CIRM Provides Ray Therapeutics $4 Million Grant to Advance Experimental Retinitis Pigmentosa Therapy

The California Institute for Regenerative Medicine has awarded a $4 million grant to Ray Therapeutics, to support development of RAY-001, an optogenetic therapy for the treatment of retinitis pigmentosa and other inherited retinal diseases.

Photo: Paul Bresge, CEO of Ray Therapeutics

Retinitis pigmentosa (RP), is a heterogeneous group of genetic diseases that cause retinal degeneration leading to near or complete blindness for most patients. The severe loss of photoreceptor cells that occurs in this genetic degenerative disease leads to partial or complete blindness. At present, no effective treatment is available to restore vision once the photoreceptor cells have been lost.

Ray Therapeutics’ lead experimental therapy RAY-001 for RP delivers light sensing channel rhodopsin to retinal cells, to potentially restore vision using the power of optogenetics. Based on the durability of treatment demonstrated in preclinical studies, RAY-001 is intended to be a one-time treatment via intravitreal injection that is sustainable for a lifetime. Unlike current RP gene therapies in development, which are targeted to specific genetic mutations or individuals with remaining photoreceptors that only address a small patient population, Ray-001 is mutation-independent.

“Ray-001 has the potential to address a significant unmet need in patients who suffer from retinitis pigmentosa. The funding and strategic support from CIRM will accelerate development of our lead optogenetics candidate into clinical trials for blind and nearly-blind patients in desperate need of new therapies, without the need for supplementary eyewear or devices for additional light stimulation,” said Paul Bresge, CEO of Ray Therapeutics. “The unanimous positive vote from CIRM’s independent reviewers, and obtaining the highest score in our application cohort, provides strong validation for our scientific rationale, program development and team. We look forward to advancing our candidate into clinical trials in retinitis pigmentosa.”

Author: Rare Daily Staff