# US 9,434,944 B2 · Modulators of miR-323-3p for the prevention or treatment of rheumatoid arthritis

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- Number: US 9,434,944 B2
- Document ID: US9434944B2
- Status: Granted
- Kind: B2
- Application number: 14/241,456
- Filed: 2012-08-31
- Published: 2014-07-10
- Granted: 2016-09-06
- Earliest claimed: 2011-08-31
- Assignees: University of Zurich
- Applicants: Steffen Gay; Mary Connolly; Caroline Ospelt
- Inventors: Steffen Gay; Mary Connolly; Caroline Ospelt
- Web page: https://patentgrep.com/patent/US9434944B2

## 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)}
- A61P 19/02: for joint disorders, e.g. arthritis, arthrosis
- C12N 2310/113: targeting other non-coding nucleic acids, e.g. antagomirs
- C12N 2310/315: Phosphorothioates
- C12N 2310/3181: Peptide nucleic acid, PNA
- C12N 2310/321: 2'-O-R Modification
- C12N 2310/3231: having an additional ring, e.g. LNA, ENA
- C12N 2310/3515: Lipophilic moiety, e.g. cholesterol
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS

## Abstract

A miR-323-3p inhibitor is provided for the prevention or therapy of rheumatoid arthritis, comprising a sequence capable of forming a hybrid to miR-323-3p as an oligonucleotide or a vector from which such oligonucleotide is transcribed, or as a miRNA decoy or sponge.

## Claims

### Claim 1

A method for treating rheumatoid arthritis in a patient in need thereof, comprising administering to the patient a miR-323-3p inhibitor comprising a hybridizing sequence of nucleotides at least 90% identical to reverse complementary sequence of SEQ ID 1 or SEQ ID 2; thereby treating the rheumatoid arthritis.

### Claim 2 (depends on 1)

The method of claim 1, wherein the hybridizing sequence comprises deoxynucleotides.

### Claim 3 (depends on 1)

The method of claim 1, wherein the hybridizing sequence comprises LNA and/or PNA nucleotides.

### Claim 4 (depends on 1)

The method of claim 1, wherein the hybridizing sequence comprises phosphothioate and/or 2′-O-methyl-modified phosphothioate nucleotides.

### Claim 5 (depends on 1)

The method of claim 1, wherein the hybridizing sequence is covalently attached to a cholesterol moiety or a peptide.

### Claim 6 (depends on 1)

The method of claim 1, wherein the miR-323-3p inhibitor is a component of a pharmaceutical composition further comprising a pharmaceutically acceptable carrier and/or excipient.

### Claim 7

A method for treating rheumatoid arthritis in a patient in need thereof, comprising administering to the patient a virus particle comprising:
a nucleotide sequence capable of forming a hybrid to SEQ ID 1 or SEQ ID 2, wherein the nucleotide sequence is the reverse complement of a nucleotide sequence that is 90% identical to SEQ ID 1 or SEQ ID 2, and wherein the nucleotide sequence hybridizes to miR-323-3p or its precursor, thereby inhibiting expression of miR-323-3p, and thereby treating the rheumatoid arthritis.

### Claim 8 (depends on 7)

The method of claim 7, wherein the virus is an adenovirus or adeno-associated virus.

### Claim 9 (depends on 7)

The method of claim 7, wherein the virus is a component of a pharmaceutical composition further comprising a pharmaceutically acceptable carrier and/or excipient.