Pre-Conference Day

Monday July 29, 2024

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

  • Annette Bak Head of Advanced Drug Delivery, AstraZeneca

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

  • Nazia Noor Vice President Global Regulatory Affairs CMC, BioNTech New Data!

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

  • Andy Geall Co-Founder & Chief Development Officer, Replicate Biosciences New Data!

9:30 am Roundtable Discussion: Comparing the CMC Package for Personalized Cancer Vaccines in an Investigator Versus Industry-Sponsored Trial Setting

  • Nazia Noor Vice President Global Regulatory Affairs CMC, BioNTech New Data!
  • Hang Yuan Chief Technical Officer & Co-founder, Innovac Therapeutics New Company!
  • Ka-Wai Wan Senior Pharmaceutical Assessor, Medicines & Healthcare products Regulatory Agency

10:00 am Discussing How to Engage with the EMA to Expedite mRNA Medicines to Patients Faster

  • Marco Cavaleri Head of Office of Biological Health Threats & Vaccines Strategy, European Medicines Agency

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

  • Jessica Rouge Associate professor, University of Connecticut New Data!

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

  • Beata Chertok Team Lead, Delivery of Intracellular Biologics, AstraZeneca

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

  • Kumar Namdev Global Head of CMC Product Development & Industrialization, Sanofi

11:30 am Comparability Strategy Considerations for mRNA CMC Changes to Advance Regulatory Strategy

  • Nazia Noor Vice President Global Regulatory Affairs CMC, BioNTech New Data!

12:00 pm Planning for Success: Creating a Regulatory Strategy for the Successful Development of a Novel mRNA Platform

  • Nitisha Pyndiah Global Director - Global Regulatory Affairs Lead, GlaxoSmithKline Plc

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 Nanoparticle Chemistry for Effective Delivery of mRNA Therapeutics

  • Beata Chertok Team Lead, Delivery of Intracellular Biologics, AstraZeneca
  • Annette Bak Head of Advanced Drug Delivery, AstraZeneca
  • Andy Geall Co-Founder & Chief Development Officer, Replicate Biosciences New Data!
  • Allen Horhota Vice President - Platform, Orna 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

  • Rachel Riley Assistant Professor, Rowan University New Data!

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

  • Hang Yuan Chief Technical Officer & Co-founder, Innovac Therapeutics New Company!

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.  

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6:45 pm Drinks Reception