# US 2014/0193366 A1 · Modulators of miR-323-3p for the Prevention or Treatment of Rheumatoid Arthritis

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- Number: US 2014/0193366 A1
- Document ID: US20140193366A1
- Status: Published application
- Kind: A1
- Application number: 14/241,456
- Filed: 2012-08-31
- Published: 2014-07-10
- Earliest claimed: 2011-08-31
- Assignees: Universitat Zurich Prorektorat MNW
- Inventors: Steffen Gay; Mary Connolly; Caroline Ospelt
- Web page: https://patentgrep.com/patent/US20140193366A1

## 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 at position 1

-12. (canceled)

### Claim 13

A method for treating rheumatoid arthritis in a patient in need thereof, comprising administering to the patient a miR-323-3p inhibitor, wherein the miR-323-3p inhibitor comprises a hybridizing sequence of nucleotides, wherein said hybridizing sequence is capable of forming a hybrid to SEQ ID 1 or SEQ ID 2; thereby treating the rheumatoid arthritis.

### Claim 14 (depends on 13)

The method of claim 13, wherein the hybridizing sequence comprises 8 to 30 nucleotides.

### Claim 15 (depends on 13)

The method of claim 13, wherein the hybridizing sequence is at least 90% identical to the reverse complimentary sequence of SEQ ID 1 or SEQ ID 2.

### Claim 16 (depends on 13)

The method of claim 13, wherein the hybridizing sequence comprises deoxynucleotides.

### Claim 17 (depends on 13)

The method of claim 13, wherein the hybridizing sequence comprises LNA and/or PNA nucleotides.

### Claim 18 (depends on 13)

The method of claim 13, wherein the hybridizing sequence comprises phosphothioate and/or 2′-O-methyl-modified phosphothioate nucleotides.

### Claim 19 (depends on 13)

The method of claim 13, wherein the hybridizing sequence is covalently attached to a cholesterol moiety or a peptide.

### Claim 20 (depends on 13)

The method of claim 13, wherein the miR-323-3p inhibitor further comprises a transcribed sequence comprising at least one decoy sequence tract at least 90% identical to sequence of SEQ ID 1 or SEQ ID 2 under control of a polymerase promoter sequence operable in a mammalian cell.

### Claim 21 (depends on 20)

The method of claim 20, wherein the miR-323-3p inhibitor comprises at least 5 decoy sequence tracts.

### Claim 22 (depends on 13)

The method of claim 13, wherein the miR-323-3p inhibitor is a component of a pharmaceutical composition further comprising a pharmaceutically acceptable carrier and/or excipient.

### Claim 23

A method for treating rheumatoid arthritis in a patient in need thereof,
comprising administering to the patient a virus particle comprising:
a transcribed sequence comprising a decoy sequence tract at least 90% identical to sequence of SEQ ID 1 or SEQ ID 2 or
a sequence being capable of forming a hybrid to SEQ ID 1 or SEQ ID 2, wherein the sequence is under control of a polymerase promoter sequence operable in a mammalian cell;
thereby treating the rheumatoid arthritis.

### Claim 24 (depends on 23)

The method of claim 23, wherein the virus is an adenovirus or adeno-associated virus.

### Claim 25 (depends on 23)

The method of claim 23, wherein the virus is a component of a pharmaceutical composition further comprising a pharmaceutically acceptable carrier and/or excipient.