retinal cells. So that’s why it’s mutation agnostic. You don’t need rods or cones. It targets ON bipolar cells and therefore is mutation agnostic and then activates those cells to sense light. And that’s how vision is created.

Dr. Do: Well, this is so exciting that we’re at this stage of drug development, and then we’ll hear after the FDA reviews in 6 months or so. And potentially, it could be coming to the clinics if the review is favorable.

Dr. Ho: Yes. In stepping back and thinking about in our career, the patients with the greatest unmet needs are a significant population. There are over 100,000 patients just in the United States with RP, about 25,000 of which are legally blind or severely blind. Those who see these patients know that they’ve had the greatest unmet need maybe in our entire career. And for decades, we’ve been trying to not create false hope, but now I think there is real hope based on science, based on the safety and efficacy. And it’s being recognized not just here in the United States, but in the European Union (EU) and regulatory agencies in Japan as fast track/unmet need/orphan disease designation. So I’m excited about the potential for real hope based on science in this particular program.

Dr. Do: Well, we’re eager to learn more, and we await more news from the FDA and your company. Thank you so much for joining us, Allen. RP