# US 11,077,131 B2 · Modulation of ciliogenesis

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- Number: US 11,077,131 B2
- Document ID: US11077131B2
- Status: Granted
- Kind: B2
- Application number: 15/776,668
- Filed: 2016-11-17
- Published: 2018-11-15
- Granted: 2021-08-03
- Earliest claimed: 2015-11-17
- Assignees: Academisch Ziekenhuis Leiden a/u Leiden University Medical Center
- Applicants: Academisch Ziekenhuis Leiden a/u Leiden University Medical Center
- Inventors: Ronald Karel Louisa Van Brempt
- Web page: https://patentgrep.com/patent/US11077131B2

## CPC

- A61K 31/7088: Compounds having three or more nucleosides or nucleotides
- A61K 38/1709: {from mammals}
- A61K 45/00: Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K 45/06: Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
- A61P 9/00: Drugs for disorders of the cardiovascular system
- A61P 11/00: Drugs for disorders of the respiratory system
- 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)}
- C12N 2310/14: interfering nucleic acids [NA]
- C12N 2310/531: Stem-loop; Hairpin
- 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
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)

## Abstract

The disclosure is based on the finding that compounds capable of binding to (or interacting with) chromatin binding/remodelling complexes (for example Polycomb group PRC1 and Trithorax group MLL) and/or modulation of the same can be used to modulate (for example switch on/off) ciliogenesis as may occur, for example, in the human pulmonary bronchial epithelium. Provided are compounds, compositions, methods and medicaments which may be used to treat and/or prevent diseases and/or conditions associated with aberrant or defective ciliogenesis.

## Claims

### Claim 1

A method of treating diseases and/or conditions of the pulmonary system and/or airways caused by a loss of ciliogenesis, said method comprising administering to a subject in need thereof a therapeutically effective amount of a compound comprising an antisense oligonucleotide or an RNAi type inhibitor that reduces, inhibits and/or ablates the expression, function and/or activity of the R2R1 gene, thereby treating the diseases and/or conditions of the pulmonary system and/or airways caused by a loss of ciliogenesis.

### Claim 2 (depends on 1)

The method of claim 1, wherein the compound is an antisense oligonucleotide.

### Claim 3 (depends on 1)

The method of claim 1, wherein the compound comprises a sequence of SEQ ID NO: 1, 2, 3 or 4 or a fragment thereof.

### Claim 4 (depends on 1)

The method of claim 1, wherein the compound is an antisense oligonucleotide which reduces the expression of the R2R1 gene.

### Claim 5 (depends on 1)

The method of claim 1, wherein the disease and/or condition is chronic obstructive pulmonary disorder (COPD).