Conference Day One- November 15, 2023
7:50 am Registration Open & Networking Coffee
8:50 am Chair’s Opening Remarks
Leveraging Physical & Chemical Properties of Non-Viral Delivery Systems for Improved Loading Capacity, Stability & Efficacy
9:00 am Optimizing the Complexing of the Payload & Delivery System to Form Stable & Efficacious Nanoparticles
Synopsis
- Leveraging properties such as surface charge on delivery system and payload to facilitate complexing and increase loading capacity
- Developing methods to better measure the amount and position of RNA in your delivery systems to create effective nanoparticles
- Overcoming the challenges with multi-complexing your delivery system with multiple payloads such as, DNA and RNA for effective delivery of next generation therapeutics
9:30 am Correlating Physical & Chemical Properties of Non-Viral Delivery Systems to Functional Parameters for Higher Efficacy
Synopsis
- Accurately characterizing the chemical and physical properties of your RNA non-viral delivery system to co-relate them to functional properties
- Manipulating properties such as surface charge, composition, and size to affect efficacy, tropism, and internalization of your delivery system
- Comparing structure-function relationships of different systems to optimize your unique delivery platform for higher efficacy
10:00 am Using Single Nanoparticle Analysis to Understand the Payload Distribution within a Formulation
Synopsis
- Learn how NanoFCMs single particle nanoflow cytometry methods are revealing new details about the distribution and position of payloads at the single particle level beyond particle size and count
- See how to use simple, inuititive methods to understand a wealth of new information for multiple cargo types (nucleic acid, proteins, small molecules)
- Hear about how users are optimizing their formulations to produce particles with particular loading characteristics
10:15 am Morning Break & Speed Networking
Synopsis
Our structured networking is the ideal opportunity to get face-to-face time with many of the brightest minds working with non-viral delivery of RNA and introduce yourself to the attendees that you would like to have more in-depth conversations with. Benchmark against industry leaders and establish meaningful business relationships to pursue for the rest of the conference and beyond
Hitting the Bullseye: Increasing Cell Specificity of Non-Viral Delivery Systems for Targeted Delivery of RNA
11:15 am Developing Non-Viral, Cell Targeting Vehicles to Increase Targeted Delivery of Therapeutic RNA
Synopsis
- Discuss how we can realize the therapeutic opportunity for cell-specific delivery of therapeutic RNA
- Reveal recent data on pre-clinical and clinical therapeutics incorporating cell targeting vehicles to promote targeted delivery of RNA
- Showcase a novel platform for the development of next-generation cell targeting vehicles to inform the development of your own delivery platforms
11:45 am Sustained delivery of RNA through VitalDose® EVA
Synopsis
- The use of an ethylene vinyl acetate implant for RNA delivery
- Formulation strategies for the release and stability of ASOs
11:55 am Targeting KRAS through a Novel, Molecular siRNA Delivery System to Target Lung, Pancreas & Cardiac Tissue
Synopsis
- Modification of the intrinsic properties of therapeutic oligonucleotides by covalent lipid-based delivery molecules
- Data from PDX models for the development of PRAMO’s drug pipeline
- Revealing novel data and insights from the development of PRAMO’s unique delivery platform to inform the creation and optimization of your own platform
12:25 pm Networking Lunch
De-Livering RNA Delivery: Engineering Non-Viral Systems with Extra-Hepatic Tropism to Target Indications Beyond the Liver
1:25 pm Panel Discussion: Exploring the Strategies to Induce Extra-Hepatic Delivery & Reduce Accumulation in Liver
Synopsis
One of the biggest challenges for delivery of RNA therapeutics has been the tendency of nanoparticles to accumulate in the liver when delivered intravenously due to the body’s natural filtration system. Changing the method of delivery (direct or subcutaneous injection) and properties such as size and surface charge has been observed to affect the accumulation of nanoparticles as well. This presents an avenue to manipulate tropism and target extra-hepatic tissues.
Join this panel to hear first-hand on:
- How can you accelerate the characterization and manipulation of nanoparticle properties that affect tropism and targeted delivery?
- How can you develop new methods for extra-hepatic delivery and increase the on:off target ratio of RNA therapeutics?
- How to capitalize on the future directions of non-viral delivery systems and overcome unmet needs such as crossing the blood brain barrier for RNA delivery to the brain
2:10 pm Showcasing Tiba’s RNABL Platform: A Novel Branched Aminolipid- Based System to Deliver RNA Beyond the Liver
Synopsis
- Tweaking formulation strategies to affect potency and tropism of the dendrimer system
- Revealing pre-clinical data on Tiba’s novel RNA delivery system evidencing extra-hepatic delivery
- Applying different classes of Tiba’s dendrimer nanoparticles to both vaccine and therapeutic modalities
2:40 pm Showcasing a Novel Iron Oxide-Based Non-Viral Delivery System to Deliver RNA Payloads to Extra-Hepatic Tissue
Synopsis
- Leveraging the chemical and structural properties and method of formulation of the TTX system to affect its tropism to extra-hepatic tissue
- Comparing the various ligands and targeting moieties under investigation for extra-hepatic cell-specific delivery of the TTX system
- Revealing novel data and insights from the development of the TTX platform to enable strategic optimization and differentiation of pipelines
3:10 pm Afternoon Networking
Tracking the Trafficking: Optmizing Cellular Uptake & Internalization to Maximize the Percentage of Payload Delivered to the Cell
4:00 pm Escaping the Endo-maze: Optimizing Your Nucleic Acid Micelle-Based Non-Viral Delivery System to Promote Endosomal Escape
Synopsis
- Grasping mechanisms of endosomal escape to promote payload release
- Shining a light on the nanoparticle properties that affect endosomal escape such as surface charge, size and monomer composition to promote endosomal escape
- Utilizing insights gained to design targeting moieties such as ligands to target the correct receptors for effective delivery
4:30 pm Manipulating Cellular Uptake, Internalization & Endosomal Escape Mechanics to Enhance the Delivery of RNA to Desired Tissues
Synopsis
- Mastering endosomal escape mechanisms for optimal release of payload in your chosen application and tissue
- Leveraging existing delivery systems as case studies to achieve successful cellular uptake, internalization and endosomal escape
- Anticipating future advancements, challenges, and strategies for non-viral delivery systems to improve amount of payload delivered to cells
5:00 pm Chair’s Closing Remarks & End of Day One
