RARE Daily

Ray Therapeutics Raises $6 Million to Advance Novel Optogenetics Platform to Treat Blinding Diseases

January 4, 2022

Ray Therapeutics, a company developing optogenetic gene therapies for patients with blinding diseases, said it raised $6 million in seed financing round led by 4BIO Capital.

Photo: Paul Bresge, CEO and co-founder of Ray Therapeutics

The funding will be used to advance its optogenetic therapy, Ray-001, into clinical trials in retinitis pigmentosa (RP), a heterogeneous group of genetic diseases that cause retinal degeneration leading to near or complete blindness for most patients. At present, no effective treatment is available to restore vision once the photoreceptor cells have been lost. More than 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.

“This seed financing round enables us to advance our optogenetic gene therapy platform, pipeline and people, which could potentially restore vision for patients with blinding diseases, beginning with retinitis pigmentosa, an area of significant unmet medical need,” said Paul Bresge, CEO and co-founder of Ray Therapeutics.

Ray Therapeutics says optogenetics is a promising approach that has the potential to restore useful vision to visually impaired and blind individuals. In RP, a patient’s photoreceptors, the primary cells required for vision, are lost and cannot regenerate. However, inner retinal neurons downstream to photoreceptors, especially retinal ganglion cells (RGCs), persist in significant numbers even in late-stage disease. Ray Therapeutics’ lead candidate Ray-001 uses an intravitreal approach where it diffuses from the vitreous into the retina and transduces primarily the RGCs.

Unlike current RP gene therapies in development, which are targeted to specific genetic mutations or individuals with remaining photoreceptors that only address a small patient population, Ray-001 is mutation-independent.

“Ray’s experienced management team have a successful track record in developing regenerative medicines in ophthalmology indications including optogenetic therapies,” said Dmitry Kuzmin, managing partner of 4BIO Capital. “As a mutation-independent approach, Ray-001 has a significant advantage in that it can potentially treat more patients as a one-time intravitreal injection that may last for a lifetime.”

Author: Rare Daily Staff

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