The company intends to use the funds to advance its lead optogenetics gene therapy Ray-001 into clinical trials for retinitis pigmentosa.Led by Paul Bresge, CEO, Ray Therapeutics is advancing 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, which uses an intravitreal approach where it diffuses from the vitreous into the retina and transduces primarily the RGCs. 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. Over 100 genetic mutations are known to cause RP and all types of inheritance patterns are recognized. Patients are typically diagnosed in their late teens, with symptoms including night blindness, reduced visual fields and eventual loss of visual acuity. As the disease progresses, retinal atrophy, and permanent loss of the light sensitive photoreceptors occur. The prevalence of RP is approximately 100,000 persons affected in the US.04/01/2022
