Director QurAlis Corporation Cambridge, Massachusetts, United States
Oligonucleotides and mRNA therapeutics have advanced over the past 20 years, as well as the number of drugs approved. With the advancement of this field, there has been an increase in novel targets and delivery mechanisms. While the field has advanced in targeted knockdown, the challenges have also increased such as local tolerability and off-target toxicity. This symposium will focus on these advancements and strategies for different modalities, including GalNAc siRNAs, ASOs, mRNA vaccines. Each talk will focus on the development strategy including type of studies performed, dose selection and the use of surrogate molecules if appropriate for an IND-enabling package and beyond.