South Korea based OliX Pharmaceuticals, Inc., a leading developer of RNAi therapeutics, has announced that the Company has signed an exclusive worldwide licensing agreement with AM Chemicals (AMC) for the rights to patents and know-how encompassing the technology to conjugate N-acetylgalactosamine (GalNAc) to OliX' asymmetric small interfering RNA (asiRNA) molecules for liver targeting.  

Under the agreement, AMC will supply a selection of GalNAc-modified phosphoramidites and solid supports to OliX Pharmaceuticals. AMC will also provide support for RNA synthesis, for which AMC is entitled to receive upfront milestone and royalty payments.

Small interfering RNA (siRNA) is a short, double-stranded RNA molecule that silences expression of genes with sequence complementarity based upon a RNA interference (RNAi) mechanism. Since the FDA-approval of the first siRNA therapeutics, Patisiran, in 2018, a number of siRNA therapeutic candidates are currently in various stages of development across multiple disease indications. Targeted delivery of siRNAs to hepatocytes has been achieved by direct conjugation of GalNAc moiety, for receptor-mediated endocytosis via the asialoglycoprotein receptor (ASGPR) that is primarily expressed on the surface of hepatocytes, allowing inhibition of disease-causing genes in the liver. Recently, FDA approved Givosiran, the GalNAc-conjugated siRNA for acute hepatic porphyria. 

"We are excited to employ AMC's GalNAc conjugate technology," said Dong Ki Lee, Ph.D., founder and Chief Executive Officer of OliX. "Liver-targeting strategy using GalNAc conjugation has become the standard in the RNAi industry. We believe that the combination of AMC's GalNAc-linker technology with OliX' proprietary asymmetric siRNA (asiRNA) will greatly accelerate the development of effective targeted therapy for various liver diseases with high unmet medical needs."