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Certain next-gen vaccines and gene therapy applications rely on the usage of adeno-associated viruses (AAV) as a delivery vehicle. To ensure the safety and efficacy of viral vector drugs, multiple critical quality attributes (CQAs) need to be well characterized.
Lipid nanoparticles (LNPs) are widely used vehicles for mRNA-based therapeutics and vaccines. However, ionizable lipids used in LNPs can be susceptible to N-oxide impurities that can cause functional loss of the mRNA cargo.
Currently, there are 3 main types of in vitro transcribed (IVT) RNA drugs. Two of these—conventional messenger RNA (mRNA) and base-modified mRNA (bmRNA), which incorporates chemically modified nucleotides—are non-replicating. The third type is self-replicating RNA (srRNA), which is based on an engineered viral genome but devoid of viral structural protein genes. Its self-replicating ability makes srRNA a promising tool for new therapeutic drugs.
Lipid-based nanoparticles (LNPs) are effective non-viral vectors for delivering messenger RNA (mRNA) products, most notably used for production of vaccines against the recent SARS-CoV-2 pandemic.
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