Vinit B. Mahajan, MD, PhD:

Hi, I'm Vinit Mahajan from Stanford University. I'm a retina surgeon and scientist and I had a chance to speak today on Nanoscope's three-year REMAIN trial. It's an amazing technology. We're taking patients with retinitis pigmentosa who have some of the lowest vision that you can imagine. These are patients with advanced degeneration and the best patient in the trial had 20/1700 vision. That's the best vision. Most of the vision is worse where we would think about it in the hand motion and light perception. These eyes don't have any photoreceptors, so how could you make them see? Nanoscope has a really amazing technology. They've created a new option and it's called optogenetics. What it does is it puts a protein inside the remaining cells of the retina to make them light sensitive. Can you take these ultra low vision patients and actually restore sight?

The REMAIN study, we've now looked at these patients over three years and the results are amazing. Patients on average gain three lines of vision, three lines of vision just like we're used to thinking about in most ophthalmology trials, and some of the patients even gained six lines of vision and this vision is sustained over three years. Now, the protein that we're putting into the eye is a synthetic protein. It's one of the most synthetic proteins ever put into a human. It even has domains from a CNME to make cells light sensitive. One of the things you're most concerned about is inflammation in any kind of gene therapy study, especially putting a synthetic protein in. The patients did really well. There were no serious adverse events. They got some oral steroid at the beginning of the study, and in the patients that had some inflammation, they got topical steroids. But overall, they did really well, no serious safety issues. What this really means is that with patients that have ultra low vision, no photoreceptors, we can think about restoring their vision with sigh. We've started with retinitis pigmentosa, but Nanoscope is now looking at geographic atrophy and Stargardt's disease as well.

The therapy is delivered as a single intravitreal injection inside the clinic. This is not a subretinal gene therapy trial and what's really important in these retinitis pigmentosa patients is that the therapy is gene agnostic. We're not trying to correct a specific gene mutation in a single disease gene. This is anybody with retinitis pigmentosa regardless of the gene.