Pre-Conference Day (May 19)Monday, 19 May 2025

Scientists will be introduced to the technology of gene editing in this workshop. Participants in the workshop will learn about the different gene editing technologies available and how they work for therapeutic purposes. In this workshop, we will cover the delivery technologies available for delivering these gene editing technologies to appropriate tissues. Some of the other questions to be considered in this workshop include: What are the recent improvements and advances in genome editing technologies? What are some strategies for handling off-target detection and mitigation? What is the current status, as well as the challenges and risks of in vivo gene editing beyond the liver? How are companies handling precision targeted integration of large genetic cargo? What are the latest tools for DMPK and (bio) analytics of gRNAs? What are the current regulatory perspectives and guidances in the current landscape of gene edited products?

This workshop will provide an overview of translational strategies, technologies and inflection points to help you accelerate your peptide discovery and development efforts. Fast-tracking new therapeutics into the clinic is both an art and a science and there are multiple factors to consider. This workshop will provide insights into pharmacology strategies for the clinic, CMC considerations, pharmacokinetics and ADME as well as IND-enabling toxicology and safety pharmacology. An overview of the regulatory considerations of various translational approaches will also be discussed. A variety of case studies and examples will also be presented, along with many lessons learned from multiple R&D programs over the years.

The workshop will focus on the specific requirements for control that are common to all therapeutic oligonucleotides. For example, common impurities from solid-state synthesis especially those which come from the starting materials, the synthetic process and degradation products. The issues of determining water in hygroscopic products. Issues with assays for both single and double stranded oligonucleotides. In addition, we will discuss how establishing an ongoing control strategy is important to determine and monitor critical quality attributes that affect the drug product and the key aspects of specification setting across the phases of development. The workshop will also touch on risk assessment in late phase quality by design approaches and the role of analysis in determining critical process parameters and their relationship to critical quality attributes.