The improvement of a patient, who's had retinitis pigmentosa (RP) for 40 years, has been reported in a phase 1/2 clinical trial of GenSight Biologics' mutation-agnostic gene therapy. The treatment introduces genetic instructions from algae to the surviving layer of retina at the back of the eye. At first, the patient only responded to amber light, but recently identified white lines on crosswalks and identified items when wearing goggles which detect changes in light intensity.
RP causes vision loss through the death of light sensing cells and affects more than 2 million people worldwide. Optogenetics therapy uses light to control the activity of brain cells to restore vision. “The findings provide proof-of-concept that using optogenetic therapy to partially restore vision is possible" Prof Botond Roska, University of Basel.
There are several promising approaches aimed at restoring light for RP patients. In 2017, Spark Therapeutics, Inc., now part of Roche, won FDA approval for their mutation-specific RP gene therapy, Luxturna. It is uncertain whether a mutation-specific approach can be used for all patients, as the disease can be caused by changes in over 70 genes.
James Pickering is our Managing Director and an eye care recruitment expert who has developed an extensive client base and candidate network. He regularly writes articles about industry developments and has received numerous recommendations from industry leaders. You can also connect with James on LinkedIn to stay up to date on the latest eye care news.