# US 12,006,499 B2 · Una amidites and uses thereof

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- Number: US 12,006,499 B2
- Document ID: US12006499B2
- Status: Granted
- Kind: B2
- Application number: 16/894,648
- Filed: 2020-06-05
- Published: 2020-12-10
- Granted: 2024-06-11
- Earliest claimed: 2019-06-06
- Assignees: Avidity Biosciences, Inc.
- Applicants: Avidity Biosciences, Inc.
- Inventors: Venkata Ramana Doppalapudi; Michael Caramian Cochran
- Web page: https://patentgrep.com/patent/US12006499B2

## CPC

- C12N 15/113: Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; {Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing (when used in plants C12N15/8218)}
- C07F 9/6512: having the nitrogen atoms in positions 1 and 3
- C07F 9/65616: {containing the ring system [IMAGE cpc-sch-C07F-1006.gif] having three or more than three double bonds between ring members or between ring members and non-ring members, e.g. purine or analogs}
- C12N 15/1136: {against growth factors, growth regulators, cytokines, lymphokines or hormones}
- C12N 2310/14: interfering nucleic acids [NA]
- C12N 2310/314: Phosphoramidates
- C12N 2310/315: Phosphorothioates
- C12N 2310/321: 2'-O-R Modification
- C12N 2310/322: 2'-R Modification
- C12N 2310/3231: having an additional ring, e.g. LNA, ENA
- C12N 2310/335: Modified T or U
- C12N 2310/344: Position-specific modifications, e.g. on every purine, at the 3'-end
- C12N 2310/3513: Protein; Peptide
- C12N 2320/32: Special delivery means, e.g. tissue-specific
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- C07F: ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM (metal-containing porphyrins C07D487/22)

## 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

### Claim 1

A compound, or a pharmaceutically acceptable salt or solvate thereof, having the structure of Formula (II):
wherein:
Ring G is
X is O, NH, or C(═O);
R1 is hydrogen, deuterium, halogen, substituted or unsubstituted C1-C10 alkyl, substituted or unsubstituted C2-C10 alkenyl, substituted or unsubstituted C2-C10 alkynyl, substituted or unsubstituted C1-C10 fluoroalkyl, substituted or unsubstituted C1-C20 heteroalkyl, substituted or unsubstituted cycloalkyl, substituted or unsubstituted heterocycloalkyl, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, substituted or unsubstituted —O-aryl, —OR6, —C(═O)R6, —C(═O)OR6, —C(═O)NR7R7, —OCH2C(═O)R6, —OCH2C(═O)OR6, or —OCH2C(═O)NR7R7;
each R2 is independently hydrogen, deuterium, or substituted or unsubstituted C1-C10 alkyl;
or two R2 are taken together with the nitrogen atom to which they are attached to form a substituted or unsubstituted C3-C10 heterocycloalkyl;
R3 is hydrogen, 4,4′-dimethoxytrityl (DMT), —R6, —OR6, or —NR7R7;
each R4 is independently hydrogen, substituted or unsubstituted C1-C6alkyl, substituted or unsubstituted aryl, or substituted or unsubstituted benzoyl;
R5 is —C(═O)R6;
each R6 and R7 are independently hydrogen, substituted or unsubstituted C1-C6alkyl, substituted or unsubstituted C1-C6fluoroalkyl, substituted or unsubstituted C3-C6cycloalkyl, substituted or unsubstituted heterocycloalkyl, substituted or unsubstituted aryl, or substituted or unsubstituted heteroaryl.

### Claim 2 (depends on 1)

The compound of claim 1, or a pharmaceutically acceptable salt or solvate, thereof, wherein: R1 is
n is 0, 1, 2, 3, 4, or 5; and
R5 is hydrogen or substituted or unsubstituted C1-C3 alkyl.

### Claim 3 (depends on 1)

The compound of claim 1, or a pharmaceutically acceptable salt or solvate, thereof, wherein: R1 is —OCH2C(═O)NR7R7; wherein each R7 is independently hydrogen or substituted or unsubstituted C1-C3 alkyl.

### Claim 4 (depends on 1)

The compound of claim 1, or a pharmaceutically acceptable salt or solvate thereof, wherein:
Ring G is
wherein each R4 is independently hydrogen, or substituted or unsubstituted benzoyl.

### Claim 5 (depends on 1)

The compound of claim 1, or a pharmaceutically acceptable salt or solvate thereof, wherein:
Ring G is
wherein each R4 is independently hydrogen or substituted or unsubstituted benzoyl; and R5 is —C(═O)R6.

### Claim 6 (depends on 1)

The compound of claim 1, or a pharmaceutically acceptable salt or solvate thereof, wherein each R2 is independently isopropyl.

### Claim 7 (depends on 1)

The compound of claim 1, or a pharmaceutically acceptable salt or solvate thereof, wherein two R2 are taken together with the nitrogen atom to which they are attached to form a substituted or unsubstituted C3-C10 heterocycloalkyl.

### Claim 8 (depends on 1)

The compound of claim 1, or a pharmaceutically acceptable salt or solvate thereof, wherein X is O; and R3 is 4,4′-dimethoxytrityl (DMT).