8:30 am Morning Coffee & Light Breakfast
8:50 am Chairs Opening Remarks
Deep Diving into Delivery Platforms for RNA-Based Vaccines & Gene Therapy Applications for Accelerated Translation Through the Clinic & Towards Patients in Need
9:00 am Discovery of Novel Ionizable Lipids for the Intramuscular Delivery of mRNA Vaccines
Synopsis
- Providing an overview on LNP-based mRNA delivery system for the development of mRNA vaccine platform
- Design and synthesis of novel ionizable lipids for the efficient delivery of mRNA vaccines
- Focusing on building Structure Activity Relationship (SAR) of novel ionizable lipids with improved immunogenicity and reduced reactogenicity towards the development of effective mRNA vaccines
9:30 am LNP & Polymer Delivery of Self-Replicating RNA: Unlocking the Potential of Low Dose RNA Vaccines & Gene Therapy
Synopsis
- LNP delivery of self-replicating RNA rabies vaccine elicits protective immunity in a majority of healthy participants down to 0.1 microgram (mcg) doses in a prime-boost schedule
- There were no severe adverse events reported across all doses tested (0.1, 1 and 10 mcg), which supports a massive increase in the therapeutic index achievable with RNA technology in humans
- Revealing preliminary data on polymer delivery of srRNA for gene therapy applications will be presented
10:00 am In Vivo Genome Editing: Translating Science from Bench to Bedside
Synopsis
- Describing our expertise in metagenomics discovery and protein engineering with the help of High-Throughput Evolution Platform for Discovery and Optimization of Novel Editors (HEPDONE) system
- Introduce our proprietary LNP mediated delivery systems targeting both liver and HSCs in the bone marrow
- Recent clinical trial readouts from our multiple ongoing in vivo genome editing programs in China will be discussed
10:30 am Morning Networking Break
Illuminating Conjugated Delivery Platforms for Targeted Delivery of RNA Therapeutics to the Liver & Beyond to Enable Cell Selectivity
11:30 am Unlocking the Full Potential of RNAi with LEADTM (Ligand & Enhancer Assisted Delivery)
Synopsis
- Delivery is the biggest hurdle to harnessing RNAi potential as a new drug modality
- GalNAc conjugate technologies have revolutionized RNAi for liver targets. Extrahepatic delivery is the new frontier for RNA
- Sanegene’s LEAD™ technology focusing on conjugate approach effectively delivers RNAi to specific tissues or cells of therapeutic relevance
12:00 pm Deep Learning-Based Design of Cell-Targeting Ligands Enables Cell- Specific, Functional SiRNA Delivery in Multiple Extrahepatic Tissues
Synopsis
- Receptor-mediated endocytosis drives cell tropism across RNA modalities
- Deep learning-based design of ligands targeting endocytosing receptors enables rapid in vivo testing of receptors for their capacity to mediate functional siRNA delivery
- This platform – Connexa – has led to the discovery of multiple receptors capable of mediating in vivo functional siRNA delivery in extrahepatic tissues
12:30 pm Mergo®: a Scalable & Programmable Conjugation-Based Delivery System for Short Oligonucleotides
Synopsis
- Sixfold is overcoming the RNA delivery challenge by systematically screening a large nd diverse conjugate space
- The unique Mergo® system uses modified RNA tags that allow rapid in vivo evaluation of novel modifications that alter biodistribution and cell uptake, and mimic how RNA is naturally shuttled between cells
- Sixfold will further present how the novel unbiased in vivo screening approach they have developed supports computational-based conjugate optimization
1:00 pm Lunch & Networking
Examining Novel Routes of Administration for More Selective Delivery to Treat Diseases Outside of the Liver
2:00 pm Roundtable Discussion – Taking Advantage of Administration Routes for Enabling Successful Extra Hepatic Delivery to Widen the Landscape of Treatable Targets
Synopsis
- Systemic delivery has only been achieved with lipid nanoparticles so far, what learnings can be taken from this and incorporated into new delivery platforms?
- How is targeted delivery being achieved through direct delivery, such as inhalation for lung targets and direct injections for brain targets?
- Discussing how to alter the physical characteristics of the delivery vehicle to ensure success via the chosen administration route
3:00 pm Afternoon Break & Networking
Overcoming the Challenge of Endosomal Escape by Employment of Enhancer Compounds to Improve RNA Payload Delivery
4:00 pm Improving Targeted Endosomal Escape through Sapreme’s Endosomal Escape Enhancer Platform for More Potent & Durable Therapeutics
Synopsis
- Harnessing developable and conjugatable compounds to improve delivery of different RNA payloads through improved endosomal escape
- Showcasing preclinical proof of concept for enhancing endosomal escape through this unique enhancer platform for more potent therapeutics
- Demonstrating how to conjugate this enhancer platform to targeting ligands for extra
- hepatic delivery
4:30 pm Stabilization & Enzymatically Gated mRNA Expression Using Nucleic Acid Nanocapsules for Enhancing Endosomal Escape
Synopsis
- Describing a novel formulation that enables room temperature stabilization of mRNA
- Showcasing how this formulation can target expression of mRNA both in vitro and in vivo using enzyme gated release mechanisms
- Leveraging how this formulation can be paired with aptamers to enable receptor targeted delivery of mRNA and enhance endosomal escape