Retinal organoids advance drug development for rare diseases

Retinal organoids are emerging as a key tool in developing therapies for rare retinal diseases. These models, created from patient cells, can mimic human retina tissue, allowing researchers to study specific genetic mutations and test treatments more effectively than traditional animal models. Recent advancements include successful proof-of-concept studies for therapies targeting conditions like Leber congenital amaurosis. While retinal organoids show promise, challenges remain in ensuring consistency and reproducibility across different batches, which researchers are addressing through strict quality control measures. The regulatory landscape is gradually shifting to accept organoids as part of the drug development process. Collaboration between researchers and regulatory bodies is essential to establish standardized protocols and validate these models for broader applications, potentially extending beyond rare diseases to more common retinal conditions.