Pre-Conference Day (Apr 26)
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.
- Sequence selection
- The role of chemical modifications
- The use of conjugates and cationic lipid encapsulation to enhances tissue and cell penetration
Cyclic peptides demonstrate potential for oral absorption and intracellular targeting. LUNA18 is N-alkyl-rich cyclic peptide composed of 11 amino acids. To manufacture LUNA18 API at large scale, liquid-phase peptide synthesis (LPPS) was developed over solid-phase synthesis. Challenges arose from N-alkyl unnatural amino acids causing low reactivity and side reactions and novel LPPS process technologies overcame these issues.
Companies working towards a Phase I IND/IMPD are usually faced with tight timelines and limited budgets to reach this important milestone. Careful planning is crucial to success. In this workshop, key nonclinical, CMC, and regulatory aspects of an IND/IMPD will be addressed. Ample time is included for a panel discussion and audience participation.
Who should attend?
Executives leading a company working towards and IND, anyone interested in CMC and nonclinical oligonucleotide activities, anyone involved in early stage drug development especially those who may be new to oligonucleotides. Quality assurance personnel, QC/analytical development chemists, toxicologists.
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.
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.