- Ray Therapeutics, a privately held, 2-year-old biotechnology startup, has raised $100 million to fund development of gene therapies that can restore visual function in people with blinding diseases.
- The Series A financing, which Ray announced Tuesday, will help the company advance its lead program for the degenerative retinal disease retinitis pigmentosa into clinical testing within the next year.
- Ray’s focus is on what’s known as visual optogenetics. The company plans to use engineered viruses to deliver light-sensitive proteins into the eye, effectively converting retinal neurons into photoreceptors, which are lost in diseases like retinitis pigmentosa.
Diseases of the eye have been a popular target for gene therapy development, due in part to the potential for efficient delivery via injection as well as the lower risk of dangerous immune responses. Luxturna, the first gene therapy for an inherited disease approved in the U.S., treats a form of childhood vision loss, and developers like Johnson & Johnson, Roche, Regeneron and Biogen have explored gene therapy for other retinal disorders.
Ray is trying something a bit different. While gene therapies like Luxturna deliver a functional copy of a gene that’s missing or disabled in people with a specific eye disease, Ray aims to shuttle DNA encoding for an engineered type of light-sensitive protein known as a channelrhodopsin.
The idea is that expression of these proteins by retinal cells will make them responsive to light, compensating for the loss of photoreceptors that would normally do that job. This could enable the retina to again send visual signals to the brain, potentially restoring some visual function.
This approach, which draws on research into optogenetics, could allow Ray to treat blindness caused by many different genetic mutations, rather than tailoring a treatment to replace one specific gene. In retinitis pigmentosa, for example, mutations in more than 60 different genes are linked to the disease.
“Since the degenerative process in conditions like retinitis pigmentosa proceeds in the same fashion for all genes and mutations, optogenetics should work for all patients with these diseases,” wrote Ray CEO Paul Bresge, in an email.
Additionally, because Ray is delivering light-sensing proteins into retinal ganglion cells — and converting them into something akin to photoreceptors — it doesn’t need photoreceptors to still be present for its treatment to work. This could allow it to treat blindness that has progressed further than what’s treatable by other methods.
For Bresge, building Ray is both personal and professional. Bresge’s daughter has retinitis pigmentosa, and her diagnosis led Bresge to change careers from real estate to biotech. He previously founded jCyte, another biotech company that is developing a cell therapy for retinitis pigmentosa.
Ray plans to advance its lead program for the disease into a first-in-human study within the next 12 months, Bresge wrote in an email. The treatment is supported by preclinical research that was published in the journal Molecular Therapy in 2019.
Bresge and Ray’s other founders saw that research as an improvement on prior attempts at optogenetic treatment, which relied on proteins less sensitive to light, requiring patients to also use high-tech goggles.
In addition to retinitis pigmentosa, Ray also plans to go after other retinal degenerative diseases like Stargardt and geographic atrophy.
The company’s Series A round was funded by Novo Holdings, Deerfield Management, Norwest Venture Partners, Platanus, MRL Ventures Fund and 4BIO Capital.