Rapid fire 4: High-Throughput Drug Screening and Repurposing for a Rare Childhood Neurodevelopmental Disorder by Tamsin Baxter

11.45am – 12pm BST, 22 April 2026 ‐ 15 mins

Morning Session

This project developed a high-throughput dual-reporter screening platform to identify compounds that modulate expression of the translation elongation factor eEF1A2, mutations in which cause a rare neurodevelopmental disorder characterised by epilepsy, autism and developmental delay. Screening libraries of existing and novel compounds identified candidate modulators of eEF1A2 expression, which were subsequently validated using dose–response analysis and western blotting of neuronal cell models. This work establishes a scalable strategy for identifying potential disease-modifying therapies for eEF1A2-related disorders.