ARVO 2024: Mutation agnostic optogenetic programme demonstrates possible treatment for patients with severe retinal degeneration

Allen Ho, MD, is Co-Director, Wills Eye Retina Service and Director, Retina Research, at Wills Eye Hospital in Philadelphia, Pennsylvania. At the 2024 ARVO Annual Meeting, Dr Ho presented a paper on the 12 month results of a mutation agnostic optogenetic programme for patients with severe vision loss from retinitis pigmentosa.

Editor's note: The below transcript has been lightly edited for clarity.

Allen Ho, MD: Hello, I'm Dr Allen Ho from Wills Eye Hospital and Mid Atlantic Retina. And today at the ARVO 2024 meeting, I'm presenting a paper on the 12 month results of an mutation agnostic optogenetic programme for patients with severe vision loss from retinitis pigmentosa. This is a randomised controlled study with a high and low dose intervention of a gene therapy injected once that shows durable visual acuity benefits and visual function benefits. So let's go into that a little bit.

This is optogenetics, which is taking a genetically engineered protein that's a light-sensitive opsin. We have opsins in our body naturally, like rhodopsin in our rods and cones, or melanopsin in our skin cells or in ganglion cells in the eye that control circadian rhythm. Optogenetics aims to activate existing layers of cells when there is a retinal degeneration or retinal photoreceptor loss. In this program, we saw 40% of the subjects having three-line gains in vision while only one out of nine subjects in the sham group showed an improvement of three line gains. A third of the subjects had six-line gains in vision. So we're talking about taking someone who [has] light perception or bare hand motions, to maybe on the chart vision, counting fingers or 2800 or 2400 vision. And the data show this was done with statistical significance.

What does this mean for our field? It means that we are on the verge of having something that is mutation agnostic for all these patients with severe vision loss due to retinal degeneration, and maybe having something for them. I used to say, you know, to these patients, to try and avoid false hope that we may have something and we're working on things. Now it seems like we can say that with more confidence and with a timeline. This is going to be submitted hopefully for a biologic application to the FDA in the United States, hopefully by the end of this year, 2024. That's the summary on optogenetics in the 1 year data set. These visual acuity results were supported by functional visual acuity assessments like shape discrimination, grabbing an object from a shelf, a round object or square object. It was also supported by statistically significant improvements from baseline in a mobility course test where a light at the end of the room is provided to the patient that is in the trial, and you have to navigate through a course to that light. So stay tuned and there is something on the horizon for these ultra low vision patients.

I serve as an advisor to a number of gene and cell therapy companies. I'm an advisor to the Nanoscope Optogenetics program and company.