February 25-27, 2025 | Boston, MA
Part of the World RNA Series

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.

This workshop will address the following questions:

• What are the construct-dependent factors that influence dsRNA formation in mRNA therapeutics, and can they be managed?
• What is the current regulatory perspective, specifications and opinion on surrogate standards for dsRNA quantification and control?
• What is known of the tolerability thresholds for dsRNA concerning immunogenicity, and how do they impact therapeutic safety?
• What are the latest analytical advancements in sensitive detection methods for dsRNA?
• What is the perspective on accurately ranking dsRNA fragment and length populations regarding their immunogenicity impact?

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.

This workshop will discuss:

• The implications of DNA templates and circular RNA in mRNA therapeutic development
• How to navigate the current advancements in techniques for analyzing nucleic acid structure, stability, and enzymatic degradation
• Which key impurity and purification challenges are specific to nucleic acid analysis, with a focus on toxicology aspects
• How we can align RNA technologies with mRNA analytical methods to create more effective and efficient treatments for a variety of diseases

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.

This workshop will answer:

• Which recent strides are being made in the development of mRNA gene editing therapies?
• What are the key analytical challenges to determine gene editing CQAs, and how can they be overcome with novel technologies and workflows?
• How can we address the unique quality and regulatory requirements for novel mRNA precision medicines compared to traditional mRNA applications?