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
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
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
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
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?