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mRNA Therapeutics – Analytical and Bioanalytical Characterization
Tuesday, March 09, 2021: 9:40 AM - 10:15 AM
Speaker(s)
Description
The modality landscape has expanded to find medicines for targets that are undruggble with small molecules or protein therapeutics. Nucleotide-based therapeutics are popular, with several recent approvals and many candidates in pharmaceutical pipelines for modulating disease associated proteins. Protein over-production is druggable by small interfering RNA (siRNA) and antisense oligonucleotides (ASO), and under-production can be treated using messenger RNA (mRNA). Nucleotide-based therapeutics pose new challenges for formulation development and analytical characterization. The molecules are large, charged, and usually require a delivery system to transfect cells and, escape the endosomal pathway to achieve cytosolic bioavailability (e.g., mRNA, siRNA) or access to the cell nucleus (e.g., ASOs). As a result, there is a need in drug discovery to characterize the molecule and the delivery system in cells in order to understand the mechanism of action and delivery system performance. This produces new analytical and automation challenges as high-throughput systems would have to be developed to design, make, test and analyze drug delivery systems. The unique nature of the molecules and formulations also creates analytical challenges associated with chemistry, manufacturing and control in early development. These new modalities give rise to distinct impurities and degradation products, which necessitates the development and validation of complex analytical methods for quality control (QC) purposes. Finally, regulatory standards for this therapeutic field are still developing as lessons are learned from clinical development and the relatively few therapies which have received commercial approval. This presentation will look at nucleotide-based therapeutics case studies, analytical and bioanalytical characterization from late discovery (i.e., focus on tracking the therapeutic and the delivery system in cells) and, early development (i.e., focus on QC method development).
Additional Info
Keywords: Please select up to 4 keywords ONLY:
Automation,Biopharmaceutical,Biotechnology,Gene Therapy
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