Age-related macular degeneration (AMD) is a prevalent cause of vision loss among individuals over the age of 65 globally. Over 18 million people in the United States alone are estimated to have milder forms of AMD, with a substantial portion expected to progress to advanced AMD. By 2040, the prevalence of AMD is anticipated to double.
Advanced AMD is characterized by two types: neovascular AMD and geographic atrophy (GA). GA affects approximately 1.5 million Americans and more than 5 million patients worldwide, and currently, there is no treatment available to improve vision in these individuals. Now, one tech startup is on a mission to develop therapy treatment to help patients suffering from this disease.
Enter Ray Therapeutics, a San Francisco, California-based biotech startup developing the next generation of visual optogenetic gene therapies to help patients with blinding diseases.
Today, Ray Therapeutics announced it has awarded the company a $4 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of its optogenetics technology platform. The funding will also be used to support the development of RTx-021, an optogenetic therapy designed for treating geographic atrophy (GA), the advanced form of age-related macular degeneration (AMD).
The treatment involves delivering light-sensing channel rhodopsin to retinal cells, potentially restoring vision through the power of optogenetics. Visual optogenetics is a groundbreaking technology that utilizes adeno-associated virus (AAV)-based gene therapy to deliver a payload expressing an optogenetic protein to the retina, achieved through a simple office-based procedure. In a statement, Ray Therapeutics CEO Paul Bresge said:
“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.”
Bresge added, “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.”
The $4 million grant from CIRM is seen as a significant boost to Ray Therapeutics’ efforts. Dr. Abla Creasey, Vice President of Therapeutics Development at CIRM, highlighted the importance of supporting promising research rapidly, especially for a next-generation visual optogenetic gene therapy that could have a substantial impact on patients with degenerative disorders.
Ray Therapeutics is at the forefront of developing innovative optogenetics gene therapies for patients with blinding diseases. The company is also working on its lead candidate, RTx-015, targeting retinitis pigmentosa—a degenerative retinal disease with significant medical needs. Ray Therapeutics’ mission is centered on utilizing optogenetics to restore vision, independent of genetic mutations, for patients dealing with inherited retinal diseases.