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Published on: February 1, 2019
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.
Abstract:
mRNA LNPs can experience a decline in activity over short periods (ranging from weeks to months). As a result, they require frozen storage and transportation conditions to maintain their full functionality when utilized. Currently approved commercially available mRNA LNP vaccines also necessitate frozen storage and supply chain management. Overcoming this significant inconvenience in the future is crucial to reducing unnecessary costs and challenges associated with storage and transport. In this study, our objective was to illuminate the potential time frame for nonfrozen storage and transportation conditions of mRNA LNPs without compromising their activity. To achieve this goal, we conducted a stability assessment and an in vitro cell culture delivery study involving five mRNA LNPs. These LNPs were constructed by using a standard formulation similar to that employed in the three commercially available LNP formulations. Among these formulations, we selected five structurally diverse ionizable lipids─C12-200, CKK-E12, MC3, SM-102, and lipid 23─from the existing literature. We incorporated these lipids into a standard LNP formulation, keeping all other components identical. The LNPs, carrying mRNA payloads, were synthesized by using microfluidic mixing technology. We evaluated the shelf life stability of these LNPs over a span of 9 weeks at temperatures of 2-8, 25, and 40 °C, utilizing an array of analytical techniques. Our findings indicated minimal impact on the hydrodynamic diameter, zeta potential, encapsulation efficiency, and polydispersity of all LNPs across the various temperatures over the studied period. The RiboGreen assay analysis of LNPs showed consistent mRNA contents over several weeks at various nonfrozen storage temperatures, leading to the incorrect assumption of intact and functional LNPs. This misunderstanding was rectified by the significant differences observed in EGFP protein expression in an in vitro cell culture (using HEK293 cells) across the five LNPs. Specifically, only LNP 1 (C12-200) and LNP 4 (SM-102) exhibited high levels of EGFP expression at the start (T0), with over 90% of HEK293 cells transfected and mean fluorescence intensity (MFI) levels exceeding 1. Interestingly, LNP 1 (C12-200) maintained largely unchanged levels of in vitro activity over 11 weeks when stored at both 2-8 and 25 °C. In contrast, LNP 4 (SM-102) retained its functionality when stored at 2-8 °C over 11 weeks but experienced a gradual decline of in vitro activity when stored at room temperature over the same period. Importantly, we observed distinct LNP architectures for the five formulations through cryo-EM imaging. This highlights the necessity for a deeper comprehension of structure-activity relationships within these complex nanoparticle structures. Enhancing our understanding in this regard is vital for overcoming storage and stability limitations, ultimately facilitating the broader application of this technology beyond vaccines.
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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