WORD+ 2025 Conference

Poster

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Clinically Related Rare Neurodevelopmental Disorders Display Distinct Functional Phenotypes in Human iPSC-derived Cortical Organoids

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Overview

• Rett syndrome (RTT) and CDKL5 deficiency disorder (CDD) are rare X-linked neurodevelopmental disorders that share overlapping phenotypic features including cognitive defects, developmental delays, and seizures.

• microBrainTM is a scalable cortical organoid platform that can be grown in high-throughput plate formats up to 384-wells.

• RTT and CDD organoids were used to screen over 5000 compounds and identify promising biological targets and molecules that corrected aberrant function through unique mechanisms of action.