# US 2014/0155466 A1 · Use of aptamers in therapy and/or diagnosis of autoimmune diseases

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- Number: US 2014/0155466 A1
- Document ID: US20140155466A1
- Status: Published application
- Kind: A1
- Application number: 14/003,675
- Filed: 2012-03-02
- Published: 2014-06-05
- Earliest claimed: 2011-03-07
- Assignees: Max-Delbruck-Centrum fur Molekulare Medizin; Charite-Universitatsmedizin Berlin
- Inventors: Ingolf Schimke; Annekathrin Haberland; Gerd Wallukat
- Web page: https://patentgrep.com/patent/US20140155466A1

## CPC

- C12N 15/115: Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith {; Nucleic acids binding to non-nucleic acids, e.g. aptamers}
- A61M 1/3679: {by absorption (A61M1/3675 takes precedence)}
- A61P 1/00: Drugs for disorders of the alimentary tract or the digestive system
- A61P 1/04: for ulcers, gastritis or reflux esophagitis, e.g. antacids, inhibitors of acid secretion, mucosal protectants
- A61P 7/08: Plasma substitutes; Perfusion solutions; Dialytics or haemodialytics; Drugs for electrolytic or acid-base disorders, e.g. hypovolemic shock (artificial tears A61P27/04)
- A61P 9/00: Drugs for disorders of the cardiovascular system
- A61P 9/08: Vasodilators for multiple indications
- A61P 9/12: Antihypertensives
- A61P 11/00: Drugs for disorders of the respiratory system
- A61P 13/02: of urine or of the urinary tract, e.g. urine acidifiers
- A61P 13/08: of the prostate
- A61P 13/12: of the kidneys
- A61P 15/00: Drugs for genital or sexual disorders (for disorders of sex hormones A61P5/24); Contraceptives
- A61P 17/00: Drugs for dermatological disorders
- A61P 17/06: Antipsoriatics
- A61P 25/00: Drugs for disorders of the nervous system
- A61P 25/08: Antiepileptics; Anticonvulsants
- A61P 25/28: for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
- A61P 27/06: Antiglaucoma agents or miotics
- A61P 33/02: Antiprotozoals, e.g. for leishmaniasis, trichomoniasis, toxoplasmosis
- A61P 37/02: Immunomodulators
- A61P 37/06: Immunosuppressants, e.g. drugs for graft rejection
- G01N 33/564: for pre-existing immune complex or autoimmune disease {, i.e. systemic lupus erythematosus, rheumatoid arthritis, multiple sclerosis, rheumatoid factors or complement components C1-C9}
- G01N 33/6854: {Immunoglobulins}
- C12N 2310/16: Aptamers
- C12N 2310/317: with an inverted bond, e.g. a cap structure
- C12N 2310/3517: Marker; Tag
- C12N 2320/10: in screening processes
- C12N 2320/30: Special therapeutic applications
- Y02A 50/30: Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- A61M: DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY (introducing media into or onto the bodies of animals A61D7/00; means for inserting tampons A61F13/26; devices for administering food or medicines orally A61J; containers for collecting, storing or administering blood or medical fluids A61J1/05); DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY (surgery A61B; chemical aspects of surgical articles A61L); DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR {(Electrotherapy, e.g. producing anaesthesia by the use of alternating or intermittent currents A61N1/36021)}
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- G01N: INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES (measuring or testing apparatus or processes other than immunoassay, involving enzymes or microorganisms C12M, C12Q)
- Y02A: TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE

## Abstract

The present invention is directed to an aptamer comprising or consisting of the nucleic acid sequence of SEQ ID No. 1, SEQ ID No. 2, SEQ ID No. 3 and/or a nucleic acid sequence being at least 80% identical to one of SEQ ID No. 1, 2 and 3 for use in therapy and/or diagnosis of autoimmune diseases, wherein the autoimmune disease is cardiomyopathy, dilated cardiomyopathy (DCM), peripartum cardiomyopathy (PPCM), idiopathic cardiomyopathy, Chagas' cardiomyopathy, Chagas' megacolon, Chagas' megaesophagus, Chagas' neuropathy, benign prostatic hyperplasia, scleroderma, psoriasis, Raynaud syndrome, pre-eclamsia, kidney allograft rejection, myocarditis, glaucoma, hypertension, pulmonary hypertension, malignant hypertension, and/or Alzheimer's disease.

## Claims

### Claim 1

An aptamer comprising a nucleic acid sequence of SEQ ID No. 1, SEQ ID No. 2, SEQ ID No. 3 and/or a nucleic acid sequence being at least 80% identical to one of SEQ ID No. 1, 2 and 3 for use in therapy and/or diagnosis of autoimmune diseases, wherein the autoimmune disease is cardiomyopathy, dilated cardiomyopathy (DCM), peripartum cardiomyopathy (PPCM), idiopathic cardiomyopathy, Chagas' cardiomyopathy, Chagas' megacolon, Chagas' megaesophagus, Chagas' neuropathy, benign prostatic hyperplasia, scleroderma, psoriasis, Raynaud syndrome, pre-eclamsia, kidney allograft rejection, myocarditis, glaucoma, hypertension, pulmonary hypertension, malignant hypertension, and/or Alzheimer's disease.

### Claim 2

An aptamer comprising the nucleic acid sequence of SEQ ID No. 1, SEQ ID No. 2, SEQ ID No. 3 and/or a nucleic acid sequence being at least 80% identical to one of SEQ ID No. 1, 2 and 3 for use as selective ingredient during apheresis, preferably during therapeutic apheresis of blood or constituents thereof of a patient suffering from an autoimmune disease, wherein the autoimmune disease is cardiomyopathy, dilated cardiomyopathy (DCM), peripartum cardiomyopathy (PPCM), idiopathic cardiomyopathy, Chagas' cardiomyopathy, Chagas' megacolon, Chagas' megaesophagus, Chagas' neuropathy, benign prostatic hyperplasia, scleroderma, psoriasis, Raynaud syndrome, pre-eclamsia, kidney allograft rejection, myocarditis, glaucoma, hypertension, pulmonary hypertension, malignant hypertension, and/or Alzheimer's disease.

### Claim 3 (depends on 1)

The aptamer of claim 1, wherein the aptamer is used in therapy and/or diagnosis in a human.

### Claim 4 (depends on 1)

The aptamer of claim 1, wherein the aptamer is a DNA aptamer.

### Claim 5 (depends on 1)

The aptamer of claim 1, wherein the aptamer consists of the nucleic acid sequence of SEQ ID No. 1, SEQ ID No. 2, SEQ ID No. 3 or a nucleic acid sequence being at least 80% identical to one of SEQ ID No. 1, 2 and 3.

### Claim 6 (depends on 1)

A pharmaceutical composition comprising the aptamer of claim 1 and at least one pharmaceutically acceptable excipient for use in therapy and/or diagnosis of autoimmune diseases.

### Claim 7 (depends on 1)

A kit comprising the aptamer of claim 1 and a container for use in therapy and/or diagnosis of autoimmune diseases.

### Claim 8 (depends on 1)

Use of the aptamer of claim 1 for the in vitro detection of an antibody being specific for a G-protein coupled receptor, preferably the human G-protein coupled receptor adrenergic alpha-1 receptor, adrenergic beta-1 receptor, adrenergic beta-2 receptor, endothelin 1 ETA receptor, muscarinic M2 receptor, angiotensin II AT1 receptor, and/or PAR receptors.

### Claim 9 (depends on 8)

The use of claim 8, wherein the antibody to be detected is an autoantibody.

### Claim 10 (depends on 8)

The use of claim 8, wherein the antibody is present in or derived from a body fluid, preferably a fluid of a human body, more preferably of human blood, plasma, serum, urine feces, synovial fluid, interstitial fluid, lymph, saliva, spinal fluid and/or lacrimal fluid.

### Claim 11 (depends on 10)

The use of claim 10, wherein the body fluid is taken from an individual suffering from or suspected to suffer from an autoimmune disease, preferably an autoimmune disease associated with presence in the serum of the patient of autoantibodies specific for a G-protein coupled receptor, more preferably autoimmune diseases associated with presence in the serum of the patient of autoantibodies specific for adrenergic alpha-1 receptor, adrenergic beta-1 receptor, adrenergic beta-2 receptor, endothelin 1 ETA receptor, muscarinic M2 receptor, angiotensin II AT1 receptor, and/or PAR receptors.

### Claim 12 (depends on 1)

An apheresis column comprising the aptamer of claim 1.

### Claim 13 (depends on 12)

The apheresis column of claim 12 for use in therapy and/or diagnosis of an autoimmune disease, wherein the autoimmune disease is cardiomyopathy, dilated cardiomyopathy (DCM), peripartum cardiomyopathy (PPCM), idiopathic cardiomyopathy, Chagas' cardiomyopathy, Chagas' megacolon, Chagas' megaesophagus, Chagas' neuropathy, benign prostatic hyperplasia, scleroderma, psoriasis, Raynaud syndrome, pre-eclamsia, kidney allograft rejection, myocarditis, glaucoma, hypertension, pulmonary hypertension, malignant hypertension, and/or Alzheimer's disease.