Pharmicell Secures Patent for Large-Scale, High-Purity Production Method of Key Gene Therapy Raw Material

Pharmicell announced on November 26 that it has developed a technology capable of producing large quantities of high-purity '2'-deoxy-2'-fluorouridine', a key intermediate for gene therapy raw materials, and has obtained a patent for this technology.

2'-F-dU (2'-fluoro-2'-deoxyuridine) is one of the 2'-fluoro-RNA derivatives, a compound in which the alcohol group at the 2' position of an RNA nucleoside is substituted with fluorine. It is widely used as a core raw material for RNAi-based therapeutics because it is incorporated into oligonucleotides and increases in vivo stability.

The cholesterol-lowering drug Leqvio (ingredient name: Inclisiran), which has received approval from the United States Food and Drug Administration (FDA), and the hereditary transthyretin-mediated amyloidosis treatment Amvuttra (ingredient name: Vutrisiran) both utilize 2'-F-RNA nucleosides.

As RNAi-based therapeutics are emerging as a new alternative for genetic and intractable diseases that have been difficult to treat with conventional drugs, demand for these therapies is expected to increase further in the future.

Pharmicell completed the development of this new technology in 2023, which improves process efficiency and productivity, replacing existing technologies that faced commercialization challenges due to limitations in mass production. The company has now obtained a patent for this technology.

A Pharmicell representative stated, "With this patent, we have secured both production efficiency and quality competitiveness. Going forward, we will expand our nucleoside product lineup and strengthen a stable supply base for raw materials in line with the growth of the global RNAi therapeutics market."

This content was produced with the assistance of AI translation services.