[Asia Economy Reporter Hyunseok Yoo] Naivek, a peptide fusion bio-specialized company, announced on the 24th that it has completed the development of 'oligonucleotide' delivery and application technology broadly applicable to mRNA (messenger ribonucleic acid) and siRNA (small interfering ribonucleic acid) and has filed a patent. Oligonucleotides refer to 'synthetic DNA and RNA molecules' used in genetic testing and drug development.
mRNA and siRNA are being developed as therapeutics to control genes that cause cancer, autoimmune diseases, and infectious diseases. Recently, mRNA-based vaccines that induce immune responses by injecting genes coding for the spike protein of the COVID-19 virus in the form of mRNA received FDA approval, drawing global pharmaceutical companies' attention to the field of mRNA application technology.
Until now, mRNA and siRNA were rapidly degraded by 'nucleases' upon entering the human body, preventing them from targeting and reaching the desired therapeutic sites. Even if they reached the target site, they needed to penetrate cells and act internally to be effective, but mRNA and siRNA have large molecular sizes and cannot permeate cell membranes, resulting in limited efficacy.
To overcome these drawbacks, lipid nanoparticles (LNPs) have mainly been used for siRNA and mRNA delivery. However, LNPs have been criticized for causing hepatotoxicity when used in high doses and for low delivery efficiency to therapeutic sites.
Naivek succeeded in solving all the problems of LNPs through the 'gene drug delivery system applied with NIPEP-TPP' technology for which it recently filed a patent. Naivek already had a NIPEP-TPP-based delivery system for delivering siRNA for tumor treatment purposes and held patents for it. The company explained that the newly introduced compound for mRNA delivery was proven effective in producing therapeutic effects in tumors, leading to an additional new patent application.
A Naivek official said, "The NIPEP-TPP technology for mRNA delivery additionally introduced a 'hydrophobic part' and an 'RNA-binding part' that were not present in the siRNA delivery design," adding, "As a result, the NIPEP-TPP for mRNA delivery has the technology to stably bind and compress the larger mRNA compared to LNPs, maintaining stable nanoparticles over a long period."
He continued, "When mRNA-containing NIPEP-TPP transporter nanoparticles were applied to cells, intracellular delivery of mRNA significantly increased, and it was verified that the expression of proteins coded by the increased mRNA also rose," stating, "This means that therapeutic substances can permeate inside cells." He added, "NIPEP-TPP has targeting functions, allowing it to accurately reach lesions such as desired cancer tissues and permeate therapeutic substances inside cells, resulting in no side effects and excellent efficacy. Currently, large-scale production process development is also underway."
Since NIPEP-TPP has already demonstrated safety in toxicity tests, the company explained that it does not have problems such as hepatotoxicity caused by LNPs or anaphylactic and allergic reactions caused by polyethylene glycol (PEG) introduced in LNPs.
A Naivek official emphasized, "Research and development related to gene delivery systems have become more active since the JP Morgan research presentation in January this year, which attracted global pharmaceutical companies' attention. Through this patent application, we can externally demonstrate Naivek's research and development capabilities related to gene drug delivery," adding, "We are discussing joint research with global pharmaceutical and biotech companies and expect visible outcomes such as technology transfer."
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