PRE-CONFERENCE WORKSHOP DAY
Tuesday, February 25, 2025
8:00 am Check-In & Light Breakfast
Workshop A | 8:30
Overcoming the Longstanding Challenges in the Characterization & Control of dsRNA to Develop Safer & Regulatory-Compliant mRNA Therapeutics
Synopsis
dsRNA presents a significant, persistent and debated challenge in the development of safe and effective mRNA medicines. The construct-dependent nature of dsRNA formation complicates efforts to standardize production and quality control, as the absence of a standardized sensitive detection and surrogate standard hinders the establishment of universally accepted guidelines for dsRNA levels. The immunogenicity of dsRNA raises concerns about tolerability, making it crucial to determine acceptable levels, fragments and lengths of dsRNA for release. This workshop aims to address these pain points and regulatory perspectives alongside
recent strides in the field to develop more sensitive detection methods and progress the ranking of dsRNA fragments and populations to better predict and control their impact on therapeutic outcomes.
10:30 am Morning Networking Break
Workshop B | 11:30
Optimizing Analytics for Next Generation Nucleic Acid-Based Therapeutics to Better Understand RNA & Achieve Successful Applications Towards Effective mRNA Therapeutics
Synopsis
Nucleic acid-based therapeutics have emerged as a promising avenue for treating various diseases, including genetic disorders, cancers, and infectious diseases. With recent advancements in technology and understanding of nucleic acid biology, there is a growing need for advanced analytical techniques to optimize the development and efficacy of these therapeutics. This workshop aims to provide participants with an in-depth understanding of advanced analytical methodologies tailored specifically for next generation nucleic acid-based therapeutics.
1:30 pm Lunch & Networking Break
Workshop C | 14:30
Enhancing Analytical Methods & Quality Control Validation to Bolster the Discovery & Clinical Progression of Novel mRNA Gene Editing Therapies
Synopsis
The advancement of mRNA gene editing technologies presents significant therapeutic potential for precision medicines. However, the development and clinical progression of these therapies is armed with increasingly rigorous validation, characterization and optimization constraints requiring both new and modified analytical tools. This workshop aims to address these challenges and explore cutting-edge solutions for analytical and quality control validation in the realm of novel mRNA-based therapies.