Engineering for Safety and Efficacy in Oligonucleotide-Based Medicines (OBMs)
Time: 1:30 pm
day: Conference Day One
Details:
- Smart data versus big data: implementing novel design of experiment principles to generate comprehensive and highly informative in vivo pharmacological datasets, tailored to fuel a machine learning (ML) model to engineer safety, specificity, and efficacy
- Precision engineering: employing both quantum computational chemistry and in vivo data to accurately modulate pharmacological effects with minimal chemical alterations and within genetic sequence constraints
- Safety-first approach: utilizing large-scale transcriptomics and human ex vivo and in vitro assays, coupled with rodent in vivo assays, to optimize the safety of OBMs, while also reducing both development time and cost
