Pamicell Advances Commercialization of New Raw Materials for Gene Therapy Drugs

Pharmicell announced on the 1st that it has confirmed the feasibility of manufacturing a new nucleoside used as a raw material for oligonucleotide synthesis in gene therapy drugs and is promoting its commercialization.

The target substance for commercialization is DMT-2’-O-TBDMS-RNA, which is used in the synthesis of oligonucleotides for antisense therapeutics and siRNA therapeutics. Oligonucleotides contribute to enhanced therapeutic effects by preventing RNA from participating in protein synthesis.

For the synthesis of oligonucleotides in the pharmaceutical RNA category, nucleosides protected by groups such as TBDMS (tert-Butyldimethylsilyl) or TOM (triisopropylsilyloxymethyl) are required. Nucleosides protected by the TBDMS group are the most essential structure, but due to difficulties in processing and purification, only a very small number of companies worldwide can supply them in large quantities.

With the advancement of gene editing technology, the demand for long-chain oligonucleotides composed of more than 80 nucleotides is increasing. Existing protecting groups cause issues such as low yield and extended synthesis time in the synthesis of long-chain oligonucleotides, leading to the demand for nucleosides protected by TOM and CEM (cyanoethoxymethyl) groups. Pharmicell is currently conducting joint research with a major US global pharmaceutical company regarding the manufacturing of nucleosides with protecting groups.

A Pharmicell representative explained, "Recently, research on nucleic acid drugs has been actively progressing, with the launch of Rekvio, a next-generation siRNA-based gene therapy for dyslipidemia, and the initiation of approval procedures for Imetelstat, the first ribonucleic acid (RNA) anticancer drug as a telomerase inhibitor." He added, "The demand for nucleosides as basic materials for manufacturing gene therapy drugs and nucleic acid pharmaceuticals is increasing."

He also stated, "Pharmicell possesses a wide range of protected DNAs/RNAs nucleoside products," and added, "We plan to expand our product lineup and increase sales in response to supply requests from global pharmaceutical companies such as Thermo Fisher Scientific and Merck."

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