Pre-Conference Day (May 19)
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.
- James Powell - SVP, Manufacturing and Boulder Site Head, Novo Nordisk
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.