Physiologically relevant iPSC-derived retinal models for ophthalmology research & drug discovery

Retinal diseases are a diverse group of degenerative and genetically driven conditions that progressively impair visual function. While recent advances have improved outcomes in some indications, most retinal disorders, including dry age‑related macular degeneration and inherited retinal diseases, still lack effective disease‑modifying treatments, reinforcing the need for physiologically relevant human model systems.

Our new whitepaper describes how human iPSC‑derived retinal models enable mechanistic studies, toxicity assessment, and improved translational relevance in ophthalmology research.

In this whitepaper, we discuss:

• Key challenges in retinal disease modeling and the limitations of traditional in vivo and in vitro systems

• Phenotypic and functional characterisation of human iPSC‑derived retinal pigment epithelium and microglia relevant to retinal degeneration and inflammation

• The use of iPSC‑derived retinal organoids to recapitulate retinal development, cellular composition, and layered architecture

• Application of physiologically relevant RPE‑based in vitro models to study dry AMD‑associated stressors, including oxidative stress, complement activation, and inflammatory signalling

• Emerging directions toward multi‑lineage and higher‑order retinal model systems to better capture human‑specific disease mechanisms

As ophthalmology moves toward gene‑targeted, cell‑based, and pathway‑specific therapies, well‑characterised iPSC‑derived retinal models are becoming central to translational research and drug discovery.