The workshop will give an overview of manufacturing processes and CMC strategies for long and complex oligonucleotides, like sgRNA or aptamers. It will emphasize the CMC development from early discovery phase throughout the clinical phases and spotlight on cleaning/carryover, process development, analytical development, raw materials selection and control, impurity characterization, analytical tools and setting of specifications. The workshop will be split into a manufacturing-focused part ranging from early process development to scale-up, and an analytical part addressing process-, and product-related impurity formation and their control. After the presentations the speakers will jointly chair a panel discussion on CMC aspects.

Topics to be Discussed:

• CMC activities for long and complex oligonucleotide therapeutics or theranostics
• Raw material selection and control
• Understanding and controlling process and product-related impurities
• Analytical tools and their application

Who Should Attend?

Anyone interested in CMC, Impurity Control Strategies, and in-house or outsourced manufacturing of oligonucleotide therapeutics during pre-clinical and clinical development of synthetic RNA Therapeutics or Theranostics. This includes R&D Researchers, Manufacturing Personnel, Quality Assurance, Project Management.

Methylated nucleotides are known to shape structures and functions of natural and therapeutic RNAs. We discovered the catalytic potential of RNA as methyltransferase ribozyme and continue to develop novel ribozymes for posttranscriptional installation of site-specific RNA modifications. Combining guide and enzyme in one molecule of functional RNA, RNA-alkylating ribozymes find synthetic applications in vitro and are promising for modifying intracellular targets.

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Since the early clinical findings of using glucagon like peptide-1 (GLP-1) to regulate blood glucose levels in the late 80´s there has been an increasing interest to discover and develop GLP-1 receptor agonists for treatment of metabolic disorders. The increased non-clinical and clinical understanding of the mechanism of action of GLP-1 and the parallel technical development have now brought the scientific community to a very high level of understanding with structural insight of the interaction of peptides and small molecules with GPCR class B receptors. The discovery of semaglutide is a fantastic example on how technology and biologic understanding have developed in parallel ending with a superior peptide for treatment of diabetes and obesity.

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