# US 2019/0167713 A1 · Medicament for the Treatment of Acute Liver Failure

> Agents: this data is available as JSON from the patentgrep API and MCP server — see https://patentgrep.com/llms.txt

- Number: US 2019/0167713 A1
- Document ID: US20190167713A1
- Status: Published application
- Kind: A1
- Application number: 16/302,502
- Filed: 2017-05-30
- Published: 2019-06-06
- Earliest claimed: 2016-06-06
- Assignees: Medizinische Hochschule Hannover
- Applicants: Medizinische Hochschule Hannover
- Inventors: Amar SHARMA; Michael OTT; Tobias CANTZ; Dakai YANG
- Web page: https://patentgrep.com/patent/US20190167713A1

## 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 31/713: Double-stranded nucleic acids or oligonucleotides
- A61K 45/06: Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
- A61K 48/0066: {Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid}
- A61K 48/0075: {characterised by an aspect of the delivery route, e.g. oral, subcutaneous}
- A61K 48/0083: {characterised by an aspect of the administration regime}
- A61P 1/16: for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
- A61P 39/00: General protective or antinoxious agents
- 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 a compound for use as a medicament in the treatment or prevention of acute liver failure, which acute liver failure is induced by intoxication prior to or following administration of the compound. Accordingly, the invention relates to the compound for use in the treatment of an intoxication that without treatment is expected to result in acute liver failure or that without treatment is sufficient to induce acute liver failure. The invention also provides the compound for use in the treatment, and a corresponding method for the treatment of an intoxication sufficient to induce acute liver failure.

## Claims

### Claim 1

Compound for use in the treatment of an intoxication sufficient to cause acute liver failure, wherein the compound has a nucleobase sequence of SEQ ID NO: 1 (miR-125b-5p).

### Claim 2 (depends on 1)

Compound according to claim 1, the compound having a nucleobase sequence consisting of SEQ ID NO: 1.

### Claim 3 (depends on 1)

Compound according to claim 1, characterized in that the nucleobase sequence is in the form of RNA, chemically stabilized RNA, chemically modified RNA and/or formulated as liposomes and/or nanoparticles.

### Claim 4 (depends on 1)

Compound according to claim 1, wherein the intoxication is at maximum 4 days prior to the treatment.

### Claim 5 (depends on 1)

Compound according to claim 1, wherein the intoxication is by paracetamol and/or other drugs and/or alcohol and/or by a virus.

### Claim 6 (depends on 1)

Compound according to claim 1, wherein the acute liver failure is by hepatocyte apoptosis and/or necrosis.

### Claim 7 (depends on 1)

Compound according to claim 1, wherein the compound is present in combination with at least one compound having a nucleobase sequence of miR-194-5p (SEQ ID NO: 2), miR-21-5p (SEQ ID NO: 3), let-7a-5p (SEQ ID NO: 4), miR-122-5p (SEQ ID NO: 5), miR-30c-5p (SEQ ID NO: 6), miR 193a-3p (SEQ ID NO: 7), miR-130a-3p (SEQ ID NO: 8), miR-29c-3p (SEQ ID NO: 9), miR-16-5p (SEQ ID NO: 10), and/or miR-23-3p (SEQ ID NO: 11).

### Claim 8 (depends on 1)

Compound according to claim 1, wherein the intoxication is caused by a synthetic or natural compound, excluding virus infections.

### Claim 9

Method for treatment of an intoxication sufficient to cause acute liver failure, comprising administration of a compound having a nucleobase sequence of SEQ ID NO: 1 (miR-125b-5p).