Retinitis pigmentosa and many other retinal conditions caused by genetic mutations, are characterized by progressive loss of retinal photoreceptors. Although many gene-specific AAV product candidates are being developed to treat several forms of inherited retinal conditions, these therapies cannot be used to treat retinas with no remaining photoreceptors. Optogenetics is a novel approach to treating these advanced forms of retinal degeneration. Through this approach, retinal ganglion nerve cells, instead of photoreceptors, are treated with an AAV vector to deliver a light-sensitive protein that enables these cells to respond to a light stimulus by transmitting a signal to the visual cortex of the brain.
AGTC is collaborating with Bionic Sight on the development of an optogenetic therapy designed to deliver a unique protein that can be activated with a light signal to the eye. This innovative approach combines AGTC’s expertise in gene delivery technology with a neuro-prosthetic device and algorithm based on deciphering the retinal code, developed by Dr. Sheila Nirenberg, founder of Bionic Sight.
Dr. Nirenberg’s advancements in retinal coding are designed to replace the function of photoreceptors, the cells in the retina that convert light as it enters the eye into electrical signals that are transmitted to the brain and form the images we see. In patients without functional photoreceptors, this signaling process is disrupted, causing blindness. Bionic Sight’s neuro-prosthetic device integrates a small camera that captures images, with a computer algorithm that converts the image into a pattern of light signals very similar to the electrical signals generated by functional photoreceptors. These signals are used to activate a light-sensitive protein that will be delivered to the eye through gene therapy. The activated protein transmits signals to the brain, allowing the visual image to be created.