# US 5,707,618 A · Adenovirus vectors for gene therapy

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

- Number: US 5,707,618 A
- Document ID: US5707618A
- Status: Granted
- Kind: A
- Application number: 08/409,874
- Filed: 1995-03-24
- Granted: 1998-01-13
- Earliest claimed: 1995-03-24
- Assignees: Genzyme Corporation
- Inventors: Donna Armentano; Helen Romanczuk; Samuel Charles Wadsworth
- Web page: https://patentgrep.com/patent/US5707618A

## CPC

- C12N 15/86: Viral vectors
- C07K 14/4712: {Cystic fibrosis}
- A61K 48/00: Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- C12N 2710/10343: viral genome or elements thereof as genetic vector
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- C07K: PEPTIDES (peptides in foodstuffs A23; obtaining protein compositions for foodstuffs, working-up proteins for foodstuffs A23J; preparations for medicinal purposes A61K; peptides containing beta-lactam rings C07D; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, C07D; ergot alkaloids of the cyclic peptide type C07D519/02; macromolecular compounds having statistically distributed amino acid units in their molecules, i.e. when the preparation does not provide for a specific; but for a random sequence of the amino acid units, homopolyamides and block copolyamides derived from amino acids C08G69/00; macromolecular products derived from proteins C08H1/00; preparation of glue or gelatine C09H; single cell proteins, enzymes C12N; genetic engineering processes for obtaining peptides C12N15/00; compositions for measuring or testing processes involving enzymes C12Q; investigation or analysis of biological material G01N33/00)
- 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)

## Abstract

The present invention relates to novel adenovirus vectors for use in gene therapy which are designed to prevent the generation of replication-competent adenovirus (RCA) during in vitro propagation and clinical use. The invention also provides methods for the production of the novel virus vectors. These vectors maximize safety for clinical applications in which adenovirus vectors are used to transfer genes into recipient cells for gene therapy.

## Claims

### Claim 1

A recombinant adenovirus vector having a deleted E1 region of the adenovirus genome, into which a heterologous gene has been inserted, and in which the protein IX gene has been relocated in the adenovirus genome to a location thereof other than the location in which said protein IX gene normally resides, such that generation of replication-competent adenoviruses is minimized or eliminated.

### Claim 2 (depends on 1)

The vector of claim 1 in which one or more open reading frames of the E4 region is deleted.

### Claim 3 (depends on 2)

The vector of claim 2, in which the protein IX gene is relocated to the E4 region.

### Claim 4 (depends on 2)

The vector of claim 2, in which ORF6 of the E4 region is retained.

### Claim 5 (depends on 4)

The vector of claim 4, in which the protein IX gene is inserted adjacent to the ORF6 gene.

### Claim 6 (depends on 1)

The vector of claim 1, in which the heterologous gene is a gene encoding CFTR.

### Claim 7 (depends on 1)

The vector of claim 1 in which the heterologous gene is operably linked to a eucaryotic promoter, so as to allow for expression of the gene.

### Claim 8 (depends on 1)

The vector of claim 1, in which the adenovirus is selected from among adenovirus serotypes 2, 4, 5 and 7.