Liver-bound

Ionis Pharmaceuticals Inc. is retooling its antisense technology with a sugar-based conjugate that boosts uptake into liver cells. By tapping the power of GalNAc sugars to latch onto hepatocyte receptors, the company believes it can increase the activity of its liver-targeting portfolio. Next, it plans to find new targeting handles to extend the strategy to other tissues.

Last month, the company published details of the new technology in the Journal of Medicinal Chemistry. The Ionis team screened N-acetylgalactosamine (GalNAc) conjugates with a variety of scaffold, spacer and ASO linker chemistries, and selected THA-GN3 to promote tissue-specific delivery by linking it to the 5' end of liver-targeted ASOs. ...