Analytical Considerations For mRNA-Based Therapies

By Mark Rogers, Ph.D. - Global Scientific Director, SGS Health & Nutrition

Embark on a journey into the revolutionary realm of mRNA-based therapies poised to transform medicine. Since its discovery in 1961, mRNA technology has made remarkable strides, culminating in the USFDA's approval of the first mRNA product. Overcoming hurdles like stability and delivery, mRNA sequence modifications have unlocked three key applications: prophylactic vaccines, therapeutic vaccines, and therapeutic drugs.

The production of mRNA drug substance entails an innovative in vitro chemoenzymatic process, while delivery challenges have been surmounted with Lipid Nanoparticles (LNP) integration. Upholding quality and safety standards, organizations adhere to guidelines covering attributes such as purity, potency, and identity. Purity assessment relies on sensitive assays, potency is gauged through functional integrity tests, and quality is ensured via standard methodologies. Furthermore, particle analysis holds paramount importance for injectables.

Accurate molecular identity confirmation of mRNA therapeutics, albeit complex, is facilitated by techniques like Sanger sequencing, RT-PCR, and high throughput sequencing for sequence confirmation, and capillary gel, capillary electrophoresis, and mass spectrometry for size determination. Safety testing encompasses bioburden, endotoxin, and sterility assessments, with ongoing enhancements in methodologies.

To propel RNA-based therapies forward, rigorous analytical scrutiny is indispensable, with machine learning and innovative technologies poised to play pivotal roles. Learn more by accessing the full article below.