Retinitis pigmentosa (RP), is a heterogeneous group of genetic diseases that cause retinal degeneration leading to complete, or nearly complete blindness for most patients. This is due to severe loss of photoreceptor cells. 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. Patients are typically diagnosed in their late teens.
The symptoms of RP include 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. Patients in the late stages of the disease, the anticipated target population for Ray-001, live in relative darkness. They are generally heavily reliant on family members or social services for assistance with daily living.
The prevalence of RP is approximately 100,000 persons affected in the US.
Optogenetics is a promising approach that has the potential to restore useful vision to visually impaired and blind individuals. In Retinitis Pigmentosa (RP) and other retinal degenerative conditions, the light-sensitive cells in the retina (photoreceptors) are lost and do not regenerate.
Our goal is to develop an effective optogenetic gene therapy to treat blindness in RP patients.
The principle behind the optogenetic approach for treating blindness involves imparting light sensitivity to light-insensitive second- and/or third-order retinal neurons (together called inner retinal neurons) by expressing genetically encoded light sensors, or optogenetic tools.
The optogenetic tools convert the light signal into an electrical signal expressed on the inner retinal neural cells to turn them into light-sensitive cells to replace the lost photoreceptor cells.
Our approach is genotype-independent and has the potential to treat all types of RP.
Our mission is to use optogenetics to restore vision, independent of genetic mutation, for patients with inherited retinal diseases.
Ray Therapeutics is developing an optogenetic therapy, Ray-001, that has the potential to restore vision in patients with inherited blindness, beginning with our lead indication in retinitis pigmentosa. Ray-001 is designed to be injected into the eye in a clinic, using an intravitreal approach, where it diffuses from the vitreous into the retina and transduces primarily the retinal ganglion cells (RGCs).
In retinitis pigmentosa, a patient’s photoreceptors, the primary cells required for vision, are lost and cannot regenerate. However, inner retinal neurons downstream to photoreceptors, especially RGCs, persist in significant numbers even in late-stage disease.
Our optogenetic therapy Ray-001 is expected to efficiently and predominantly transduce RGCs. The gene coded therapeutic protein localizes to the RGC membrane and transduces incoming light into electrical signals sent to the visual cortex for vision.
Although there are investigational gene therapy programs for RP patients, most are targeted to specific genetic mutations and patients that still have remaining photoreceptors, any of which only addresses a small percentage of the overall RP patient population.
Based on the durability of treatment demonstrated in Ray’s animal studies, Ray-001 is intended to be a one-time treatment via intravitreal injection that is sustainable for a lifetime.
This is a ground-breaking new approach to treat inherited retinal diseases, using the power of optogenetics.