Table 1.
Tominersen | WVE-120101 and WVE-120102 | |
---|---|---|
Backbone chemistry | Phosphorothioate (PS) and Phosphodiester (PO) linkages | Phosphorothioate (PS) and Phosphodiester (PO) linkages |
Wing chemistry | 2′-O-methoxyethyl (2′-MOE) | 2′-O-methyl (2′-O-Me) |
Sequence | ctocoaogTAACATTGACaococoac |
WVE-120101 ggocoaocAAGGGCACAGaocououc WVE-120102 guogocoaCACAGTAGATgoaogogg |
Stereochemistry | Stereorandom | Stereopure |
Number of isomers | 8192 | 1 |
Selectivity | Nonselective | Selective: targets CAG expansion associated SNPs |
Population coverage | 100% |
WVE-120101: 20–56% WVE-120102: 24–51% Combinatorial use: 36–70% [32] |
Lower-case letters indicate ribose nucleosides with 2′ sugar modifications; upper-case letters indicate deoxyribose nucleosides; O indicates phosphodiester (PO) linkage; non-labeled linkages indicate chiral phosphorothioate (PS) linkages; underlined letters indicate single nucleotide polymorphism (SNP)