A gene editing therapy showed safety and improvement in cone photoreceptor function in patients with CEP290-associated inherited retinal degeneration due to an IVS26 variant, according to a phase 1/2 study.
“This trial shows CRISPR gene editing has exciting potential to treat inherited retinal degeneration,” study author Mark E. Pennesi, MD, PhD, told Healio. “There is nothing more rewarding to a physician than hearing a patient describe how their vision has improved after a treatment.”
The therapy, EDIT-101 (Editas Medicine), is a (CRISPR)-CRISPR-associated protein 9