Conference Day Two

Thursday, 27th June

8:30 am Check-In & Light Breakfast

9:20 am Chair’s Opening Remarks

Revolutionizing mRNA Delivery Approaches & Adhering to Compliance Requirements & Considerations to Streamline Approval of Your mRNA Therapeutics

9:30 am Exploring the Development & Optimization of mRNA Delivery Systems to Advance mRNA Therapeutics & Vaccines

Synopsis

  • What are the possible methods of characterizing your mRNA delivery systems to understand their performance under different conditions?
  • How to optimize the interaction between your mRNA delivery systems and cellular machinery that can lead to degradation of the mRNA to improve the stability and efficacy of mRNA delivery systems
  • What are the challenges associated with endotoxin testing in the context of lipid-based formulations

10:00 am Addressing the Innovation in mRNA Formulation to Overcome Stability Challenges & Enhance Your mRNA Vaccines

  • Hossam Samy Associate Director, QC Technical Service., Alnylam Pharmaceuticals

Synopsis

  • Statistical methodologies used for stability data trending and shelf life assessment per Q1E guideline
  • Challenges with interpretation and trending of stability data for testing performed at external/ contract testing laboratories (CTL)

10:30 am Morning Break & Networking

Emphasizing the Importance of High-Quality Raw Material Testing & Complying with Regulatory Guidelines to Ensure Efficacy & Stability of Your mRNA Therapeutics

11:30 am The Use of Risk Assessment to Evaluate the Quality Impact of Raw & Starting Materials Used in mRNA Manufacturing

  • Angel Tai Manager, External QA Operation, Sanofi

Synopsis

  • Explanation of how risk assessment helps identify, evaluate, and mitigate potential quality risks associated with raw and starting materials
  • Discussion of key considerations for conducting risk assessments in compliance with regulatory expectations
  • Illustration of how risk assessment helps prioritize critical quality attributes and parameters for raw and starting materials

12:00 pm Panel Discussion: Control of Enzymes, Nucleotides, Lipids & Other Raw Materials to Ensure Product Quality

Synopsis

  • Discuss the complexities and variability associated with enzymes, nucleotides, and lipids
  • Examining regulatory guidelines related to the control of raw materials in mRNA production
  • How can you ensure compliance with standards set by regulatory bodies?
  • What are the strategies for effective supply chain management to ensure a consistent and high-quality raw material supply
  • What are the challenges in quality control for plasmid manufacturing, considering its role as a GMP starting material

1:00 pm Networking Lunch

Advancing Methods for mRNA CQA Applications to Ensure High Quality Analysis & Explore Risk Assessments to Help Identity, Evaluate & Mitigate Potential Quality Risks

2:00 pm Explore the Concept of Platform Methods in mRNA Quality Control & the Need for Maintaining Validation Status in a Quality Control Environment

Synopsis

  • Discuss the verification process for platform methods and the regulatory perspective on the extent of verification required for new molecules
  • What are the innovative platforms for mRNA therapeutic development, focusing on adherence to regulatory guidance
  • Examine challenges in maintaining consistency across platforms and addressing varying regulatory expectations

2:30 pm Update on the Development of Standardized mRNA/LNP CQAs for Therapeutic Applications

  • Steven Wolk Vice President of Chemistry & Boulder Site Head, Editas Medicine

Synopsis

  • Current state of existing regulatory guidance
  • Review of the proposed list of CQAs, and analytical methods
  • Areas for additional development
  • CRISPR-specific considerations

3:00 pm Unveiling LNP Payload, Heterogeneity & Correlation With in-vivo Transfection by Single Particle Analysis

  • Sixuan Li Research Associate, Johns Hopkins University

Synopsis

  • Discussing the complexities of analyzing LNP payload, including mRNA encapsulation efficiency and distribution
  • Addressing challenges related to LNP heterogeneity and its impact on mRNA delivery efficacy
  • Case studies demonstrating how single particle analysis can elucidate correlations between LNP properties and mRNA delivery outcomes

3:30 pm End of the 2nd mRNA Quality Control & Compliance Summit 2024