Adeno-associated virus (AAV)–based drug development often requires early decisions about serotype and promoter design that can strongly influence downstream program success. Although rodent and other in vivo models are commonly used for preclinical AAV vector evaluation, these studies are throughput limited, lengthy, and most importantly, fail to correspond to human clinical results.

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In this application note, we show how 28bio CNS-3D Brain Organoids enable high-throughput assessment of AAV transduction, expression dynamics, functional impact, and viability in a single model. This iPSC-derived brain organoid technology with spontaneous neural activity supports earlier, more informed AAV vector prioritization ahead of in vivo testing enabling faster, more efficient gene therapy development.

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