IP Library Patent Application 18587877
Patent Application
App. No. 18/587,877

UNA AMIDITES AND USES THEREOF

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Patent No.
US None
App. No.
18/587,877
Abstract

Disclosed herein are compositions and pharmaceutical formulations that comprise a binding moiety conjugated to a modified polynucleic acid molecule and a polymer. Also described herein include methods for treating a disease which utilize a composition or a pharmaceutical formulation comprising a binding moiety conjugated to a polynucleic acid molecule and a polymer.

Claims (36)

1 - 20 . (canceled)

21 . An oligonucleotide conjugate of Formula (I):

A-B   Formula (I);

wherein,

A is a binding moiety;

B is an oligonucleotide comprising a compound of, or a compound derived from, Formula (II-1), (II-2), or (II-3); and

wherein:

Ring G is

X is O, NH, or C(═O);

L 1 and L 2 are each independently absent or a linking moiety selected from a phosphodiester, amide and ester linkage:

R 1 is hydrogen, deuterium, halogen, substituted or unsubstituted C 1 -C 10 alkyl, substituted or unsubstituted C 2 -C 10 alkenyl, substituted or unsubstituted C 2 -C 10 alkynyl, substituted or unsubstituted C 1 -C 10 fluoroalkyl, substituted or unsubstituted C 1 -C 20 heteroalkyl, substituted or unsubstituted cycloalkyl, substituted or unsubstituted heterocycloalkyl, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, substituted or unsubstituted —O-aryl, —OR 6 , —C(═O)R 6 , —C(═O)OR 6 , —C(═O)NR 7 R 7 , —OCH 2 C(═O)R 6 , —OCH 2 C(═O)OR 6 , or —OCH 2 C(═O)NR 7 R 7 ;

each R 2 is independently hydrogen, deuterium, or substituted or unsubstituted C 1 -C 10 alkyl;

or two R 2 are taken together with the nitrogen atom to which they are attached to form a substituted or unsubstituted C 3 -C 10 heterocycloalkyl;

R 3 is hydrogen, 4,4′-dimethoxytrityl (DMT), —R 6 , —OR 6 , or —NR′R 7 ;

each R 4 is independently hydrogen, substituted or unsubstituted C 1 -C 6 alkyl, substituted or unsubstituted aryl, or substituted or unsubstituted benzoyl;

R 5 is —C(═O)OR 6 ; and

each R 6 and R 7 are independently hydrogen, substituted or unsubstituted C 1 -C 6 alkyl, substituted or unsubstituted C 1 -C 6 fluoroalkyl, substituted or unsubstituted C 3 -C 6 cycloalkyl, substituted or unsubstituted heterocycloalkyl, substituted or unsubstituted aryl, or substituted or unsubstituted heteroaryl.

22 . The oligonucleotide conjugate of claim 21 , further comprising at least one 2′ modified nucleotide selected from 2′-O-methyl, 2′-O-methoxyethyl (2′-O-MOE), 2′-O-aminopropyl, 2′-deoxy, 2′-deoxy-2′-fluoro, 2′-O-aminopropyl (2′-O-AP), 2′-O-dimethylaminoethyl (2′-O-DMAOE), 2′-O-dimethylaminopropyl (2′-O-DMAP), 2′-O-dimethylaminoethyloxyethyl (2′-O-DMAEOE), 2′-O—N-methylacetamido (2′-O-NMA) modified nucleotide, locked nucleic acid (LNA) or ethylene nucleic acid (ENA).

23 . The oligonucleotide conjugate of claim 21 , further comprising at least one modified internucleotide linkage selected from a phosphorothioate, a phosphorodithioate, a methylphosphonate, a phosphotriester, or an amide linkage.

24 . The oligonucleotide conjugate of claim 21 , wherein the compound of Formula (II-2) is located at the 5′-terminus of the oligonucleotide.

25 . The oligonucleotide conjugate of claim 24 , wherein the binding moiety is conjugated to the 5′-terminus of the oligonucleotide.

26 . The oligonucleotide conjugate of claim 21 , wherein the compound of Formula (II-1) is located at the 3′-terminus of the oligonucleotide.

27 . The oligonucleotide conjugate of claim 21 , wherein at least two consecutive compounds of Formula (II-1) are located at the 3′-terminus of the oligonucleotide.

28 . The oligonucleotide conjugate of claim 26 , wherein the binding moiety is conjugated to the 5′ terminus or 3′ terminus of the oligonucleotide.

29 . The oligonucleotide conjugate of claim 27 , wherein the oligonucleotide comprises a guide strand and a passenger strand, and wherein the at least two consecutive compounds of Formula (II-1) are located at the 3′-terminus of the oligonucleotide of the guide strand.

30 . The oligonucleotide conjugate of claim 27 , wherein the oligonucleotide comprises a guide strand and a passenger strand, and wherein the at least two consecutive compounds of Formula (II-1) are located at the 3′-terminus of the oligonucleotide of the passenger strand.

31 . The oligonucleotide conjugate of claim 30 , wherein oligonucleotide comprises a modified internucleotide linkage between the at least two consecutive compounds of Formula (II-1).

32 . The oligonucleotide conjugate of claim 21 , wherein the compound of Formula (II-1) is located at an internal position within the oligonucleotide.

33 . The oligonucleotide conjugate of claim 32 , wherein at least two consecutive compounds of Formula (II-1) are located at the internal position within the oligonucleotide.

34 . The oligonucleotide conjugate of claim 32 , wherein the internal position is within position 2-10 from the 3′ end of the oligonucleotide.

35 . The oligonucleotide conjugate of claim 34 , wherein the oligonucleotide comprises a guide strand and a passenger strand, and wherein the internal position is within position 2-10 from the 3′ end of the guide strand.

36 . The oligonucleotide conjugate of claim 34 , wherein the oligonucleotide comprises a guide strand and a passenger strand, and wherein the internal position is within position 2-6 from the 3′ end of the guide strand.

37 . The oligonucleotide conjugate of claim 33 , wherein the binding moiety is conjugated to the 5′ terminus or 3′ terminus of the oligonucleotide.

38 . The oligonucleotide conjugate of claim 21 , wherein the binding moiety comprises an antibody or antigen binding fragment thereof.

39 . The oligonucleotide conjugate of claim 21 , wherein the binding moiety comprises a peptide or a small molecule.

40 . The oligonucleotide conjugate of claim 21 , wherein the binding moiety is conjugated with the oligonucleotide via a linker.

Assignments (2)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Mar 20, 2026
From: AVIDITY BIOSCIENCES, INC.
To: ATRIUM THERAPEUTICS, INC.
Reel/Frame 074143/0886 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded May 15, 2024
From: DOPPALAPUDI, VENKATA RAMANA; COCHRAN, MICHAEL CARAMIAN
To: AVIDITY BIOSCIENCES, INC.
Reel/Frame 067426/0556 →