Workshop E: Evaluating Toxicity in Preclinical Models: Reviewing Distribution & Safety of Delivery Methodology to Produce More Effective & Tolerable Therapeutics
Time: 3:00 pm
day: Pre-Conference Workshop Day Track E
Details:
This workshop provides an in-depth exploration of key considerations in intrathecal and conjugate delivery techniques, aiming to achieve optimal distribution and penetrance while addressing factors such as affinity, efficacy, and specificity for efficient oligonucleotide delivery to the brain.
Participants will gain insights into:
- Quantifying and mitigating acute neurotoxicity induced by ASOs and siRNAs in the central nervous system
- Maximizing therapeutic impact by assessing dose frequency, optimizing distribution profiles, and enhancing efficacy while minimizing drug toxicity
- Understanding the importance of even distribution and penetrance in intrathecal delivery
- Balancing affinity, efficacy, and specificity to optimize delivery efficiency
- Determining preclinical efficacy, safety, and PK/PD correlation to prioritize ASOs and siRNAs of interest
- Exploring innovative approaches and technologies for improving intrathecal delivery outcomes
