Conference Day Two
Thursday, March 9, 2023

8:20 am Morning Refreshments

9:20 am Chair’s Opening Remarks

Creating Confidence Through Delivery Analytics

9:30 am Panel Discussion:Adapting Assays Demonstrating Targeted Intracellular Delivery

Synopsis

  • How can available assays help understand particle behaviour?
  • What technologies and imaging techniques can be applied if conventional assays aren’t providing biodistribution information?
  • Routes industry and academia are deploying to validate formulation expressions intracellularly

Achieving Endosomal Escape & Evading Endosomal Degradation

10:15 am RNA Medicines: Chemical Evolution of Synthetic Carriers

  • Ernst Wagner Professor, Ludwig- Maximilians- Universität München

Synopsis

  • Precise carriers made of short sequences of artificial amino acids optimized by evolution cycles
  • Highly effective cell entry and endosomal escape triggers fast and potent (>80%) siRNA, mRNA, Cas9/sgRNA and RNA LNP transfer at very low (~ 5-50 ng) dosage
  • Highly efficient transfer (1μg mRNA/mouse) into tumor, spleen, muscle, liver, lung by mRNA polyplexes or LNPs

10:45 am Morning Break

Synopsis

The perfect opportunity for face-to-face time with key opinion leaders ahead of the meeting’s close. Establish meaningful business relationships to build upon for 2023 and gain individual insight into the pioneering work ongoing.

11:45 am Acid Degradable Lipid Nanoparticles Deliver mRNA to Multiple Tissues In Vivo

  • Niren Murthy Professor, Department of Bioengineering, University of California, Berkeley

Synopsis

  • New acid degradable linkages
  • Lipid nanoparticles that rapidly degrade within cells
  • Transfection of non-liver organs

12:15 pm Roundtable Discussion: What’s Next on the Horizon? Disease Model Indicators for a Targeted Intracellular Delivery Approach

Synopsis

  • What disease indications could most benefit from intracellular drug delivery investment?
  • Will specific payloads / technologies witness accelerated progress over the next 12 months?
  • Utilize this unique industry touchpoint to discuss and gain and understanding of where intracellular targeted delivery platform efforts should be diverted to support novel pipeline progression and research collaborations

1:15 pm Networking Lunch

Synopsis

Remain connected with your Targeted Intracellular Delivery community to stay ahead of the curve on the technological advances that could take your drugs towards clinical development & beyond.

If you are new to the space and need advice, get in touch here and we can facilitate the most meaningful conversations for your work.

2:30 pm Shuttle Peptides Enable Delivery of Proteins, PMOs and CRISPRAssociated Nucleases to Airway Epithelial Cells

  • David Guay Vp Innovation and Technology, Feldan Therapeutics

Synopsis

  • Development and optimization of the shuttle peptides that enable rapid delivery of small molecules, PMOs, and proteins to cells without endosomal entrapment
  • Shuttle peptide optimization for delivering CRISPR-associated nuclease and base editor ribonucleoproteins
  • Delivery to mouse, pig and monkey airway epithelial cells in vivo

3:00 pm Informing Design by Characterizing Endo/Lysosomal Maturation, Payload Escape, and Autophagosomal Capture during Nucleic Acid Delivery

  • Sean Bedingfield Research Engineer, Lilly Genetic Medicine, Eli Lilly & Company

Synopsis

  • What in vitro methods have been explored for understanding endosomal escape?
  • What methodologies are available to monitor the siRNA in the body?
  • What do we understand about different platforms and methods of cytosolic delivery?

3:30 pm Chair’s Closing Remarks & End of Conference