With approvals in siRNA technology, more ASOs hurtling towards human trials in neurology, and exciting headlines in Alzheimer’s and ALS, the draw of CNS targets for oligonucleotides has never been greater. Massive investments and growing pipelines in pharma and biotech alike are working to make these life-changing therapies a reality for previously undruggable targets.
Tackle genomic target validation, backbone chemistry, effective intracellular delivery, and potency, increased stability for reduced dosing, novel cerebral organoids, CNS device delivery, and CSF oligo distribution to uncover the best approaches for each phase in your pipeline. Take away invaluable lessons that will translate directly into optimizing your oligonucleotide pipeline for the CNS.
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