# US 2016/0354489 A1 · Gene therapy compositions for use in the prevention and/or treatment of non-alcoholic fatty liver disease

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

- Number: US 2016/0354489 A1
- Document ID: US20160354489A1
- Status: Published application
- Kind: A1
- Application number: 15/024,821
- Filed: 2014-09-25
- Published: 2016-12-08
- Earliest claimed: 2013-09-26
- Assignees: Universitat Autònome de Barcelona
- Applicants: Universitat Autonoma De Barcelona
- Inventors: Fàtima BOSCH TUBERT; Laia VILÁPRATS
- Web page: https://patentgrep.com/patent/US20160354489A1

## CPC

- A61K 48/0058: {Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct}
- A61K 38/50: acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
- A61K 48/005: {characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered}
- C12N 7/00: Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof (preparing medicinal viral antigen or antibody compositions, e.g. virus vaccines, A61K39/00)
- C12N 9/80: acting on amide bonds in linear amides {(3.5.1)}
- C12N 15/86: Viral vectors
- C12Y 305/01: in linear amides (3.5.1)
- C12N 2750/14141: Use of virus, viral particle or viral elements as a vector
- C12N 2750/14143: viral genome or elements thereof as genetic vector
- C12N 2750/14145: Special targeting system for viral vectors
- C12N 2750/14171: Demonstrated in vivo effect
- C12N 2830/008: cell type or tissue specific enhancer/promoter combination
- 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)
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- C12Y: ENZYMES

## Abstract

The present invention relates to the field of gene therapy and, more particularly, to methods for the prevention and/or treatment of non-alcoholic fatty liver disease (NAFLD). The viral vectors disclosed in the present invention are preferably parvoviral vector, more preferably adeno-associated vectors (AAV) containing the gene encoding Sirtuin 1 (Sirt1) in the prevention and/or treatment of NAFLD. The present invention also discloses the pharmaceutical compositions comprising the viral vector and the method of prevention and/or treatment of NAFLD and related diseases by the administration of said pharmaceutical compositions.

## Claims

### Claim 1

A viral construct comprising a nucleotide sequence encoding Sirtuin 1 (Sirt1), or a functionally equivalent variant thereof, said viral construct being operably linked to a liver-specific promoter.

### Claim 2 (depends on 1)

The viral construct as defined in claim 1 which is a parvoviral construct.

### Claim 3 (depends on 2)

The viral construct as defined in claim 2 wherein the parvoviral construct is an AAV construct.

### Claim 4 (depends on 3)

The viral construct as defined in claim 3 wherein the AAV construct is an AAV2, AAV5, AAV7, AAV8 or AAV9.

### Claim 5 (depends on 3)

The viral construct as defined in claim 3 wherein the AAV construct is an AAV8.

### Claim 6 (depends on 1)

The viral construct as defined in claim 1, wherein the liver specific promoter is selected from: albumin promoter, phosphoenol pyruvate carboxykinase (PEPCK) promoter or alpha1-antitrypsin promoter.

### Claim 7 (depends on 6)

The viral construct as defined in claim 6 wherein the liver specific promoter is the alpha1-antitrypsin promoter.

### Claim 8 (depends on 1)

The viral construct as defined in claim 1 wherein Sirt1 corresponds to a human Sirt1.

### Claim 9 (depends on 1)

A virion comprising the viral construct as defined in claim 1 packaged inside a capsid and, optionally a lipidic envelope surrounding said capside.

### Claim at position 10

10-12. (canceled)

### Claim 13 (depends on 1, 9)

A pharmaceutical composition comprising the viral construct as defined in claim 1 or the virion as defined in claim 9 and a pharmaceutically acceptable carrier.

### Claim at position 12

14-15. (canceled)

### Claim 16 (depends on 9, 13)

A method for treating a disease selected from: non-alcoholic fatty liver disease (NAFLD), insulin resistance, diabetes mellitus, metabolic syndrome or hyperlipidemia comprising administering the virion according to claim 9 or the pharmaceutical composition according to claim 13.

### Claim 17 (depends on 16)

The method according to claim 16, wherein the disease is non-alcohol fatty liver disease (NAFLD).