Conference Day One - Wednesday, September 18
7:30 am Check In & Morning Coffee
8:20 am Chair’s Opening Remarks
Accelerating mRNA Process Development Through Platform-Level Advancement to Expedite Drug Manufacturing
8:30 am Panel Discussion: Planning for the Future of mRNA Drugs: How Can CMC Platforms Support the Next Wave of Approvals in the mRNA Field?
9:00 am Platform-Based Approaches to Streamline mRNA Drug Substance Process Development & Validation
Synopsis
- Significant experience and knowledge gained through development of the COVID-19 vaccine provides a basis for a robust mRNA platform technology
- Taking a risk-based approach to leverage platform knowledge and streamline process development and validation for a multi-valent mRNA vaccine
- Strategies to incorporate regulatory framework for platform technologies and continuous learnings to feed forward to future mRNA programs
9:30 am mRNA-LNP Manufacturing Platform
Synopsis
Typical mRNA production process involves four key steps:
- plasmid DNA (pDNA) expression in E.coli, linearization and pDNA purification
- in-vitro transcription (IVT) reaction
- mRNA purification
- mRNA encapsulation
Orthogonal analytics are required to enable safer product and cheaper manufacturing process
Critical steps to lower the manufacturing costs will be presented
10:00 am Morning Break & Speed Networking
Research Scale Track
Overcoming Bottlenecks with IVT Reactions for Faster Scale Up While Maintaining mRNA Yield & Quality
11:00 am Optimizing IVT Reactions to Effectively Scale-Up mRNA Production from Research to Pre-Clinical Scale & Beyond
11:30 am Session Reserved For Syngoi
12:00 pm Addressing Purification Challenges Resulting from Scale Up of Design of Experiments (DOE) Optimized IVT Reactions
IND-Enabling & Clinical Scale Track
Highlighting Process Analytical Technologies (PATs) to Predict & Control mRNA Quality, Stability & Potency
11:00 am Spotlighting a Novel Fluidics Approach to the Manufacturing of RNA That is Fully Pure at the Outset & Implementing a Novel On-Line Assay to Determine RNA Quality
11:30 am Innovative Cap Analog Design for Enhanced mRNA Immune Evasion
Synopsis
- This presentation delves into innovative cap analog designs that enhance mRNA immune evasion and translation efficiency
- The talk explains the role of 5’ mRNA end capping in distinguishing self from non-self RNA and how various cap structures affect immune recognition
- Using structural insights from literature studies, novel cap structures are proposed that are hypothesized to evade IFIT1 binding, thereby improving translation under immune stress
- The efficacy of these designs is showcased through in vitro and in vivo models of inflammation, demonstrating their potential to improve mRNA-based therapeutic outcomes
- Attendees will gain valuable knowledge on the rational design of mRNA cap analogs and their potential impact on RNA therapeutics
12:00 pm Outlining Analytical & Potency Assays for RNA Product Predictability of Efficacy for Multivalent Drug Products
12:40 pm Lunch Break & Networking
Enhancing Next Generation mRNA Process Development to Accelerate the Production of Novel Modalities
1:40 pm Improving the Purification Process for Self-Amplifying mRNA (sa-mRNA) to Enhance Encapsulated LNP Vaccine Drug Products
2:10 pm Overcome dsRNA Challenge: Innovation in Assays & Enzymes
Synopsis
- Enhanced mitigation of dsRNA is imperative
- ds-SensorTM: State-of-the-Art dsRNA Assay
- Next-Generation T7 showcases comprehensive performance enhancements
2:40 pm Selecting the Optimal Circularization Method for Circular RNA-Based Therapeutics, Depending on the Target Product Profile
Getting a Fast Start into the Clinic: Tackling Bottlenecks While Scaling Up mRNA Drug Product Manufacturing
1:40 pm LNP Process Characterization & Optimization for Phase III Readiness for a Personalized Cancer Therapy
2:10 pm Hitchhikers Guide to Lean Manufacturing: Redefining the concept of mRNA development
Synopsis
Moving towards higher sensitivity for detection of residuals
Developing manufacturing flexibility to meet patient needs.
Optimizing upstream operations regarding integrity, capping efficiency, and potency
Navigating regulatory restrictions on raw materials and process
Speaker TBD