Delivery & Manufacturing Focus Day

8.30am - 3.45pm EST | 5.30am - 12.45pm PST

8:30 am Opening Registration & Coffee Networking

Novel Techniques & Next Generation Strategies for Enhanced mRNA Delivery

9:00 am Discussing Next Generation Delivery Techniques & Platforms for Highly Effective Delivery of mRNA

  • Daniel Griffith Anderson Professor of Chemical Engineering, Institute for Medical Engineering and Science, Koch Institute for Integrative Cancer Research at MIT

Synopsis

  • Outlining future perspectives of an mRNA platform for delivery, which will allow stable, effective, and safe delivery of wide-ranging mRNA therapies
  • Benchmarking strategies for successful delivery and considering the critical elements for success

9:30 am Biomimetic Peptide Coacervates as Safe & Efficient Delivery Vehicles of Macromolecular Therapeutics

  • Ali Miserez Professor, Nanyang Technological University, Singapore

Synopsis

  • Peptide coacervate microdroplets self-assembled by liquidliquid phase separation are capable to recruit a wide range of therapeutics in a safe and rapid fashion
  • These coacervates readily cross the cell membrane and can be chemically designed such as to release their cargos through various stimuli, such as pH, temperature, or external magnetic field
  • These delivery agents are also safe: therapeutics are recruited under aqueous conditions and they do not elicit any cytotoxicity

10:00 am Novel Delivery Appriaches to Enhance Stability, Effectivity & Safety of mRNA Therapies

  • Yizhou Dong Associate Professor, The Ohio State University 

Synopsis

  • Discussing key hurdles for mRNA delivery, from choosing the active ingredient through to the optimal route of delivery
  • Reviewing an effective delivery method to ensure vaccine safety, effectivity, potency, and tolerability
  • Overview of clinical and pre-clinical insights from effective approaches to mRNA vaccine delivery

10:30 am Commercialization and Technology Transfer of mRNA Therapeutics and Lyophilized Vaccines

  • Dushyant Varshney Executive Vice President & Chief Technology Officer, Arcturus Therapeutics

Synopsis

• The novel development and manufacturing technologies for mRNA  therapeutics and lyophilized vaccines
• Current challenges and success in large-scale production of mRNA vaccines
• Global technology transfer, process validation rapid  commercialization, and supply strategies

11:00 am Live Panel Q&A – Ask Speakers your Burning Questions

  • Daniel Griffith Anderson Professor of Chemical Engineering, Institute for Medical Engineering and Science, Koch Institute for Integrative Cancer Research at MIT
  • Ali Miserez Professor, Nanyang Technological University, Singapore
  • Yizhou Dong Associate Professor, The Ohio State University 
  • Dushyant Varshney Executive Vice President & Chief Technology Officer, Arcturus Therapeutics

11:20 am Morning Break

12:00 pm Panel Discussion: Debating Between Techniques & Strategies for Optimized Delivery of mRNA Treatments

  • Uwe Gottschalk Chief Scientific Officer, Lonza
  • Daniel Griffith Anderson Professor of Chemical Engineering, Institute for Medical Engineering and Science, Koch Institute for Integrative Cancer Research at MIT
  • Venkata R Krishnamurthy Director & Senior Advisor, Eli Lilly & Company 
  • Mouhannad Jumaa Senior Vice President of Pharmaceutical Development, Turn Biotechnologies
  • Aalok Shah Associate Director, Formulation Design, Strand Therapeutics Inc.

Synopsis

With the continuous success off mRNA-based candidates, it is clear mRNA-based therapies are a promising tool for the therapeutic intervention against oncology and infectious diseases. Despite this, translation of mRNA-based therapeutics from the bench to the bedside is limited due to various factors impeding the successful and effective delivery of mRNA molecules. From inhalational delivery, lipid nanoparticles, intranasal vaccine administration to naked mRNA, we will breakout to discuss and debate the various tools being used, and what is on the horizon. Input from a planned participant survey will be used to focus on technologies and strategies being considered most important by the audience for delivery of mRNA therapeutics and vaccines.

12:45 pm Lunch Break

Placing Focus on CMC & Process Development to Advance mRNA as an Effective Therapy

1:45 pm Deliberating Novel Delivery & Advanced Manufacturing of mRNA Therapies to Optimize Commercialization

Synopsis

Optimized CMC & Process Development of mRNA Vaccines

  • Considering the optimal development and design of mRNA molecules for therapeutic application in vaccines for infectious disease
  • Establishing effective processes for the manufacturing and analytical development of mRNA treatments
  • Identifying novel methods to enhancing stability and function of mRNA candidates

2:15 pm A Platform Manufacturing Approach to CRISPR/Cas9 Genome Editing Therapeutics

  • Kristy Wood Senior Director, Technical Operations, Intellia Therapeutics

Synopsis

  • Intellia focuses on platform manufacturing approaches for both mRNA and sgRNA drug substances and LNP drug products
  • Limited batch manufacturing and material availability in early development can present challenges for process and analytical development
  • Leveraging platform manufacturing decreases development time, increases speed to clinic, and helps regulators gain familiarity with complex CMC

2:45 pm Process Development – From Design & Development to Technology Transfer of mRNA Drug Substance Manufacturing Process

  • Jimmy Chu Scientist II, mRNA Process Development, bluebird bio

Synopsis

  • Establishing processes for the manufacture and scale-up of mRNA for gene editing application
  • Exploring optimal analytical development of mRNA

3:15 pm Panel Discussion: Delving into Optimized the Process Development to Support mRNA Therapeutic Development & Assess Clinical Translation Efficiency

Synopsis

Ask our experts your burning questions, and dive into the roadmap to success when discovering and developing your mRNA therapeutics. Come prepared with questions, thoughts and ideas as this panel will be an interactive session, providing the opportunity to discuss these important topics with other mRNA experts, and discover how to advance your mRNA therapies through clinic and to the patient.

4:00 pm End of Delivery & Manufacturing Focus Day