This invention generally relates to the field of phosphoramidite derivatives. In particular, the invention relates to N- Acetylgalactosamine (GalNAc) compounds, in particular phosphoramidite or phosphonoamidite molecules of formula (I) with only one GalNAc moiety (formula II) and to conjugates of nucleic acid molecules with such N-Acetylgalactosamine containing molecules. Also provided are methods for preparation of these molecules and possible uses thereof, in particular in medicine. Wherein R1 is H or C1-6alkyl; R2 is a triphenylmethyl-based hydroxyl protecting group R3 is a phosphorus-containing group, particularly a phosphoramidite or a phosphonoamidite group, and K is represented by the general formula (II).
本发明一般涉及
磷酰胺衍
生物领域。特别地,本发明涉及N-乙酰半
乳糖胺(GalNAc)化合物,特别是公式(I)的
磷酰胺或
磷酸酰胺分子,仅具有一个GalNAc基团(公式II),以及含有这种N-乙酰半
乳糖胺分子的核酸分子结合物。还提供了制备这些分子的方法及其可能的用途,特别是在医学上。其中,R1为H或C1-6烷基;R2为基于三苯甲基的羟基保护基;R3为含
磷基团,特别是
磷酰胺或
磷酸酰胺基团;K由通式(II)表示。