Europe – Promising mRNA tech comes with regulatory, CMC headaches

As the buzz builds around messenger RNA (mRNA) technology’s use for two leading COVID-19 candidates, manufacturers and regulatory professionals are facing facts: This is not simple technology.

Complex manufacturing processes, delivery vehicles that must be treated more as drug substances than excipients, and potential immunogenicity headaches are among the challenges industry faces as this promising technology is harnessed to address an increasing number of health conditions.

At October’s virtual Euro Convergence conference, Tracy Meffen, RAC, vice president for quality and regulatory affairs at Genevent Sciences Corporation, walked attendees through the basics of the technology, placing focus on chemistry, manufacturing and control (CMC) considerations from a regulatory affairs perspective.

Two types of RNA therapeutics work in different ways to create opposing effects, explained Meffen. By carrying genetic information in single-stranded RNA that enables protein synthesis, mRNA therapeutics up-regulate proteins that are faulty or missing. In contrast, the double-stranded RNA strands of siRNA therapeutics degrade mRNA after transcription, thereby preventing translation and eliminating excessive proteins that cause disease because they are faulty or overabundant…