Category | Advanced therapy
Although the COVID-19 pandemic spurred the rise of mRNA technology thanks to its pivotal role in the development of Pfizer-BioNTech’s and Moderna’s vaccines, researchers have been studying mRNA’s potential for almost 30 years. Indeed, the versatility and programmability of mRNA molecules make them attractive candidates for a variety of therapeutic interventions, including for infectious diseases, cancer immunotherapy, genetic disorders, autoimmune disorders, and rare diseases. A potential concern with naked unprotected mRNA is poor cellular penetration due to the large molecule size, negative charge, and rapid degradation qualities. Therefore, to help maximize efficacy, mRNA molecules can be encapsulated in different carriers/vectors to enhance their delivery into target cells.
Examples of such carriers/vectors are lipid nanoparticles (LNPs); additionally, polymeric nanoparticles (PNPs) can be used. Although LNPs are the industry’s most commonly used and well-studied vehicle for mRNA delivery, they’re still subject to certain challenges, such as limited cargo capacity, batch-to-batch variability, biological interactions, off-target effects, potential toxicity, and more. Additionally, mRNA-LNPs need to be stored frozen at −20°C or −80°C, which poses a challenge for transportation and long-term storage.1 What’s more, the intellectual property (IP) landscape around specific lipids is increasingly complex to navigate; indeed, more than 5,600 IP publications related to LNPs in drug delivery have been published.2
Considering those limitations, researchers are exploring alternative delivery methods, including retrovirus-like protein PEG10, biological membrane-based vehicles, organ or cell-specific mRNA delivery, and inhaled, intranasal, or oral mRNA. To help evaluate the options for mRNA encapsulation, many biotechnology and pharmaceutical companies rely on CDMOs with end-to-end mRNA experience — specifically with encapsulation. At Thermo Fisher Scientific, we can support your mRNA encapsulation with commercially available lipids and formulations, or work with your team using your own IP-specific lipid.
Watch our recent webinar to learn how we stay up to date with industry trends and approach mRNA encapsulation.