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 mRNA LNP Process Science & Engineering: First Steps Towards a Novel Modality
Synopsis
Details to be revealed
11:00 am Morning Networking Break
Illuminating Conjugated Delivery Platforms for Targeted Delivery of RNA Therapeutics to the Liver & Beyond to Enable Cell Selectivity
12:00 pm 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
12:30 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
1:00 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
1:30 pm Lunch & Networking
Examining Novel Routes of Administration for More Selective Delivery to Treat Diseases Outside of the Liver
2:30 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
3:30 pm Afternoon Break & Networking
Achieving Extra Hepatic Delivery Through Novel & Innovative Platform Designs Enabling Treatment of a Wider Landscape of Diseases
4:00 pm Lipid Nanoparticle Mediated Oral Nucleic Acid Delivery to Enable Extra- Hepatic Delivery
Synopsis
• Need for Oral Delivery of Nucleic Acids
• Formulation approaches for LNPs for oral delivery
• Versatility of platform formulation that could be used for siRNA, miRNA and mRNA
4:30 pm Optimization of Fusogenix Proteolipid Vehicles for Extra-Hepatic Delivery of mRNA
Synopsis
• Using the Fusogenix PLV technology as a nucleic acid delivery system
• Delivery of different cargos with a focus on mRNA and decreased toxicity
• mRNA encapsulated PLV delivery targeted to extra-hepatic tissues such as lung, eye, and muscle
5:00 pm Leveraging Innovative Lipid Nanoparticle Strategies for Advancing Delivery of mRNA in Epigenomic Therapeutics
Synopsis
• Illustrating strategies for delivering to specific epigenomic targets supported by preclinical to clinical data
• Expanding mRNA delivery to pulmonary tissue through nebulization and inhalation
• Showcasing preclinical data demonstrating successful target engagement for treatment of respiratory diseases