8:00 am Check-In & Light Breakfast
8:50 am Chair’s Opening Remarks
The Future of RNA Delivery: Illuminating Next Generation Non-Viral Delivery Vehicles Beyond LNPs for Greater Biological & Commercial Success
9:00 am Expert Panel Discussion – De-Risking RNA Delivery Platforms Through Uncovering the Most Suitable Platforms for Your RNA Payloads to Ensure Effective Treatment
Synopsis
- With an array of nonviral delivery carriers in development, how do you identify which is the most suitable method for your RNA therapeutic?
- How can the field leverage and learn from approved carriers, such as LNPs, to overcome 2024 IP complexities?
- When developing a novel delivery vehicle, how is the field de-risking their platforms to ensure a commercial success given the crowded IP landscape?
10:00 am RNABL: A Polyester Dendrimer-Based Platform for Therapeutic RNA Delivery
Synopsis
- RNABL dendrimers are a class of molecularly defined highly branched biodegradable molecules with lower reactogenicity and better safety characteristics
- RNABL nanoparticles displayed improved characteristics compared to other non-viral nucleic acid delivery platforms tested
- Structural modifications of RNABL delivery materials resulted in organ-specific nucleic acid delivery (e.g. lungs, spleen, heart)
10:30 am Morning Break & Speed Networking
Synopsis
This networking session is your opportunity to get face-to-face with many of the brightest minds working in the non-viral RNA delivery systems field and establish meaningful business relationships to pursue for the rest of the conference.
11:30 am StealthX, a Non-Viral Exosome Delivery Platform for Future RNA Therapeutics
Synopsis
- Showcasing exosome production, characterization, and manufacturing for successful reproducibility of exosome-based delivery vehicles
- Uncovering the potential of exosomes to deliver RNA-based therapeutics effectively to the target of interest
- Targeted delivery of ASO to skeleton muscle by exosome carrying a targeting moiety
12:00 pm ARMMs: A Non-Viral Delivery Platform for RNA Delivery with Engineerable Tropism
Synopsis
- Introducing ARRDC1-mediated microvesicle (ARMM) biology and how it can be coopted to generate a non-viral delivery platform for macromolecular therapeutics with excellent prospects for redosability and safety
- Demonstrating how modification of ARRDC1 allows loading of therapeutic RNAs into ARMMs for cellular delivery
- Showcasing how engineering enables alteration of ARMMs surface proteins and fusogens to direct payloads to cells and tissues of therapeutic interest
12:30 pm Unlocking Novel Peptide Delivery Platforms for More Selective RNA Delivery to Cells Outside the Liver
Synopsis
- Rational design of a peptide-based delivery system to overcome hepatic accumulations and to selectively target diseased tissues
- Unlocking new RNA-based therapeutic platform that leverages novel peptide technology capable of targeting and systemically delivering therapeutic RNA and gene editing machinery into the pathological cell to combat or prevent disease
- Demonstrating efficient mRNA delivery through systemic administration to rescue tumor suppressor functions as a potential therapeutic approach in cancers
1:00 pm Lunch & Networking
Unleashing Novel LNP Chemistry & Characterization to Optimize Formulation & Reduce Toxicity for Ensuring Safe RNA Therapeutic Delivery
2:00 pm Quantitative Nanoparticle Characterization with Flow NanoAnalyzer: Understanding Your Particles & Payload
Synopsis
- Learn how the unique features of the Flow NanoAnalyzer enable it to detect and chatacterize single nanoparticles and quanitify their payloads
- Understand how scientists are using NanoFCM methods and data to optimize formulation and processing conditions to create safe and effective new medicines
2:15 pm The Development of scLNP for Extrahepatic RNA Delivery
Synopsis
- Showcasing an overview of Corti’s scLNP platform
- Highlighting a case study example of scLNP enabled selective delivery of RNA to disease relevant cells
- Illustrating the immunological considerations of non-viral delivery system and the feature of scLNPs
2:45 pm In Vivo Delivery of RNA by Targeted Lipid Nanoparticles (tLNP) to Hematopoietic Stem Cells in Humanized Mice & Non-Human Primates
Synopsis
- Showcasing strategies for in vivo RNA extrahepatic delivery
- Uncovering strategies to conjugate targeting ligands to LNPs to enable targeted and specific delivery
- Outlining LNP compositions to reduce toxicity and enable redosability
3:15 pm Afternoon Networking Break & Scientific Poster Session
Synopsis
Contribute to the conversation and share your cutting-edge research with your fellow RNA community to showcase your breakthrough discoveries to a vast audience of experts. Register your place to submit an abstract for review to showcase your poster*
*Please visit the website for the T&Cs for presenting a poster
Achieving Extra Hepatic Delivery Through Novel & Innovative Platform Designs Enabling Treatment of a Wider Landscape of Diseases
3:45 pm Lipid Nanoparticle-Mediated Oral Nucleic Acid Delivery
Synopsis
Details to be revealed
4:15 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
4:45 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