In April 2024, the Else Kröner Fresenius Foundation approved and funded a proposal to establish a new interdisciplinary Else Kröner Fresenius Center for Optogenetic Therapies (EKFZ). The goal of this center is to harness the great potential of optogenetics – the control of cells with light via light-sensitive proteins (opsins) – for clinical use. Optogenetics enables spatially confined, cell-type-specific control of organs and promises a transformative improvement of clinical care beyond that provided by conventional electrical medical devices for a wide range of patients.

Optogenetic therapies combine gene therapy with optical medical devices. They offer broader applicability than traditional gene therapy and promise greater efficacy, closer to restoring natural function than current medical devices, albeit with increased regulatory challenges. The EKFZ aims to develop and translate four therapies that demonstrate a compelling balance of anticipated benefits and manageable risks. Building on the tools and expertise provided by five EKFZ platforms as well as support structures, the teams will develop and translate optogenetic approaches for cochlear implants, retinal stimulation, gastric pacemaking, and brain computer interfaces for patients suffering from deafness, blindness, gastroparesis, and motor deficits. The EKFZ platforms will address common needs for optimized opsins, efficient, specific, and safe viral gene transfer for stable opsin expression, experimental and computational disease modeling, efficient immunomodulation and deep phenotyping, as well as power-efficient, long-term stable, and safe optical medical devices. We will involve patients as well as patient organizations from the start to learn about their perspectives, needs, and concerns, inform them on emerging therapies, and recruit them for clinical trials. The scope of the EKFZ ranges from fundamental strategies of optogenetic therapies, to comprehensive preclinical studies and early clinical trials. Supported by the EKFZ regulatory and advisory support team, the clinical trial center, and the early clinical trial unit, we plan to launch clinical trials on optogenetic restoration of hearing and vision within the funding period. Through the EKFZ academy, we are dedicated to delivering excellent training focused on clinical translation to students and early career researchers from medicine, natural science, and engineering. The EKFZ will communicate the findings to the scientific, biotech, and medtech communities as well as to the general public through a broad spectrum of engaging activities. We expect the EKFZ to become an internationally leading center for developing and translating optogenetics into innovative therapies that address unmet medical needs.