Degenerative retinal diseases such as retinitis pigmentosa (RP) and geographic atrophy (GA) affect approximately 10 million people worldwide, leading to significant and often irreversible vision loss. Optogenetic therapies have emerged as a promising avenue for treating these diseases by utilizing light-sensitive proteins (opsins) to confer light sensitivity to surviving retinal cells, such as retinal ganglion cells (RGCs). In these therapies, visual inputs are converted into stimulation patterns via specialized camera-equipped glasses. Despite the potential for significant improvements in vision restoration, optogenetic approaches face considerable challenges, such as achieving sufficient resolution and light sensitivity.
In response to these challenges, the EKFZ Center for Optogenetic Therapies is pioneering the development of advanced optogenetic strategies, focusing on novel opsins like ChReef, which are characterized by high efficacy at lower light intensities, potentially expanding the dynamic range of visual perception while reducing the risk of phototoxicity. This initiative is part of a broader project to develop a gene therapy medicinal product (GTMP) and its associated specialized stimulation devices for human patients. Through rigorous preclinical research in mouse and primate models, including high-density multi-electrode recordings and functional ultrasound imaging, the center is evaluating the performance of these novel optogenetic tools. This foundational work aims to pave the way for the introduction of a new standard of care for patients suffering from debilitating vision loss. Clinical trials are planned by 2030, opening up new possibilities.