# US 9,040,493 B2 · RNA antagonists targeting GLI2 for the treatment of leukemia

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- Number: US 9,040,493 B2
- Document ID: US9040493B2
- Status: Granted
- Kind: B2
- Application number: 13/384,163
- Filed: 2010-01-15
- Published: 2012-09-13
- Granted: 2015-05-26
- Earliest claimed: 2009-07-15
- Assignees: Santaris Pharma A/S
- Applicants: Yixian Zhang; Zhengxing Qu
- Inventors: Yixian Zhang; Zhengxing Qu
- Web page: https://patentgrep.com/patent/US9040493B2

## CPC

- A61K 31/7088: Compounds having three or more nucleosides or nucleotides
- A61P 35/02: specific for leukemia
- A61P 37/02: Immunomodulators
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS

## Abstract

The present invention relates to oligomer compounds (oligomers) for the treatment and prevention of acute myeloid leukemia, which target GLI2 mRNA in a cell, leading to reduced expression of GLI2.

## Claims

### Claim 1

A method for treating acute myeloid leukaemia or pre-leukaemia, comprising administering to a patient an oligomer consisting of 10-30 nucleobases wherein the oligomer is perfectly complementary to a corresponding region of SEQ ID NO:1, all linkages between nucleobases are phosphorothioate linkages, and comprises nucleotide analogues having a modified sugar.

### Claim 2 (depends on 1)

The method of claim 1 wherein the oligomer is not perfectly complementary along its entire length to any portion of SEQ ID NO:2.

### Claim 3 (depends on 1)

The method of claim 1 wherein the oligomer comprises the sequence of SEQ ID NO:19.

### Claim 4 (depends on 1)

The method of claim 1 wherein the is between 10-18 nucleobases in length.

### Claim 5 (depends on 1)

The method of claim 1 wherein the nucleotide analogues having a modified sugar are selected from the group consisting of: Locked Nucleic Acid (LNA); 2′-O-alkyl-RNA, 2′-OMe-RNA, 2′-amino-DNA, and 2′-fluoro-DNA.

### Claim 6 (depends on 5)

The method of claim 5 wherein the nucleotide analogues are LNA.

### Claim 7 (depends on 1)

The method of claim 1 wherein the oligomer is a gapmer.

### Claim 8 (depends on 1)

The method of claim 1 wherein the oligomer inhibits the expression of GL12 or GL12 mRNA in a cell which is expressing GL12 or GL12 mRNA.

### Claim 9 (depends on 1)

The method of claim 1 wherein the oligomer comprises at least one non-nucleotide or non-polynucleotide moiety.

### Claim 10 (depends on 1)

The method of claim 1 wherein the preleukemia is myelodysplastic syndrome or myeloproliferative disease.

### Claim 11 (depends on 1)

The method of claim 1 wherein the oligomer comprises SEQ ID NOs: 112, 114, 118, 120, or 132.

### Claim 12 (depends on 1)

The method of claim 1 wherein the oligomer is between 12-18 nucleobases in length.

### Claim 13 (depends on 1)

The method of claim 1 wherein the oligomer is between 12-16 nucleobases in length.