Roundtable Discussion: Predicting & Mitigating Long-Term CNS Neurotoxicity: Leveraging Predictive Assays to Optimise Oligonucleotide Safety
- Discover mechanistic drivers of neurotoxicity by moving beyond simple correlations with sequence motifs and using advanced assays such as calcium oscillation and multielectrode array (MEA) systems to understand potential longterm CNS safety risks
- Evaluating these predictive neurotoxic models and advances necessary before the first human trials
- Explore safer oligonucleotide design strategies by identifying potentially neurotoxic sequences early, optimising backbone and end modifications, and prioritising chemistries before in vivo testing