# US 12,758,572 B2 · Treatments of non-alcoholic steatohepatitis (NASH)

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- Number: US 12,758,572 B2
- Document ID: US12758572B2
- Status: Granted
- Kind: B2
- Application number: 18/374,023
- Filed: 2023-09-28
- Published: 2024-01-25
- Granted: 2026-10-13
- Earliest claimed: 2016-06-28
- Assignees: Deutsches Krebsforschungszentrum Stiftung des Offentlichen Rechts
- Applicants: Deutsches Krebsforschungszentrum Stiftung Des öFfentlichen Rechts
- Inventors: Mathias Heikenwälder; Lars Zender; Achim Weber
- Web page: https://patentgrep.com/patent/US12758572B2

## CPC

- A61K 31/7105: Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
- A61K 9/0014: {Skin, i.e. galenical aspects of topical compositions (non-active ingredients are additionally classified in A61K47/00; A61K9/0009, A61K9/0021, A61K9/7015, A61K9/7023 take precedence; cosmetic preparations A61K8/00, A61Q; preparations for wound dressings or bandages A61L26/00)}
- A61K 9/0019: {Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner (non-active ingredients are additionally classified in A61K47/00)}
- A61K 9/0053: {Mouth and digestive tract, i.e. intraoral and peroral administration}
- A61K 31/00: Medicinal preparations containing organic active ingredients
- A61K 31/405: Indole-alkanecarboxylic acids; Derivatives thereof, e.g. tryptophan, indomethacin
- A61K 31/616: by carboxylic acids, e.g. acetylsalicylic acid
- A61K 31/713: Double-stranded nucleic acids or oligonucleotides
- A61P 1/16: for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
- 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 2310/11: Antisense
- C12N 2310/20: involving clustered regularly interspaced short palindromic repeats [CRISPR]
- 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 present invention provides novel compounds that target thrombocyte activity or aggregation capacity through cellular components for the treatment of diseases associated with Non-Alcoholic Fatty Liver Disease (NAFLD). The invention provides these compounds for treating non-alcoholic steatohepatitis (NASH), a progressed stage of NAFL (non-alcoholic fatty liver), in order to avoid the development of liver cirrhosis and Hepatocellular Carcinoma (HCC). Further provided are pharmaceutical compositions, comprising the compounds of the invention, and methods for screening new NASH therapeuticals.

## Claims

### Claim 1

A method for the treatment of non-alcoholic steatohepatitis (NASH), wherein said method comprises administering, to a subject in need of such treatment, an inhibitor of Gp1b, wherein the inhibitor is an antigen-binding fragment of an anti-Gp1b antibody that specifically and selectively binds to a Gp1b protein and inhibits its function.

### Claim 2 (depends on 1)

The method according to claim 1, wherein the treatment is an alleviation or a reduced progression of NASH.

### Claim 3 (depends on 1)

The method according to claim 1, wherein the treatment is a reduced, stalled, or reversed progression of NASH into liver cirrhosis.

### Claim 4 (depends on 1)

The method according to claim 1, wherein the subject is a NASH-patient at risk to develop cirrhosis and/or HCC.

### Claim 5 (depends on 1)

The method according to claim 1, wherein the treatment is performed in a patient at risk of developing NASH, wherein the patient is a diabetic patient, an obese patient, or a patient suffering from metabolic syndrome or from another metabolic disorder.

### Claim 6 (depends on 1)

The method according to claim 1, wherein the subject to be treated does not have a condition selected from the group consisting of alcoholic liver injury, drug-induced liver injury, chronic active hepatitis, hepatic steatosis and hepatocyte apoptosis.

### Claim 7 (depends on 1)

The method according to claim 1, wherein the patient suffers from an inflamed fatty liver.

### Claim 8 (depends on 1)

The method according to claim 1, wherein the antigen-binding fragment of an anti-Gp1b antibody is a Fab fragment that specifically and selectively binds to a Gp1b protein and inhibits its function.
