Investigations into mRNA Lipid Nanoparticles Shelf-Life Stability under Nonfrozen Conditions

Anne-Gaëlle Reinhart1, Anja Osterwald2, Philippe Ringler3

  • 1Roche Pharma Research and Early Development, Therapeutic Modalities, pCMC, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd., Grenzacherstrasse 124, Basel 4070, Switzerland.

Molecular Pharmaceutics
|November 17, 2023
PubMed

Insights

mRNA lipid nanoparticles (LNPs) can maintain activity without freezing for extended periods. This study found specific LNPs, like C12-200, retain functionality at room temperature, easing storage and transport challenges for mRNA technology.

Area of Science:

  • Nanotechnology
  • Biotechnology
  • Pharmaceutical Sciences

Background:

  • mRNA lipid nanoparticles (LNPs) are crucial for drug and vaccine delivery.
  • Current mRNA LNP formulations require frozen storage, posing logistical and cost challenges.
  • Developing stable, non-frozen storage conditions is essential for broader mRNA LNP applications.

Purpose of the Study:

  • To assess the stability and activity of mRNA LNPs under non-frozen storage conditions.
  • To identify mRNA LNP formulations that maintain functionality at ambient temperatures.
  • To understand the structure-activity relationships influencing LNP stability.

Main Methods:

  • Synthesized five structurally diverse mRNA LNPs using microfluidic technology with varying ionizable lipids (C12-200, CKK-E12, MC3, SM-102, lipid 23).
  • Evaluated shelf-life stability over 9 weeks at 2-8°C, 25°C, and 40°C using analytical techniques (hydrodynamic diameter, zeta potential, encapsulation efficiency, polydispersity).
  • Assessed in vitro activity via EGFP protein expression in HEK293 cells and analyzed LNP architecture using cryo-EM.

Main Results:

  • Most LNPs showed minimal changes in physical characteristics (size, charge, encapsulation) during non-frozen storage.
  • RiboGreen assays suggested consistent mRNA content, but in vitro activity varied significantly.
  • LNP 1 (C12-200) and LNP 4 (SM-102) showed high initial transfection efficiency.
  • LNP 1 (C12-200) maintained activity for 11 weeks at 2-8°C and 25°C.
  • LNP 4 (SM-102) retained activity at 2-8°C but declined at 25°C over 11 weeks.

Conclusions:

  • Specific mRNA LNP formulations, particularly those with C12-200 lipid, demonstrate potential for non-frozen storage and transport.
  • In vitro activity assays are crucial for assessing LNP functionality, as physical stability metrics can be misleading.
  • Understanding LNP architecture and structure-activity relationships is key to overcoming storage limitations and expanding mRNA LNP applications beyond vaccines.

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