Optogenetic Gene Therapy Company Ray Therapeutics Awarded $4 Million Grant – Pulse 2.0

Ray Therapeutics a biotechnology company developing optogenetic gene therapies for patients with retinal degenerative conditions announced that the California Institute for Regenerative Medicine (CIRM) has awarded the company a $4 million grant to support the development of Ray-001, an optogenetic therapy for the treatment of retinitis pigmentosa and other inherited retinal diseases.

Retinitis pigmentosa (RP) is known as a heterogeneous group of genetic diseases that cause retinal degeneration leading to near or complete blindness for most patients. And 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 therapy RAY-001 for the treatment of retinitis pigmentosa, delivers light-sensing channelrhodopsin to retinal cells, to potentially restore vision using the power of optogenetics. And 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 the 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.

KEY QUOTES:

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. The unanimous positive vote from CIRMs 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.

Paul Bresge, Chief Executive Officer, Ray Therapeutics

Our goal is to always move the most promising research forward as fast as we can. A one-time treatment for retinitis pigmentosa such as Ray-001 would have significant impact for patients with this degenerative disorder. This technology also has the potential to serve the needs of underserved communities because RP has high prevalence in underserved, particularly Hispanic, ethnic populations. We look forward to supporting Ray Therapeutics in bringing this life-changing regenerative therapy to patients with genetic blinding disorders.

Dr. Maria T. Millan, President and Chief Executive Officer, California Institute for Regenerative Medicine (CIRM)

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Optogenetic Gene Therapy Company Ray Therapeutics Awarded $4 Million Grant - Pulse 2.0