主权项 |
1. A pharmaceutical composition comprising (i) one or more pharmaceutically acceptable adjuvants, diluents, carriers and/or excipients and (ii) an RNA modified with a 5′-cap structure according to formula (I): wherein R1 is selected from the group consisting of optionally substituted alkyl, optionally substituted alkenyl, optionally substituted alkynyl, optionally substituted cycloalkyl, optionally substituted heterocyclyl, optionally substituted aryl, and optionally substituted heteroaryl, R2 and R3 are independently selected from the group consisting of H, halo, OH, and optionally substituted alkoxy, or R2 and R3 together form O—X—O, wherein X is selected from the group consisting of optionally substituted CH2, CH2CH2, CH2CH2CH2, CH2CH(CH3), and C(CH3)2, or R2 is combined with the valence produced by removal of the hydrogen atom at position 4′ of the ring to which R2 is attached, wherein the bridging linkage from R2 to the 4′ position is defined as an —O—CH2— group or —CH2—O— group, R5 is selected from the group consisting of S, Se, and BH3, R4 and R6 are independently selected from the group consisting of O, S, Se, and BH3, n is 1, 2, or 3, wherein the stereochemical configuration at the P atom comprising the substituent R5 corresponds to that at the Pβ atom of the D1 diastereomer of beta-S-ARCA: |