- Current models available for retinal research and drug development are limited by poor recapitulation of human retinal physiology as seen in various animal models; and cell models lacking the complexity of retinal structure and function.
- Advancements in human-specific therapeutic modality candidates further drives a need for human relevant in vitro models. Induced pluripotent stem cell (iPSC) – derived retinal organoids mimic early retinogenesis resulting in a laminated retinal structure containing major retinal cell types organised in multiple nuclear and synaptic layers. We developed robust production approaches to routinely and reproducibly generate retinal organoids at scale to support early safety and efficacy testing of drug candidates.
- Controlling key parameters that affect variability and defining quality control measures allows us providing a retinal organoid platform that can provide predictive data. We show two examples where retinal organoids can be used in early drug discovery: for predicting retinal toxicity and for testing novel gene therapy vectors. We also describe further enhancement of the model by incorporating immune cells. The development and validation of the retinal organoid model will provide the missing link between compound screening and clinical trials and serve as a model for testing the efficacy and toxicity of drugs.
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