Messenger RNA (mRNA) is pioneering the development of an entirely new class of drugs which shows much promise in biotherapeutics. This novel drug platform builds on the discovery that modified mRNA can direct the body’s cellular machinery to produce nearly any protein of interest. Once delivered, modified mRNA can direct ribosomes to express proteins or antibodies within targeted tissues, thereby harnessing the body’s own cellular machinery to produce the broadest possible array of therapeutics across diverse therapeutic areas and across numerous routes of administration for serious diseases that are not treatable today. In order for mRNA to reach the cellular apparatus, it must be protected from degradation mechanisms that rapidly inactivate mRNA, such as enzymatic degradation by RNases that are ubiquitous in the environment and body. To overcome these degradation pathways, we have chosen to physically encapsulate the mRNA in nanoparticles so that the mRNA payload can safely reach its cellular target. The overall structure of these nanoparticles is obtained through the simultaneous model fitting of small angle neutron scattering (SANS) data collected by varying neutron scattering length density (NSLD) of the solvent (e.g. different ratios of H<sub>2</sub>O to D<sub>2</sub>O). In addition, small angle x-ray scattering (SAXS) further validates the SANS results, providing insights into the mRNA structure within the nanoparticle.

Determining the mRNA nanoparticle structure using SANS and SAXS

253rd National Meeting (2017)

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