Pharmicell announced on the 16th that its project, "Development of a Commercial Manufacturing Process for Locked Nucleic Acid (LNA) Monomers Applied to Gene Therapy," has been selected for the 2023 Regional Key Industry Promotion Project organized by the Small and Medium Business Technology Information Promotion Agency.
Recently, interest in gene therapy has increased following the development of mRNA vaccines amid the COVID-19 pandemic. Clinical research for new therapeutic development is actively underway. Gene therapy involves short-stranded oligonucleotide therapeutics that target RNA, which receives genetic information from DNA containing genetic data and is involved in protein synthesis. Oligonucleotides work by binding complementarily to RNA, preventing RNA from participating in protein synthesis, thereby enhancing therapeutic effects.
Oligonucleotides composed of natural nucleosides have the disadvantage of being easily degraded in the body. To improve this, active attempts are being made to develop gene therapies composed of modified nucleosides. Oligonucleotides made of locked nucleic acid RNA have the advantages of enhanced target binding affinity and increased stability in the body, which boosts therapeutic efficacy.
A Pharmicell representative explained, "Locked nucleic acids are actively researched not only in antisense oligonucleotide (ASO) therapeutics but also in RT-PCR, siRNA therapeutics, and miRNA therapeutics." He added, "They can be applied in all fields where DNA and RNA are used, so the utility value of locked nucleic acid monomers is significant," and stated, "Through this project, we will expand the supply capacity of raw materials for oligonucleotide manufacturing."
He also said, "We will strengthen our position as a leading supplier of raw materials in the growing gene therapy market."
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