Manufacturing and Bioprocessing – Biomolecular

2019 PharmSci 360

Delivering RNA for Therapeutic Applications

Speaker(s)

• 

  Matthias Kretschmer, PhD

  Alnylam Pharmaceuticals
  Cambridge, Massachusetts

Control of impurities for siRNA Therapeutics

This presentation will explain the general approach for control of impurities for siRNA therapeutics.
Control of impurities for Onpattro, the first approved siRNA therapeutic, follows a tiered approach using manufacturing stage appropriate methods designed for individual single strands, siRNA (duplex) drug substance and final (lipid nano particle) drug product. Based on the quality attributes tested, methods are of orthogonal nature and asses impurities both as specified groups as well as individually.

Learning Objectives:

• Understand how physical and chemical properties of RNA-based therapeutics are analytically defined and controlled.
• Gain insight into unique aspects of RNA-based therapeutics.
• Understand the specific technical challenges that have to be overcome to routinely test RNA-based therapeutics.
• Learn from examples how a tiered approach is used to assure safety, purity and efficacy of the new therapeutic modality.
• Upon completion, the attendees will come away with a holistic view of the field of RNA-based therapeutics from the analytical/CMC scientists perspective.