Prisys Biotechnology specializes in non-human primate (NHP) research and translational studies. Developing lipid nanoparticles (LNPs) for the efficient and safe delivery of mRNA therapeutics to different tissues and organs remains a significant challenge. One key challenge in mRNA delivery is translating preclinical findings from rodent models to non-human primates (NHPs), which share greater physiological and pharmacokinetic similarities with humans.
This article introduces two studies that demonstrate approaches for optimizing LNPs for mRNA delivery in NHP models and evaluating their translational potential.
In the first paper, Researchers showed that the same LNPs loaded with mRNA encoding human ornithine transcarbamylase (hOTC), a liver enzyme involved in urea cycle, had significantly lower protein expression levels in the livers of NHPs than in mice, indicating a species-dependent difference in LNP activity. They further investigated the factors that affect the LNP performance in different species, focusing on LNP size and polyethylene glycol (PEG) lipid content.
They found that smaller LNPs had higher protein expression levels in NHP livers than larger LNPs, which was opposite to the trend observed in mice. They also found that the optimal PEG lipid content for small LNPs in NHPs was 2.2-2.8%, which was higher than the typical value used for mice. By adjusting the LNP size and PEG lipid content, they were able to increase the protein expression levels in NHP livers by 8-fold compared to the original LNPs.
In the second paper, Researchers applied their optimized LNPs to deliver mRNA encoding human factor IX (hFIX), a blood clotting factor, to NHPs. They achieved sustained and therapeutic levels of hFIX expression in the plasma of NHPs for over 6 months, with no signs of toxicity or immunogenicity. This is the first report of long-term mRNA expression in NHPs using LNPs, and it demonstrates the potential of Prisys Biotechnology's LNPs for treating hemophilia and other genetic diseases.
By providing preclinical CRO services based on non-human primates, Prisys Biotechnology is committed to the field of mRNA therapeutics and bringing novel mRNA drugs to the market. By optimizing LNPs for delivery with Prisys Biotech's NHPs, researchers have overcome a major hurdle in the preclinical development of mRNA drugs and paved the way for clinical trials. They are currently conducting several clinical studies using its LNPs for various indications. Prisys Biotechnology is also collaborating with academic and industry partners to explore new applications and opportunities of NHPs for mRNA therapeutics.
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