Standard Guide for Characterization of Encapsulation, Extraction, and Analysis of RNA in Lipid Nanoparticle Formulations for Drug Delivery

Importancia y uso:

5.1 Nucleic acid therapeutics based on the utilization of mRNA or modifications thereof are increasingly important for enabling the production of therapeutic proteins, vaccines, genome editing modalities, etc. To establish the safety, efficacy, quality and regulatory status of mRNA-based therapeutics, it is essential to have robust and reproducible methods and protocols for (1) performing the extraction, (2) determining the identity and purity, and (3) characterizing the encapsulation efficiency of mRNA extracted from selected delivery vehicles. In this guide, qualitative and quantitative methods for obtaining these mRNA quality attributes are described in reference to mRNA encapsulated in LNP delivery vehicles.

5.2 This guide is intended principally for determining selected quality attributes of mRNAs extracted from LNP drug delivery formulations. As such, measurement guidance will be helpful to RNA suppliers, RNA therapy manufacturers, and industry regulators of RNA-LNP therapeutics.

Subcomité:

E56.08

Volúmen:

14.02

Palabras clave:

lipid nanoparticle (LNP); ribonucleic acid (RNA); RNA therapies;