Next Generation mRNA Delivery Day
Monday, July 29
Regulatory Affairs Strategy Day
Monday, July 29
8:00 am Morning Check In & Coffee
8:50 am Chair’s Opening Remarks
Advancing Novel Lipid Discovery to Improve Nanoparticle Composition for Safe Systemic Delivery of LNP-mRNA
9:00 am Overcoming Anti-PEG Antibody Responses to Increase Potency & Decrease Adverse Side Effects of mRNA-LNP Formulations
9:30 am Phospholipids in Vaccines
Synopsis
- The amphiphilic nature and safety profile of phospholipids make them ideal excipients for LNP formulations.
- The lipid composition of LNPs and their impact on the delivery of mRNA.
- Helper lipid selection can modulate the properties of LNP delivery and stability.
- Phospholipids and their role in delivering adjuvanted liposomal vaccines.
10:00 am AI-Assisted Design of IV-Administered Organ-Targeting LNPs for Effective Nucleic Acid Delivery to Extrahepatic Organs
8:50 am Chair’s Opening Remarks
Tackling mRNA CMC Challenges Through Regulatory Engagement & Platform Strategies for Faster Manufacturing
9:00 am Anticipating & Planning for CMC-Related Regulatory Hurdles to Advance the Manufacturing of Next Generation mRNA Vaccines
9:30 am Aligning Quality Control & Regulatory Submissions for Personalized Cancer Vaccines to Get Life-Saving Drugs to Patients Faster
10:00 am Discussing How to Engage with the EMA to Expedite mRNA Medicines to Patients Faster
10:30 am Morning Networking Break
Innovating Nanoparticles Beyond LNPs to Access Specific Organs & Supercharge Your mRNA Delivery
11:00 am Crafting a Multi-Layered Nano Formulation Approach to mRNA Stabilization & Cell-Specific Delivery Using a Novel Nucleic Acid Delivery Platform
11:30 am yEVs Enabling the High-Efficiency Introduction of Diverse Cargos, Including mRNA, Functional Antibodies, & Enzymes, to a Broad Range of Cell & Tissue Types
12:00 pm Engineering Futuristic & Next Generation Nanoplatforms for Delivery of mRNA Therapeutics: Academia-to-Industry Innovation Mind Shift
Building Pre-Clinical, Process, Analytical & Comparability Strategies to Create a Holistic Regulatory Package for Your mRNA Platform
11:00 am Creating an mRNA Process & Analytical Toolbox to Meet Platform Requirements in a Regulatory Context
11:30 am Comparability – Regulatory Strategy Considerations for mRNA Product CMC Changes
12:00 pm Planning for Success: Creating a Regulatory Strategy for the Successful Development of a Novel mRNA Platform
12:30 pm Lunch Break & Networking
Optimizing System Design to Increase Delivery Efficiency & Unlock Novel Therapeutic Applications of mRNA
1:30 pm Panel Discussion: Delving into the Latest Innovations in LNPs & Beyond for Effective Delivery of mRNA Therapeutics
2:00 pm Moving Beyond the Needle: mRNA Vaccine Delivery Via Dermal, Pulmonary, & Nasal Pathways
2:30 pm Designing Lipid Nanoparticles for mRNA Delivery to Study & Treat Diseases in Pregnancy
Engaging with the Regulatory Nuances for Different Disease Areas to Create Effective mRNA Drugs Against All Indications for All Patients
1:30 pm Evolving the Regulatory Landscape to Unlock the Potential of mRNA Platforms for Rare Diseases
2:00 pm Disease-Specific & Personal: mRNA Therapy Immunogenicity Risk for Rare Diseases
2:30 pm Addressing the Differences in Regulatory Strategy for Various Batch Sizes of Personalized Cancer Vaccines
3:15 pm Afternoon Networking Break
3:45 pm
Breakthrough Innovations Workshop
Save millions of lives, trillions of dollars, and enable real global equity
Synopsis
The R3: RNA Readiness and Response program, took a page from the semiconductor industry transformation in the 1980s, which developed multi-product foundries with standardized processes and design tools that exploded global innovation.
This session will cover R3 advances in:
- Manufacturing processes, with the potential for 10mg to 1kg of RNA-based product, or up to 100 products, per day, in a 35 m2 footprint
- Vaccines and therapeutic products that target >30X dose-reduction and >100X cost-reduction compared to state-of-the-art
- In-silico design tools that enable >5X dose-reduction and manufacturability verification with >90% accuracy, for a democratized access to the RNA Ecosystem
Key Takeaways
- Standardized, multiproduct, continuous RNA manufacturing processes enable GMP production of 100 milligram-scale RNA therapeutics, as well as 1 billion RNA vaccine doses, in one day, in less than 35 m2.
- Digital design and verification tools democratize access to RNA manufacturing providing >5X increase in biological performance and >90% accuracy in manufacturability prediction.
- The same fab-less / pure-play foundry model that developed the semiconductor industry, can now enable a vibrant, self-sustaining RNA Ecosystem providing equitable access to RNA-based products and economically sustainable pandemic preparedness.