Optogenetics Start-Up Raises $6M for Retinal Therapy

(Sakuie, Pixabay)

4 Jan 2022. A new company developing a therapy using light-sensitive proteins for treating an inherited eye disorder is raising $6 million in seed funds. Ray Therapeutics in San Diego, is based on research in optogenetics, an emerging process that combines light signals with genetic engineering for treating disease, in this case retinitis pigmentosa.

Retinitis pigmentosa is caused by genetic defects that damage cells in the retina affecting night vision, and sometimes peripheral and central vision, leading to blindness. The retina is a layer on the back of the eye with light-sensitive cells called photoreceptors that convert light into neural signals, carried into the brain by the optic nerve. National Eye Institute estimates retinitis pigmentosa affects 1 in 4,000 people, with few effective therapies so far for restoring vision loss from the disease.

Optogenetics is a process using light waves combined with genetics to influence biological processes. One of the first fields to apply optogenetics is neuroscience, involving responses to light signals by photosensitive proteins on neurons. Zhuo-Hua Pan, professor of ophthalmology at Wayne State University in Detroit and scientific director of the school’s vision research center, studies optogenetics for treating inherited eye disorders.

Pan’s research led an optical gene therapy technique that expresses a form of rhodopsin, a receptor protein found in retinal cells that supports regeneration of photoreceptors. In tests with lab animals. Pan and colleagues demonstrated the ability to restore vision with rhodopsin, by converting surviving inner retinal neurons into photosensitive cells like those damaged in people with retinitis pigmentosa. Pan is also a scientific advisor to the company.

Designed to work independently of mutations

Ray Therapeutics adapts Pan’s research for its treatment candidate for retinitis pigmentosa code-named Ray-001. Ray-001 is designed as a one-time treatment delivered with benign adeno-associated viruses into a person’s vitreous, the gel-like fluid in the eye with fibers that attach to the retina. The vitreous carries Ray-001 to retinal ganglion cells that the company says survive in large numbers, even in advanced cases of retinitis pigmentosa, where they help regenerate photoreceptor cells.

Once injected, says the company, Ray-001 stimulates regrowth of photoreceptors from surviving retinal ganglion cells. Ray Therapeutics says more than 100 mutations are associated with the disease, but unlike other gene therapies, Ray-001 works on any form of retinitis pigmentosa.

Ray Therapeutics is raising $6 million in seed funds, led by life science venture investor 4BIO Capital in London. No other participants in the round are disclosed. “As a mutation-independent approach,” says 4BIO Capital managing partner Dima Kuzmin in a statement, “Ray-001 has a significant advantage in that it can potentially treat more patients as a one-time intravitreal injection that will last for a lifetime.”

The company says the funds will help advance Ray-001 to clinical trials. “This seed financing round led by top-tier advanced therapies investor 4BIO Capital,” adds Ray Therapeutics co-founder and CEO Paul Bresge, “enables us to advance our optogenetic gene therapy platform, pipeline, and people.

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