Roundtable Discussion: Not All Modalities Are Created Equal: Overcoming Formulation Challenges in Intrathecal Delivery to Speed CMC & Improve Patient Outcomes
Time: 11:40 am
day: Day One - Morning - Track B
Details:
- How do different modalities uniquely challenge formulation for safe and effective intrathecal delivery?
- What formulation strategies – buffers, excipients, conjugates – are proving most effective or problematic for IT delivery of ASOs and siRNAs?
- What are regulators looking for in safe and scalable IT formulations, and how can we align preclinical testing with clinical expectations?
- What are the risks of bolus dosing on CSF composition, and how can formulation mitigate neurotoxicity across species?
