Understanding Toxicological Liabilities of Intrathecal Oligos to Enable Safer CNS Delivery
1:30 pm - Thursday 3rd December 2026
- Characterization of common CNS and off-target toxicological liabilities associated with intrathecal oligonucleotide delivery across preclinical models
- Application of in vitro assay systems to predict and mechanistically understand CNS toxicity and support translational risk assessment
- Strategies to differentiate sequence-specific versus platform-related toxicology signals when evaluating oligonucleotide candidates
- Integration of toxicology data into candidate selection frameworks to improve safety margins and de-risk direct CNS delivery approaches
