9:00 am Workshop A: Streamlining Preclinical Assessment from Dish, to Rat, to NHP for Improved Oligonucleotide Translation in the CNS
Time: 9:00 am
day: Pre-Conference Workshop Day - Track A P1
Details:
As oligonucleotide therapeutics advance in neurodegeneration and ophthalmology, optimizing in vitro and in vivo assays is critical to improving delivery, minimizing off-target effects, and ensuring translatability into clinical success. This workshop will explore best practices in assay development for RNA-based therapeutics, from high-throughput screening (HTS) strategies to NHP validation.
Join this session to:
- Refine in vitro assays to better demonstrate deep brain penetration to target cells in the human system
- Design relevant cellular models, use transcriptomics for off-target analysis, and ensure preclinical assays translate into clinical outcomes
- Compare what has worked for in vitro vs what will be used for in vivo
- Strategies for assessing unintended gene knockdowns, systemic distribution challenges, and immune response screening for off-target and toxicity profiling
- Lessons from recent oligonucleotide failures due to cytokine-driven inflammation and novel chemistries to mitigate immune activation
