Pre-Conference Workshop Day
Tuesday, June 21 | 9:00am - 4:00pm

9.00 - 12.00

Exploring Mechanisms of Cellular Uptake & Endosomal Escape for Target-Specific Delivery

Thanks to the advances in the field of non-viral RNA delivery systems, RNA therapeutics and vaccines are gaining momentum and opening doors to addressing some of the key challenges historically associated with targeting pathways and a broad range of diseases with RNA-based therapeutics.
However, a bottleneck remains in the poor understanding of the parameters that correlate with endosomal escape versus target-specific delivery.

This workshop aims to delve deep into:

  • Better understanding the mechanism of endosomal escape of the cargo that is effectively delivered to a target cell
  • Elucidating the role of cellular components during cellular uptake of nanocomplexes
  • How different cellular components influence target-specific delivery

Jeonghwan Kim

Postdoctoral Scholar

Oregon State University

Ramsey Majzoub

Senior Principal Scientist

Intellia Therapeutics

Siddarth Patel

Associate Director

Senda Biosciences

Tatiana Bronich


Northeastern University

1.00 - 4.00

CNS Delivery of RNA Therapeutics

Taking into consideration the pre-clinical advancements of RNA payloads, there is now a greater necessity to characterize ways to deliver RNA payloads safely and efficiently to the CNS and overcome the challenges of crossing the blood brain barrier. Combating the challenges associated with targeted and efficient delivery of non-viral RNA delivery systems to the CNS requires a deeper focus and will be explored in this workshop to provide solutions

This workshop aims to delve deep into:

  • Targeted delivery of RNA constructs to specific sites in the brain
  • Lipid- and polymeric-nanoparticle formulations
  • In vitro and in vivo studies in disease models
  • Novel nose to brain techniques for RNA therapeutics