Pre-Conference Workshop Day
Tuesday, February 13, 2024
8:00 am Registration & Morning Coffee
8:50 am Chair’s Opening Remarks
9:00am Workshop A
How to Create a Reference Document to Supplement Industry Regulatory Guidance
Synopsis
In the dynamic and rapidly evolving landscape of mRNA therapeutics, the development of a comprehensive reference document that supplements industry regulatory guidance is a vital tool for ensuring the safe and effective development of these groundbreaking treatments. In order to do so, we can look at the entire process from research and development to clinical trials and post-market surveillance. This includes evaluating the manufacturing process, assessing the safety and efficacy of the therapeutic, and monitoring for any adverse events. Ultimately, a thorough review of standards and regulatory requirements is necessary to ensure that mRNA therapeutics are both safe and effective in protecting public health.
Join this session to discuss:
• The quality issues regarding mRNA therapeutics and how we can overcome them
• What is expected by the regulatory agencies for the clinical and non-clinical requirements
• The regulatory guidelines for CMC’s and manufacturing testing to allow a streamline approval
• The advances of having a platform approach for vaccines to allow a more rapid approval of your mRNA vaccines
11:00 am Morning Break & Networking
12:00pm Workshop B
Exploring the Next Generation of RNAs & Discussing the Current State of Associated Analytical Methods to Better Understand RNA & Achieve Successful Applications Towards Effective mRNA Therapeutics
Synopsis
In recent years, research into RNA has been expanding rapidly, revealing new insights into their structure, function, and potential uses. Despite the growing interest in RNA, however, there is still much to be learned about these molecules, particularly when it comes to their analytical methods. While there are a variety of techniques available for studying RNA, such as RNA sequencing and Northern blotting, many of these methods have limitations and can be difficult to use. As a result, scientists are constantly exploring new ways to analyse RNA, both to better understand its role in the cell and to develop new applications for this versatile molecule.
Join this session to discuss:
• The differences between techniques used for circular RNA compared to those used for standard mRNA
• The potential of self-amplifying RNA as the new effective vaccine technology
• The advantages of using siRNA therapy compared to conventional antiviral drugs and delve into the potential ways to deliver siRNA-based treatments
• How we can align RNA technologies with mRNA analytical methods to create more effective and efficient treatments for a variety of diseases
2:00 pm Networking Lunch
3:00pm Workshop C
Innovations in Addressing dsRNA Challenges for Advancing mRNA Therapeutics
Synopsis
One of the main challenges for mRNA therapeutics is the presence of doublestranded RNA (dsRNA), which can trigger unwanted immune responses and limit the efficacy of mRNA therapies. A key development in tackling dsRNA challenges is the refinement of mRNA design and modification techniques. Scientists have been working to engineer mRNA molecules that have reduced dsRNA content, thereby minimizing the potential for immune activation. Overcoming these hurdles will pave the way for more precise, potent, and safer mRNA therapies with the potential to revolutionize the treatment of various diseases.
Join this session to discuss:
• dsRNA ability to effectively target specific mRNA sequences, resulting in increased specificity and accuracy in mRNA analysis
• The possible strategies to improve its specificity and reduce the potential for offtarget effects
• The regulatory hurdles in the use of dsRNA in mRNA analytical development to ensure safety and efficacy of these technologies