Chemical, pharmaceutical and analytical development of oligonucleotides continues to be challenging due to the increased complexities of development programs and an evolving regulatory landscape. As contract organizations are a major part of most development efforts, it is becoming increasingly important to consider business issues such as intellectual property, know how, confidentiality and other factors when choosing a manufacturing partner as these factors impact downstream development such as site/process changes; business development; licensing, etc. This workshop will present strategies and case studies for managing the business aspects of process and analytical development for commercial and regulatory success.

Hear in-depth presentations on what you need to know about chemical, analytical and pharmaceutical development of peptides as you develop your peptide therapeutic. Topics covered will include updates on the latest synthesis technologies and analytical methods. Learn also about the latest in regulatory trends including the new process validation guidance. Participate in an interactive discussion to ask your questions to the experts.

This course will cover the essential subject matter required to quickly and efficiently move a discovery candidate into the clinic. Specific examples to be discussed will address the realities of short timelines, limited drug supply and technological peculiarities. Additionally, attention will be given to the goals for three very different formulations: toxicology; clinical proof-of-concept; and efficiency improvements.

1. What is the goal of formulation?
2. Toxicology formulation considerations
3. Pre-formulation studies (pilot formulations)
4. Formulation development: parenteral
5. Formulation development: non-parenteral
6. Supporting analytical strategy
7. Evolution of the drug product specification
8. Clinical batch considerations - production, supply chain, and stability support

Session 1 The course will outline approaches to conjugate, purify and analyze peptide-oligonucleotide conjugations. Session 1 will address the various methods of conjugating peptide to oligonucleotides

• Linker chemistry (amide, ester disulfide)
• Solid phase versus solution phase conjugation
• Monufunctional versus bifunctional linkers
• Location and placement of linkers

Session 2 will address purification strategies for peptide-oligonucleotide conjugates

• Purification methods applicable to conjugates (anion exchange and RP HPLC)
• Desalting
• Potential pitfalls and how to avoid them

Session 3 will address analytical methods and equipment

• Analytical anion exchange HPLC
• Ion pair RP-HPLC
• LCMS and impurity profiles
• Size exclusion chromatography

Dr. Houston leads both the oligonucleotide and peptide chemistry efforts at Marina Biotech including research and development activities. Previously, he held the position of Director of Chemistry at Cytovax Biotechnologies where he led the research and manufacturing efforts of the company's peptide-protein conjugate-based vaccine. Dr. Houston received his B.Sc. and Ph.D. in Chemistry from the University of Waterloo and completed a Postdoctoral Fellowship in Protein Engineering in the laboratory of Dr. Robert Hodges at the University of Alberta.

This course will discuss creative strategies for accelerating the process of oligonucleotide lead discovery, lead optimization and candidate selection, with an emphasis on successful translation from discovery to pre-clinical studies. Hear practical experiences and lessons learned on the path from discovery to IND from speakers representing leading oligonucleotide therapeutic companies.

You will learn about:

• Novel chemistry motifs and platforms to accelerate candidate selection
• Identifying chemistries for enhancing target specificity and in vivo stability
• Sequence design and modifications to reduce immune responses and off-target effects
• Strategies for identifying oligonucleotide candidates that will translate from in vitro to in vivo to pre-clinical studies
• Library synthesis and creative molecular design strategies