SAN DIEGO--(BUSINESS WIRE)--Ray Therapeutics, a biotechnology company developing optogenetic gene therapies for patients with retinal degenerative conditions, announced today that the California Institute for Regenerative Medicine (CIRM) has awarded the company a $4M grant to support development of Ray-001, an optogenetic therapy for the treatment of retinitis pigmentosa and other inherited retinal diseases.
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 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. 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, Chief Executive Officer, 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."
“Our goal is to always move the most promising research forward as fast as we can,” said Dr. Maria T. Millan, President and Chief Executive Officer, California Institute for Regenerative Medicine (CIRM). “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.”
About Ray Therapeutics
Ray Therapeutics is developing novel optogenetics gene therapies for patients with blinding diseases. The company is developing its lead candidate Ray-001 in retinitis pigmentosa, a degenerative retinal disease with significant unmet medical need. The company’s mission is to use optogenetics to restore vision, independent of genetic mutation for patients with inherited retinal diseases. Ray Therapeutics is based in San Diego, CA. For additional information, please visit www.raytherapeutics.com.
About CIRM
At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission.
To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today’s most promising stem cell technologies.
With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is the world’s largest institution dedicated to helping people by bringing the future of cellular medicine closer to reality.
For more information go to www.cirm.ca.gov.
