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Ray Therapeutics Nets $4M CIRM Grant to Advance Geographic Atrophy Gene Therapy

NEW YORK – Ray Therapeutics on Monday said it will use a $4 million grant from the California Institute for Regenerative Medicine (CIRM) to expedite the clinical development of an optogenetic therapy for geographic atrophy (GA).

San Francisco-based Ray Therapeutics is developing RTx-021 as a gene therapy for improving vision in patients with GA, an advanced form of age-related macular degeneration (AMD). RTx-021 uses an adeno-associated virus to deliver channelrhodopsins, proteins that can sense light, to retinal cells. The company wants to develop a gene therapy that can deliver these optogenetic proteins to patients' retinas as an office-based procedure.

AMD is the most common reason in the US people over the age of 65 lose their vision. Approximately 18 million people have milder forms of AMD, but a significant segment develop one of two advanced forms of the disease: neovascular AMD and GA. The 1.5 million people in the US with GA have no treatments that improve their vision.

"RTx-021 has the potential to address a significant unmet need for patients with GA AMD," Ray Therapeutics CEO Paul Bresge said in a statement. "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."

In addition to RTx-021, Ray Therapeutics is also developing its lead candidate RTx-015 as a genotype-agnostic treatment for retinitis pigmentosa. Earlier this year, the company raised $100 million in an oversubscribed Series A financing. In 2022, the company brought in $6 million in seed funds.