Below is What Will Be Covered:

Summary:

The workshop will introduce the general chemical structure of oligonucleotide and then summarise the oligonucleotide medicines currently approved, their type and disease indication. Following this the different modalities will be discussed (ASO SiRNA etc. etc.). A summary of the challenges for this class will be provided. This will be an extensive introduction to set the scene for the rest of the course.

The following sessions will go into detail about the design attributes of oligonucleotides (sequence, chemical modifications, conjugates and other tissue and cellular delivery options). The workshop will then go on to discuss what happens to the molecules once they are systemic in the body including their disposition in the body, metabolism and clearance. A summary of the known general toxicological liabilities is also discussed.

Finally the CMC aspects will start with the formulations currently used, some brief considerations for clinical trials and finish with as summary of the synthesis and analytical control of active oligonucleotides

Why are we doing it? What is the challenge for market? What are the FEAR factors? What is the topic?

Synthetic oligonucleotides are now a class of therapeutics that have become very important with at least 15 approved medicines with 12 approved in the last 6 years. Originally designed for rare diseases we expect oligonucleotide medicines to also treat diseases with a significant world-wide patient population (e.g. dyslipidaemia, hepatitis, cancer). There are small and medium sized biotechnology companies specialising in oligonucleotides as well as big pharma investing heavily in them and there is a rapidly increasing CDMO presence. As a result of these investments and the huge potential for oligonucleotides as transformative medicines, more and more scientists across drug discovery and development disciplines are turning their hand to oligonucleotides. With this expansion we see the needs for a primer introduction to help onboarding of new scientists and also scientists who want to expand their development knowledge. To our current knowledge there is currently no course that introduces scientists across disciplines to oligonucleotides, what they are, how they work, how they are designed, what happens to them in the body how they are made and what the current challenges are?

Themes Include:

• Current state of oligonucleotide therapeutics
• Introducing oligonucleotide molecular structure
• Various therapeutic biological mechanisms
• Challenges for this class of medicines
• Metabolism, disposition, clearance and toxicology
• Clinical considerations
• Formulation
• Chemistry, manufacturing and analysis
• Regulatory landscape