NIO Neurotech Summit
Translating a Clinically-Viable Therapy for Blindness Using Light-Sensitive Proteins
Implantable electrical devices (e.g., microelectronic retinal prostheses) can be used to stimulate neural tissue but are complicated by the broad and indiscriminant spread of activation. To drive retinal circuits more naturalistically, optical neural modulators (e.g., channelrhodopsin-2) can be genetically targeted to specific cell types, allowing for precise activation using light stimulation. Eos Neuroscience, Inc. has spent the last 3 years translating these light-sensitive proteins into a clinically-viable therapy for treating blindness due to photoreceptor disease.
Using a specific regulatory sequence in combination with a novel adeno-associated virus (AAV), we targeted expression of ChR2 to a specific set of retinal cells and subsequently restore light sensitivity and visual function in 3 genetically different mouse models of blindness. We found robust expression of ChR2 in the ON bipolar cells (our cellular target) and measurements of visually-guided behavior convincingly shows that treated mice, with no existing rods or cones, have restored visual function. Finally, we evaluated the safety of our therapy by studying the biodistribution, ocular toxicology, systemic immune response, and the potential light toxicity of chronic high-intensity light stimulation. Our findings suggest that our ChR2-based therapy is safe and does not provoke an inflammatory or immune response. Taken together, these data provide a strong foundation for a clinically-viable treatment for blindness. We continue to optimize our therapy, which includes improving sensitivity and evaluating efficacy in nonhuman primates, as well as aggressively moving towards preclinical and clinical testing.
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