# US 2019/0167762 A1 · Efficient systemic treatment of dystrophic muscle pathologies

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

- Number: US 2019/0167762 A1
- Document ID: US20190167762A1
- Status: Published application
- Kind: A1
- Application number: 16/185,248
- Filed: 2018-11-09
- Published: 2019-06-06
- Earliest claimed: 2014-06-27
- Assignees: Genethon; Royal Holloway and Bedford New College
- Applicants: Genethon; Royal Holloway and Bedford New College
- Inventors: George Dickson; Thomas Voit; Philippe Moullier; Caroline Le Guiner
- Web page: https://patentgrep.com/patent/US20190167762A1

## CPC

- A61K 38/1719: {Muscle proteins, e.g. myosin or actin}
- A61K 48/005: {characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered}
- A61K 48/0058: {Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct}
- A61K 48/0075: {characterised by an aspect of the delivery route, e.g. oral, subcutaneous}
- A61P 21/00: Drugs for disorders of the muscular or neuromuscular system
- A61P 21/04: for myasthenia gravis
- 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 15/86: Viral vectors
- C12N 2750/14132: Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
- C12N 2750/14143: viral genome or elements thereof as genetic vector
- C12N 2750/14171: Demonstrated in vivo effect
- C12N 2840/007: cell or tissue specific
- 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

A composition comprising a gene therapy product for use in the treatment of a dystrophic disease in a subject, advantageously in humans, wherein: the gene therapy product comprises a nucleic acid sequence encoding a functional microdystrophin; the composition is systemically administered.

## Claims

### Claim at position 1

1-16. (canceled)

### Claim 17

A gene therapy product, comprising an adeno-associated viral (AAV) vector which harbors a nucleic acid sequence encoding a human ΔR4-R23/ΔCT microdystrophin, wherein the AAV vector is an AAV2/8 vector, and wherein the nucleic acid sequence comprises SEQ ID NO: 1.

### Claim 18 (depends on 17)

The gene therapy product according to claim 17, wherein the nucleic acid sequence consists of SEQ ID NO: 1.

### Claim 19

A pharmaceutical composition, comprising a gene therapy product and a pharmaceutically acceptable carrier, wherein the gene therapy product comprises an adeno-associated viral (AAV) vector which harbors a nucleic acid sequence encoding a human ΔR4-R23/ΔCT microdystrophin, and wherein the AAV vector is an AAV2/8 vector, and wherein the nucleic acid sequence comprises SEQ ID NO: 1.

### Claim 20 (depends on 19)

The pharmaceutical composition according to claim 19, wherein the nucleic acid sequence consists of SEQ ID NO: 1.

### Claim 21 (depends on 19)

The pharmaceutical composition of claim 19, wherein the pharmaceutical composition is formulated as an injectable solution for intravenous administration to a human.

### Claim 22 (depends on 20)

The pharmaceutical composition of claim 20, wherein the pharmaceutical composition is formulated as an injectable solution for intravenous administration to a human.