# US 2017/0283476 A1 · Myc nucleic acids and uses thereof

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- Number: US 2017/0283476 A1
- Document ID: US20170283476A1
- Status: Published application
- Kind: A1
- Application number: 15/476,429
- Filed: 2017-03-31
- Published: 2017-10-05
- Earliest claimed: 2016-04-01
- Assignees: Avidity Biosciences, Inc.
- Applicants: Avidity Biosciences LLC
- Inventors: Hanhua HUANG; Rob BURKE
- Web page: https://patentgrep.com/patent/US20170283476A1

## CPC

- C07K 14/47: from mammals
- C07K 14/4748: {Tumour specific antigens; Tumour rejection antigen precursors [TRAP], e.g. MAGE}
- C12N 15/09: Recombinant DNA-technology
- C12N 15/1135: {against oncogenes or tumor suppressor genes}
- C12Q 1/68: involving nucleic acids
- C12Q 1/686: Polymerase chain reaction [PCR]
- C07K 14/00: Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C12N 2310/00: Structure or type of the nucleic acid
- C12N 2310/14: interfering nucleic acids [NA]
- C12N 2310/315: Phosphorothioates
- C07K: PEPTIDES (peptides in foodstuffs A23; obtaining protein compositions for foodstuffs, working-up proteins for foodstuffs A23J; preparations for medicinal purposes A61K; peptides containing beta-lactam rings C07D; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, C07D; ergot alkaloids of the cyclic peptide type C07D519/02; macromolecular compounds having statistically distributed amino acid units in their molecules, i.e. when the preparation does not provide for a specific; but for a random sequence of the amino acid units, homopolyamides and block copolyamides derived from amino acids C08G69/00; macromolecular products derived from proteins C08H1/00; preparation of glue or gelatine C09H; single cell proteins, enzymes C12N; genetic engineering processes for obtaining peptides C12N15/00; compositions for measuring or testing processes involving enzymes C12Q; investigation or analysis of biological material G01N33/00)
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- C12Q: MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS (immunoassay G01N33/53); COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES

## Abstract

Disclosed herein are molecules and pharmaceutical compositions that mediate RNA interference against MYC. Also described herein include methods for treating a disease or disorder that comprises a molecule or a pharmaceutical composition that mediate RNA interference against MYC.

## Claims

### Claim 1

A polynucleic acid molecule that mediates RNA interference against MYC, wherein the polynucleic acid molecule comprises at least 80% sequence identity to a sequence selected from SEQ ID NOs: 1-12, wherein the polynucleic acid molecule hybridizes to a MYC target sequence with less than 4 mismatched bases, wherein the polynucleic acid molecule comprises at least one 2′ modified nucleotide, at least one modified internucleotide linkage, or at least one inverted abasic moiety, and wherein the polynucleic acid molecule is from about 10 to about 50 nucleotides in length.

### Claim 2 (depends on 1)

The polynucleic acid molecule of claim 1, wherein the polynucleic acid molecule comprises at least 85%, 90%, 95%, 96%, 97%, 98%, 99%, or 100% sequence identity to a sequence selected from SEQ ID NOs: 1-12.

### Claim 3 (depends on 1)

The polynucleic acid molecule of claim 1, wherein the polynucleic acid molecule hybridizes to a MYC target sequence with less than 3 mismatched bases, less than 2 mismatched bases, or less than 1 mismatched bases.

### Claim 4 (depends on 1)

The polynucleic acid molecule of claim 1, wherein the at least one 2′ modified nucleotide comprises 2′-O-methyl, 2′-O-methoxyethyl (2′-O-MOE), 2′-O-aminopropyl, 2′-deoxy, T-deoxy-2′-fluoro, 2′-O-aminopropyl (2′-O-AP), 2′-O-dimethylaminoethyl (2′-O-DMAOE), 2′-O-dimethylaminopropyl (2′-O-DMAP), T-O-dimethylaminoethyloxyethyl (2′-O-DMAEOE), or 2′-O—N-methylacetamido (2′-O-NMA) modified nucleotide.

### Claim 5 (depends on 1)

The polynucleic acid molecule of claim 1, wherein the at least one 2′ modified nucleotide comprises locked nucleic acid (LNA) or ethylene nucleic acid (ENA).

### Claim 6 (depends on 1)

The polynucleic acid molecule of claim 1, wherein the at least one inverted basic moiety is at at least one terminus.

### Claim 7 (depends on 1)

The polynucleic acid molecule of claim 1, wherein the at least one modified internucleotide linkage comprises a phosphorothioate linkage or a phosphorodithioate linkage.

### Claim 8 (depends on 1)

The polynucleic acid molecule of claim 1, wherein the polynucleic acid molecule is from about 10 to about 30 nucleotides in length.

### Claim 9 (depends on 1)

The polynucleic acid molecule of claim 1, wherein the polynucleic acid molecule is at least 16, 17, 18, 19, 20, 21, 22, 23, 24, or 25 nucleotides in length.

### Claim 10 (depends on 1)

The polynucleic acid molecule of claim 1, wherein the polynucleic acid molecule comprises at least one of: from about 5% to about 100% modification, from about 10% to about 100% modification, from about 20% to about 100% modification, from about 30% to about 100% modification, from about 40% to about 100% modification, from about 50% to about 100% modification, from about 60% to about 100% modification, from about 70% to about 100% modification, from about 80% to about 100% modification, and from about 90% to about 100% modification.

### Claim 11 (depends on 1)

The polynucleic acid molecule of claim 1, wherein the polynucleic acid molecule comprises about 1, about 2, about 3, about 4, about 5, about 6, about 7, about 8, about 9, about 10, about 11, about 12, about 13, about 14, about 15, about 16, about 17, about 18, about 19, about 20, about 21, about 22, or more modified nucleotides.

### Claim 12 (depends on 1)

The polynucleic acid molecule of claim 1, wherein the polynucleic acid molecule further hybridizes to a second polynucleotide to form a double-stranded polynucleic acid molecule.

### Claim 13 (depends on 12)

The polynucleic acid molecule of claim 12, wherein the second polynucleotide comprises at least one modification.

### Claim 14 (depends on 12)

The polynucleic acid molecule of claim 12, wherein the second polynucleotide comprises a sequence having at least 80%, 85%, 90%, 95%, 96%, 97%, 98%, 99%, or 100% sequence identity to a sequence selected from SEQ ID NOs: 1-12.

### Claim 15 (depends on 1)

The polynucleic acid molecule of claim 1, wherein the polynucleic acid molecule is RNA.

### Claim 16 (depends on 1)

The polynucleic acid molecule of claim 1, wherein MYC is c-MYC gene, L-MYC gene, or N-MYC gene.

### Claim 17 (depends on 1)

The polynucleic acid molecule of claim 1, wherein MYC is c-MYC gene.

### Claim 18 (depends on 1)

A pharmaceutical composition comprising:
a) a molecule of claim 1; and
b) a pharmaceutically acceptable excipient.

### Claim 19 (depends on 18)

The pharmaceutical composition of claim 18, wherein the pharmaceutical composition is formulated as a nanoparticle formulation.

### Claim 20 (depends on 18)

The pharmaceutical composition of claim 18, wherein the pharmaceutical composition is formulated for parenteral, oral, intranasal, buccal, rectal, or transdermal administration.

### Claim 21 (depends on 1)

A method of treating a disease or disorder in a patient in need thereof, comprising administering to the patient a composition comprising a molecule of claim 1.

### Claim 22 (depends on 21)

The method of claim 21, wherein the disease or disorder is a cancer.

### Claim 23 (depends on 22)

The method of claim 22, wherein the cancer comprises a MYC-associated cancer.

### Claim 24 (depends on 22)

The method of claim 22, wherein the cancer comprises bladder cancer, breast cancer, colorectal cancer, endometrial cancer, esophageal cancer, glioblastoma multiforme, head and neck cancer, kidney cancer, lung cancer, ovarian cancer, pancreatic cancer, prostate cancer, or thyroid cancer.

### Claim 25 (depends on 22)

The method of claim 22, wherein the cancer comprises acute myeloid leukemia, CLL, DLBCL, or multiple myeloma.

### Claim 26 (depends on 1)

A method of inhibiting the expression of MYC gene in a primary cell of a patient, comprising administering a molecule of claim 1 to the primary cell.

### Claim 27 (depends on 26)

The method of claim 26, wherein the method is an in vivo method.

### Claim 28 (depends on 26)

The method of claim 26, wherein the patient is a human.