Bionic Sight Doses First Patient in Clinical Trial for Optogenetic Therapy
Treatment combines gene therapy and a device that generates and delivers retinal code
The company Bionic Sight has dosed the first patient in a Phase 1/2 clinical trial for its optogenetic treatment for people with advanced vision loss from retinitis pigmentosa (RP). The company, which began the 20-participant trial in March 2020, plans to continue dosing patients in the second half of 2020. The study is taking place at Ophthalmic Consultants of Long Island.
Bionic Sight’s approach involves two components: 1) An optogenetic treatment that bestows light sensitivity to retinal ganglion cells which survive after photoreceptors are lost to an advanced retinal disease like RP, and 2) A device, worn like a pair of glasses, that captures the scene a person is looking at and generates vision-enabling code, which is sent through ganglion cells and optic nerve to the brain.
Unlike other emerging optogenetic approaches, which may use glasses or goggles to enhance the shape and intensity of the image, the Bionic Sight device produces electrical impulses, similar to those produced by photoreceptors. Bionic Sight believes that generation of more natural retinal code will lead to better vision for patients than other optogenetic alternatives.
The approach is designed to work for people with only bare light perception, regardless of the gene mutation causing their retinal disease.
The video above, titled “Cracking the Code to Beat Blindness” provides more details about Bionic Sight’s technology.
Sheila Nirenberg, PhD, a principal and founder at Bionic Sight and a professor at Weill Medical College of Cornell University, led development of the device which generates and delivers the electrical impulses which are sent to the optogenetically treated ganglion cells. She is a recipient of a MacArthur “Genius” Award for deciphering this neural code of the retina.
Applied Genetic Technologies Corporation (AGTC), a developer of gene therapies for retinal degenerative diseases and other conditions, developed the optogenetic gene therapy being used to make ganglion cells light-sensitive in the Bionic Sight clinical trial.
“While optogenetics for retinal disease has moved into clinical trials, the approach is still at an early stage of development. We are still learning its potential for restoring sight to those who have lost most or all of their vision to conditions like RP,” says Brian Mansfield, PhD, executive vice president for research and interim chief scientific officer at the Foundation Fighting Blindness. “Bionic Sight’s approach is both sophisticated and compelling. We are excited to learn how well it helps patients regain meaningful vision.”
The Foundation Fighting Blindness notified RP patients in its My Retina Tracker Registry about Bionic Sight’s clinical trial. More than 15,000 patients are currently in the registry, which was developed for people with inherited retinal diseases. The goal of the registry is to help researchers learn more about these rare retinal conditions and identify patients for clinical trials.