On-demand Session: CMC Considerations for mRNA based Therapeutics

uRNA: has a strong intrinsic adjuvant effect, with strong antibody response, generating CD8+T cells CD4+T cells;
modRNA: has moderate intrinsic adjuvant effect, with a very strong antibody response and lower cytokine induction, generating CD4+T cells CD8+T cells;
saRNA: has a long duration, a higher likelihood for good efficacy antibody response even when using a lower dosage.

by Jo-Anne Riley, Senior Scientist, Oligonucleotide Services, Intertek Pharmaceutical Services

CMC Considerations for mRNA based Therapeutics

Messenger ribonucleic acid (mRNA) has the potential to treat a wide range of human diseases by exploiting natural in vivo protein synthesis machinery. With advancement of mRNA based therapeutics through clinical phases comes the requirement for mRNA characterisation and establishment of GMP validated analytical methods for quality control.Testing can include measuring the size or molecular weight of the mRNA, characterisation of the 5′ cap structure and poly (A) tail, as well as tests for impurities which can be directly linked to translation efficiency and immunogenicity.Analysis of the associated delivery systems, such as lipid nanoparticles, also forms a key part of mRNA drug product testing.This presentation by Jo-Anne Riley, Senior Analyst at Intertek Pharmaceutical Services, discusses analytical approaches to characterise mRNA in early-stage development to support effective therapeutic programs.

Find out more at intertek.com/pharmaceutical.