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.
