A new paradigm in translational research: Human-derived retinal organoids and retina-on-chip models driving gene therapy into clinical trials
Kevin Achberger (University of Tübingen), Alex M. Bailey (Tern Therapeutics) & Kwi Hye Kim (REGENXBIO Inc.)
Recent advances in human-relevant in vitro models are reshaping translational research by enabling more predictive approaches to therapeutic development. In this webinar, Dr. Kevin Achberger (Eberhard Karls University Tübingen), Dr. Alex M. Bailey (Tern Therapeutics) and Dr. Kwi Hye Kim (REGENXBIO Inc.) will present a platform that integrates hiPSC-derived retinal organoids, retinal pigment epithelial cells, and retina-on-chip technology to model complex retinal diseases and support gene therapy development.
Using this approach, researchers recapitulated key features of neuronal ceroid lipofuscinosis type 2 (CLN2), a rare neurodegenerative disorder caused by TPP1 mutations and associated with progressive vision loss. The model enabled evaluation of an AAV-based gene therapy, demonstrating restoration of TPP1 expression and reduction of disease-associated pathology in human retinal tissue.
Importantly, these findings supported the approval of a clinical trial in the UK for children with CLN2-related vision loss—without prior efficacy testing in animal models, which do not adequately reflect the human disease. This work highlights a shift in translational research, showing that integrated organoid and organ-on-chip systems can advance gene therapy into clinical trials based on human-relevant data.
The webinar will discuss the development and application of this platform and its broader implications for improving predictive power and accelerating the path from research to clinical evaluation.
