Workshop Overview:

This workshop will address early drug development and CMC of oligonucleotide therapeutics. Preparing for IND/IMPD is a critical and strategic step for successful start of clinical development. A detailed discussion of moving oligonucleotide therapeutics from discovery to clinical trials will include a description of CMC strategies for early clinical development, process development, raw materials selection and control, GMP synthesis, analytical controls and specifications. The workshop will be split into a chemistry part from early synthesis to initial scale-up, addressing impurities and their control and a regulatory part addressing what is needed for the preparation of IND-IMPD dossiers based on existing guidelines. Participants will gain a basic understanding of the considerations and requirements for taking an oligonucleotide therapeutic into first-in-human clinical trials. After the presentations the speakers will jointly chair a panel discussion on CMC aspects.

Topics to be covered and format:

• 2 presentations, 45-minutes each followed by a 15-minute open discussion
• 30 - 60-minute panel discussion on CMC aspects
• CMC activities for complex and emerging oligonucleotide therapeutics
• Impurities and their control
• Preparation of an IND and regulatory aspects

Who should attend?

Anyone interested in development of complex oligonucleotide therapeutics; Anyone interested in outsourcing the manufacturing of oligonucleotide therapeutics to a CMO / CRO. This includes R&D Researchers, Manufacturing Personnel, Quality Assurance, Project Management, Business Development and Scientific Management