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US 2022/0025370 A1Published application
Oligomer-conjugate complexes and their use
AssigneeIonis Pharmaceuticals, Inc.
InventorsThazha P. Prakash, Eric E. Swayze, Walter F. Lima, Garth A. Kinberger
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Abstract
Oligonucleotides, chemically-modified oligonucleotides, and oligonucleotide-conjugate complexes for use in research, diagnostics, and/or therapeutics are described herein. In some embodiments, oligonucleotides comprising a stabilized phosphate moiety covalently attached to the 5′-terminal nucleoside are provided.
Claims22
-529. (canceled)
An oligomeric compound comprising an oligonucleotide consisting of 10-30 linked nucleosides and at least one conjugate group, wherein the oligonucleotide comprises at the 5′ position a moiety having one of the formulas:
- wherein:
- T1 is a phosphorus moiety having the formula:
- wherein:
- Ra and Rc are each, independently, OH, SH, protected hydroxyl, protected thiol, C1-C6 alkyl, substituted C1-C6 alkyl, C1-C6 alkoxy, substituted C1-C6 alkoxy, amino or substituted amino;
- Rb is O or S;
- and
- Q1 and Q2 are each, independently, H, halogen, C1-C6 alkyl, substituted C1-C6 alkyl, C1-C6 alkoxy, or substituted C1-C6 alkoxy;
- each substituted group comprises one or more optionally protected substituent groups independently selected from halogen, OJ1, N(J1)(J2), =NJ1, SJ1, N3, CN, OC(═X2)J1, OC(═X2)N(J1)(J2) and C(═X2)N(J1)(J2);
- X2 is O, S or NJ3;
- and
- J1, J2 and J3 are each, independently, H or C1-C6 alkyl.
8 more elements
Dependencies are inferred from the claim text.