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 approach to 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 transformed into stimulation patterns via specialized camera-equipped glasses. Despite the potential for substantial improvements in vision restoration, optogenetic approaches face significant 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 new 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 associated specialized stimulation devices for human patients. Through rigorous preclinical research on mouse and primate models, including high-density multi-electrode recordings and functional ultrasound imaging, the center evaluates the performance of these new optogenetic tools. This foundational work aims to pave the way for introducing a new standard of treatment for patients suffering from debilitating vision loss. Clinical trials are planned to be conducted by 2030, opening new possibilities.