Roundtable Discussion: Achieving Safe, Deep Brain Penetration – Can Intrathecal & Systematic Delivery Reach the Regions That Matter?
- Evaluating whether IT delivery can achieve significant exposure in deep brain structures implicated in Huntington’s and Parkinson’s disease
- Examining emerging chemistry-driven distribution strategies such as C16-siRNA conjugates and modified ASOs, and interrogating non-human primate data to understand current depth and dose limitations
- Comparing IT optimization strategies against systemic BBB shuttle approaches to determine which modality is better suited for specific diseases requiring deep brain penetration