1.30 Demonstrating Broad Brain Distribution, Target Engagement & Durability with Di-siRNAs after Intrathecal Administrations in NHPs – Focus on Huntington’s Disease
Time: 1:30 pm
day: Day Two Track 2 PM
Details:
- Demonstrating how broad distribution results in potent target downregulation throughout the brain, sustained over 6 months
- Showing how RISC-loading assessments support long duration of gene silencing in deep brain regions.
- Leveraging microscopy-based and Mesoscale Discovery assays to evaluate robust distribution in the brain Highlighting how broad, potent, and long-term downregulation of Huntingtin is safe and well-tolerated in NHP
Bruno Godinho, Senior Scientist, Atalanta Therapeutics
