SAN FRANCISCO--(BUSINESS WIRE)--Ray Therapeutics, a leading optogenetics company, announced today that the California Institute for Regenerative Medicine (CIRM) has awarded the company a $4M grant to support development of RTx-021, an optogenetic therapy for the treatment of geographic atrophy (GA), the advanced form of age-related macular degeneration (AMD).
RTx-021 is a next-generation visual optogenetic gene therapy to improve vision in patients with GA. The treatment delivers light sensing channel rhodopsin to retinal cells, to potentially restore vision using the power of optogenetics. Visual optogenetics is a disruptive technology that uses adeno-associated virus (AAV)-based gene therapy to deliver a payload expressing an optogenetic protein to the retina using a simple office-based procedure.
“RTx-021 has the potential to address a significant unmet need for patients with GA AMD. The additional funding and strategic support from CIRM will accelerate development of our 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 positive vote of confidence from CIRM provides strong validation for our scientific rationale, program development and team. We look forward to advancing our candidate into clinical trials in GA AMD where patients have limited options.”
“Our goal is to always move the most promising research forward as fast as we can,” said Dr. Abla Creasey, Vice President of Therapeutics Development, California Institute of Regenerative Medicine (CIRM). “A next-generation visual optogenetic gene therapy treatment for geographic atrophy (GA) age-related macular degeneration would have significant impact for patients with this degenerative disorder. We look forward to supporting the Company in bringing this life-changing regenerative therapy to patients with degenerative blinding disorders.”
AMD is the most common cause of vision loss in persons over the age of 65 in the United States and the world. It is estimated that over 18 million people in the United States are living with milder forms of AMD and a high proportion will progress to advanced AMD. The prevalence of AMD is expected to double by 2040. Advanced AMD is of 2 types: neovascular AMD and geographic atrophy (GA). Approximately 1.5 million Americans and more than 5 million patients worldwide are affected by GA and there is no treatment to improve vision in these patients.
About Ray Therapeutics
Ray Therapeutics is developing novel optogenetics gene therapies for patients with blinding diseases. The company is developing its lead candidate RTx-015 in retinitis pigmentosa, a degenerative retinal disease with significant unmet medical need. Our mission is to use optogenetics to restore vision, independent of genetic mutation for patients with inherited retinal diseases. Ray Therapeutics is based in San Francisco, CA. For additional information, 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.
