7:45 am Morning Networking Coffee

8:20 am Chair’s Opening Remarks

  • Anna Senczuk Director - Process Development & mRNA CMC , Seqirus

Accelerating Analytical Methods of mRNA Drug Substance for Optimal Product Characterization

8:30 am Analytical Characterization of mRNA-Based Therapeutics & Vaccines

  • Andreas Kuhn SVP - RNA Biochemistry & Manufacturing, BioNTech SE

Synopsis

  • As a still new technology for therapeutics and vaccines, the critical quality attributes for mRNA need further refinement
  • For this, it is important to characterize mRNA as good as possible and correlate this to its functionality
  • Here, a set of assays for the characterization of mRNA will be presented

9:00 am Analytical Characterization of mRNA Replacement Therapy for Rare Genetic Disease

  • Zheng Meng Senior Director - Analytical Development, Ultragenyx Pharmaceutical

Synopsis

  • Potential critical quality attributes for mRNA replacement therapy
  • Examples of mRNA potency analysis
  • Safety related impurity control

9:20 am Trehalose and Sucrose: Essential Components of Platform Biopharma Formulations and Covid 19 applications

Synopsis

  • Introduction of Pfanstiehl and its high quality/purity GMP components
  • Commercial Biotherapeutics Stabilized with Trehalose and Sucrose
  • Examples for utilizations and functionalities of Sucrose and Trehalose in COVIS-19 related formulations and applications

9:40 am Panel Discussion – Benchmarking Regulatory Harmonization for State-of-the-Art Analytical Methods, Qualification, and Validation

  • Vijay Dhawan Head – Analytical Development/QC, Genomic Medicine, Sanofi
  • Andreas Kuhn SVP - RNA Biochemistry & Manufacturing, BioNTech SE
  • Elaine Peters Director – Cell & Gene Therapy Analytical Capabilities, Genentech
  • David Cirelli Senior Director - Analytical Research & Development , Pfizer
  • Shefal Parikh Director - mRNA Process Development, Beam Therapeutics
  • Zheng Meng Senior Director - Analytical Development, Ultragenyx Pharmaceutical

Synopsis

With the expansion of mRNA-therapeutic applications at an accelerated pace, there is now a greater necessity to characterize mRNA to better understand their mechanisms of action and the performance systems. Novel analytical approaches, specifically within production, purification and high throughput automation needs to be developed to address design, test and analyse such systems. Validation of analytical methods for mRNA-based vaccines and therapeutics to improve quality control requires a deeper focus and will be explored in this panel discussion to combat the challenges and provide solutions for the future of analytical development

  • How to better test, characterize and validate mRNA to assess drug substance quality and efficacy?
  • Evaluating regulatory guidelines for cGMP, method validations and elemental impurities
  • How to address scale-up challenges you might encounter while characterizing mRNA-based vaccines and therapeutics?

10:30 am Morning Break & Networking Break

Exploring New Methods to Develop Next Generation mRNAs

11:30 am Chemical Synthesis of mRNA & The Structure Activity Relationship – Live Digital Presentation

Synopsis

  • Chemical synthesis
  • Non-enzymatic Capping
  • Unnatural nucleic acids

12:00 pm Overcoming Production Hurdles for Next Generation of mRNA Molecules

Synopsis

  • Advantages and issues of different mRNA formats: unmodified mRNA, modified mRNA, circular mRNA, self-amplifying mRNA
  • Novel chemically synthesized mRNA
  • Ongoing pre-clinical and clinical usages of synthetic mRNAs

12:30 pm Networking Lunch Break

Afternoon Chair

Redesigning Production Processes to Develop Effective mRNA-Based Therapeutics and Vaccines

1:30 pm Panel Discussion – Developing High-Quality mRNA to Ensure Optimal Functionality

  • Thomas Aune Professor, Vanderbilt University Medical Center
  • Kristie Bloom Researcher, University of the Witwatersrand
  • Craig Martin Professor - Chemistry , University of Massachusetts Amherst
  • Elvan Cavac Scientist II - mRNA Upstream Process Development , Tessera Therapeutics

Synopsis

  • Enhancing DNA template quality to ensure better preparation for mRNA production
  • How to improve methods for mRNA production from cell free processes to synthesis and modifications
  • What roles do modified nucleotides play in activity and immunogenicity?

2:00 pm Mechanism-based Improvements to RNA Manufacturing Yields & Purity

  • Craig Martin Professor - Chemistry , University of Massachusetts Amherst

Synopsis

  • A simple DNA modification dramatically improves purity (and in some cases, yield)
  • A new universal reagent allows for multiple rounds of repeat batch synthesis
  • The future is in continuous flow synthesis of RNA.

2:30 pm Afternoon Networking Break

Optimizing mRNA Upstream & Downstream Process Methods for Improved Efficiency & Quality

3:10 pm Benchmarking IVT Reaction Conditions for Optimal mRNA Drug Substance Yield

  • Xinyue Zhang Investigator - mRNA Process and Analytical Development, GeneLeap Biotech

Synopsis

  • Transforming upstream bioprocessing with bioreactions from pharma 4.0
  • Optimizing IVT reaction mixtures ratio to reduce by-product formation for greater yield
  • Assessing key parameters of IVT for reaction volumes titers, mixing speed, and temperature sensitive variations
  • Optimized IVT to have less pressure for downstream

3:40 pm Novel Purification Approach & Tools to Define mRNA Specifications, Quality & Manufacturing

  • Shefal Parikh Director - mRNA Process Development, Beam Therapeutics

Synopsis

  • How to effectively define contaminant types and purity profiles in raw materials, process and overall drug substance?
  • Unravelling end-to-end purification workflow for mRNA drug substance following the IVT reaction
  • How to define suitable purification profile differences and consistency between therapies that are in line with regulatory expectations?

4:10 pm Chair’s Closing Remarks & End of mRNA Process Development & Manufacturing Summit 2022