Optimization of an Optogenetic-Based Vision Therapy for Retinitis Pigmentosa

DESCRIPTION (provided by applicant): Eos Neuroscience, Inc., in collaboration with its academic partners, is developing a novel technology combining gene therapeutics and optical engineering techniques to restore functional vision to individuals that are blind from photoreceptor related diseases such as Retinitis Pigmentosa or Age-Related Macular Degeneration. We believe that this technology will be widely applicable to the general public, in the United States and worldwide, which suffers from these debilitating and blinding diseases. In brief, Eos Neuroscience, Inc. is creating a technology that will restore photosensitivity in the remaining cells of the retina after the photoreceptors have died or are no longer functional, thus restoring the ability of theretina to respond to light stimulation. To this end, we have created a delivery mechanism using an adeno-associated virus (AAV) that is proven safe and effective at delivering genes into cells. We will use this mechanism to express light-sensitive proteins into the spared, functioning cells of the retina. Additionally, we are completing the remaining required safety and efficacy studies necessary to gain preclinical readiness. In the course of our Phase I funding/operating period, we completed studies toevaluate both physiological and behavioral efficacy measures in murine models of retinal degeneration. Here, we will attempt to further improve our therapeutic vector by employing more sensitive opsins and evaluate the efficacy of these opsins through physiological and behavioral measures. PUBLIC HEALTH RELEVANCE: Photoreceptor diseases such as retinitis pigmentosa (RP) and age-related macular degeneration (AMD) are leading causes of blindness, affecting approximately 15 million people worldwide. Current therapies are targeting single genetic defects or are using electrical stimulation to restore visual function - neither of which is capabl of being a treatment that can be applied broadly to photoreceptor disease. To this end, Eos Neuroscience, Inc.has established a technology using genetically expressed light sensitive proteins to restore light sensitivity in patients suffering from photoreceptor degeneration, a technology that can be applied broadly and accurately for the treatment of these diseases.