OIS Retina: What’s on the Horizon for Gene and Cell Therapy?


Click here to watch the video version of this podcast.

A panel of cell and gene therapy experts gathered at the recent sold-out OIS Retina Innovation Summit in New York City to talk about the rapidly evolving technology and its potential in ophthalmology.

Paul Bresge, CEO of Ray Therapeutics, kicked off the discussion by explaining the benefits and risks of optogenetics, a method of genetic engineering. In ophthalmology, developers make potentially restorative changes to the genetic code via retinal ganglion cells. “It’s a complimentary and different approach” from cell and gene therapy, he said. That’s because, instead of stopping the progression of disease, optogenetics aims to restore functional vision.

Next, Tom Aaberg Jr., MD, PhD, CMO of Neurotech Pharmaceuticals, discussed the potential of the company’s implantable device that contains genetically modified cells. He talks about what the company is learning from dual Phase III studies.

Glenn Yiu, MD, PhD, a professor at the University of California Davis in Sacramento, took a short but deep dive into biodistribution and transception efficiency in alternative delivery modes to intravitreal injections.

Rounding out the discussion, Claire Gelfman, PhD, chief scientific officer for Foundation Fighting Blindness, gave the patient perspective on these innovations. Thanks to the success of Luxturna for inherited retinal disease, she said more patients are willing to consider gene therapy. Cell therapies, which lack clinical proof right now, may take more time and education. But things change in an instant.

Listen to the podcast to hear the full conversation, including:
• How Neurotech Pharmaceuticals overcame Chemistry, Manufacturing, and Controls, as well as clinical operations challenges when developing its therapy to treat macular telangiectasia.
• Results from early studies of Ray Therapeutics’ optogenetic therapy, Ray-001.
• Delivery mechanisms that can potentially overcome the limitations of intravitreal injections and their immunogenic effect.
• What patients really think about emerging cell and gene therapies.
• The scoop on Foundation Fighting Blindness, including its free genetic testing and counseling services, its extensive patient registry, and its venture arm.

Moderating the panel was OIS Retina cochair John S. Pollack, MD, a retina specialist with Illinois Retina Associates and CMO of jCyte.

Click “play” to listen.