Conjugate compositions comprising phosphoryl guanidine linkages and uses thereof
AssigneeAvidity Biosciences, Inc.
InventorsVenkata Ramana DOPPALAPUDI, Michael Caramian COCHRAN, Subbarao NALLAGATLA, Giuseppe DELLO IACONO, Sami Abdulwahab ABDULKADIR, Maryam JORDAN, Marc Andrew HARTMANN
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Abstract
Disclosed herein are compositions and pharmaceutical formulations that comprise a binding moiety conjugated to at least one oligonucleotide. In some cases, the oligonucleotide comprises at least one modified internucleotide linkage. Also, described herein include methods for treating a disease which utilize a composition or a pharmaceutical formulation comprising a binding moiety conjugated to at least one oligonucleotide.
Claims35
A conjugate of Formula (I):
- wherein,
- A is a binding moiety;
- B is a double-stranded oligonucleotide comprising a guide strand and a passenger strand;
- X is a bond or a linker;
- n is a number≥2;
- and
- wherein the guide strand or the passenger strand comprises at least one modified internucleotide linkage comprising the structure of Formula (II):
- wherein each R11, R12, R13, and R14 is independently selected from —H, —C1-10 alkyl, —C2-10 alkenyl, —C2-10 alkynyl, or —C6-10 aryl;
- optionally, wherein R12 and R13, together with an atom to which they are bound, form a 5-8 membered heterocyclic substituent moiety, selected from the group consisting of N-pyrrolidinyl, N-piperidinyl, N-azepanyl, N-azocanyl, and imidazolidine.
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Dependencies are inferred from the claim text.