hiPSC-Based Functional Screening for CNS RNA Therapeutics

This poster presents Ncardia’s human iPSC-derived neuronal platform for functional screening of antisense oligonucleotides (ASOs), combining efficacy assessment with high-throughput evaluation of potential CNS safety liabilities.

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• How human iPSC-derived neurons and astrocytes can be integrated into an automated ASO screening workflow spanning cell culture, treatment, RNA extraction and RT-qPCR 

• How a robust RT-qPCR assay can assess ASO efficacy and target knockdown, with inter-plate variability below 5% in the reported assay setup

• How calcium imaging can detect dose-dependent changes in spontaneous neuronal activity and distinguish between functionally tolerated and neurotoxic ASOs

• How multi-electrode array (MEA) recordings capture ASO-induced changes in firing activity, burst frequency and neuronal network behaviour

• How functional responses across a panel of 70 preclinical ASO candidates can support translational interpretation of potential CNS safety liabilities