In this episode of Raising Biotech, host Surani Fernando delves into the world of optogenetics with Paul Bresge, CEO and founder of Ray Therapeutics, and Professor José-Alain Sahel, a distinguished pioneer in the field from the University of Pittsburgh and Vision Institute in Paris. The conversation covers Ray’s mission to develop gene therapies for severe retinal diseases, the company’s origins leading to an impressive $100 million Series A round and the challenges of advancing optogenetic treatments to clinical trials.

Ray Therapeutics focuses on restoring vision for patients suffering from advanced retinal disorders like retinitis pigmentosa and Stargardt’s disease. Bresge explains that their lead asset, an optogenetic gene therapy, targets remaining retinal cells in patients who have lost most of their photoreceptor cells. By using an AAV gene therapy to reprogram these cells, Ray’s platform aims to restore functional vision, potentially providing a treatment where no options currently exist for late-stage disease sufferers.

The motivation behind Ray Therapeutics is personal for Bresge, whose daughter was diagnosed with retinitis pigmentosa. This personal connection fueled his determination to enter the biotech field, eventually leading him to co-found Ray Therapeutics in 2021 with Sean Ainsworth, formerly CEO of RetroSense. Drawing on groundbreaking work from optogenetics pioneer Dr. Zhuo-Hua Pan, they built Ray’s platform to develop therapies that can be activated under normal lighting conditions—eliminating the need for light-enhancing devices, which have been a major limitation in other programs.

Ray Therapeutics initially secured $6 million in seed funding and a $4 million grant from the California Institute of Regenerative Medicine, before raising $100 million in a 2023 Series A round led by Novo Holdings. Bresge attributes their success to the strength of their science and management team, particularly their development of a light-sensitive protein functional in normal lighting and the replication of promising animal study results, distinguishing Ray in the competitive optogenetics space.

Professor Sahel, while not directly involved with Ray, offers an expert perspective on the company’s approach. He notes that Ray’s efforts to eliminate the need for light-amplifying devices mark a significant step forward in the field. However, he emphasizes that achieving the delicate balance between light sensitivity and functionality will be key as they move toward clinical trials.

Looking ahead, Ray Therapeutics plans to begin trials in 2024 with a focus on retinitis pigmentosa, aiming to achieve meaningful efficacy results within weeks of dosing. Bresge remains optimistic about Ray’s chances for success and the potential for optogenetics beyond ophthalmology, hinting at future neurological applications. With sufficient funding to reach major milestones, he keeps an open mind about strategic partnerships or even a possible IPO, but for now, the focus remains on getting the therapy to patients.

Interested in how Ray Therapeutics plans to revolutionize vision restoration and make optogenetic treatments a reality? Tune into this episode of Raising Biotech for insights from Paul Bresge and Professor José-Alain Sahel.

[This episode was published on 28 May 2024]