Pre-Conference Day
The purpose of this workshop is to introduce scientists to therapeutic oligonucleotides. This workshop will discuss the different types of therapeutic oligonucleotides and how they work in the body to treat disease. Difference modalities such as SiRNA, ASO, MiRNA and Aptamer will be covered. The key factors for the design of the molecules will be described. The workshop will also discuss the challenges of delivery to the appropriate tissue and into the appropriate cell, and the strategies currently employed to address these. The toxicology, metabolism and clearance in the body will be covered. Finally, the workshop will discuss formulation options and how the drug substances and drug products are manufactured and controlled.
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?
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.