This workshop will examine CMC development of oligonucleotide therapeutics through interactive discussions. Review the key CMC concepts for oligonucleotide therapeutics and the successes and challenges for the platforms that have already progressed to product approval. Explore the use of oligonucleotides in complex and emerging modalities and assess the next generation of manufacturing platforms. Assess oligonucleotide impurities, their guidelines and how these should be controlled and addressed through interactive discussions. Delve into the regulatory expectations by phase of development and how to plan for the end goal, and understand how this can be applied to future projects
FEATURED SPEAKERS
CEO and Founder, MikeWebbPharma Ltd
Mike Webb PhD was the VP of Development Chemistry and Analysis for GSK in the UK and was heavily involved in developing GSK’s oligonucleotide pipeline. Since leaving GSK in 2016 he has been consulting with Big Pharma and Biotechnology companies in the development, analysis and manufacture of therapeutic oligonucleotides from pre-clinical to marketing submission.
Workshop agenda
Key CMC development milestones for oligonucleotide therapeutics –Complex and emerging modalities / manufacturing platforms
Review of key CMC concepts for oligonucleotide therapeutics
CMC challenges and successes for the platforms that have already progressed to product approval
Review of the ‘next generation’ manufacturing platforms and use of oligonucleotides in complex and emerging modalities
Leveraging CMC synergies between ‘legacy oligonucleotides’ and complex /emerging modalities and acknowledging the new frontiers in the CMC development path
Addressing impurities and their control
ICH Impurity guidelines
Oligonucleotide Impurities: ID and control strategies, and Formation and fate
We thought the analytics for 20’mers and siRNA were complex. When you multiply the chain length by 5 – and / or conjugate the oligonucleotide, what is the multiplier for the complexity of impurity control?
Mass spec-based impurity method vs. orthogonal chromatography methods, or all of above?
Connecting Module 3 and Module 2 in demonstrating impurity control
Attending to Regulatory expectations by phase of development
Planning for the end goal – NDA vs. BLA – oligonucleotide manufacturing platform
Pre-IND and Phase 1
Phase 2/3
Late stage, pre-marketing
Key Takeaways and Discussion
Delegates will have the chance to discuss and benchmark concepts and challenges of oligonucleotide development and CMC based on learnings from first 3 sessions
Outlook into the future of oligonucleotide therapeutic development
CEO and Founder
MikeWebbPharma Ltd
CEO and Founder, MikeWebbPharma Ltd
Mike Webb PhD was the VP of Development Chemistry and Analysis for GSK in the UK and was heavily involved in developing GSK’s oligonucleotide pipeline. Since leaving GSK in 2016 he has been consulting with Big Pharma and Biotechnology companies in the development, analysis and manufacture of therapeutic oligonucleotides from pre-clinical to marketing submission.
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