# US 2014/0194361 A1 · Vault Agents for Treating Chronic Kidney Disease

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- Number: US 2014/0194361 A1
- Document ID: US20140194361A1
- Status: Published application
- Kind: A1
- Application number: 14/050,280
- Filed: 2013-10-09
- Published: 2014-07-10
- Earliest claimed: 2010-04-21
- Inventors: Susanne B. Nicholas; Leonard H. Rome; Valerie A. Kickhoefer
- Web page: https://patentgrep.com/patent/US20140194361A1

## CPC

- C07K 14/47: from mammals
- A61P 13/12: of the kidneys
- C07K 5/0817: {the first amino acid being Arg}
- C07K 5/1013: {the side chain containing O or S as heteroatoms, e.g. Cys, Ser}
- C07K 14/78: Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
- A61K 38/00: Medicinal preparations containing peptides (peptides containing beta-lactam rings A61K31/00; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, A61K31/00; ergot alkaloids of the cyclic peptide type A61K31/48; containing macromolecular compounds having statistically distributed amino acid units A61K31/74; medicinal preparations containing antigens or antibodies A61K39/00; medicinal preparations characterised by the non-active ingredients, e.g. peptides as drug carriers, A61K47/00)
- 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)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- 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 invention relates to compositions of vault complexes containing cell adhesion inhibiting agents, such as a RGD-peptide, and methods of using the vault complexes in the treatment of diseases, such as chronic kidney disease.

## Claims

### Claim 1

A vault complex comprising a cell adhesion modifying substance.

### Claim 2 (depends on 1)

The vault complex of claim 1, wherein the cell adhesion modifying substance inhibits integrin binding.

### Claim 3 (depends on 2)

The vault complex of claim 2, wherein the cell adhesion modifying substance is an RGD-containing peptide.

### Claim 4 (depends on 3)

The vault complex of claim 3, wherein the RGD-containing peptide is cyclic.

### Claim 5 (depends on 4)

The vault complex of claim 4, wherein the RGD-containing peptide is GRGDSP.

### Claim 6 (depends on 4)

The vault complex of claim 4, wherein the cyclic RGD-containing peptide is attached to mINT.

### Claim 7 (depends on 1)

The vault complex of claim 1, wherein the vault complex comprises MVP or modified MVP.

### Claim 8 (depends on 1)

The vault complex of claim 1, wherein the vault complex further comprises VPARP or modified VPARP, or a portion of VARP or a modified portion of VPARP.

### Claim 9

A pharmaceutical composition for treating and/or preventing chronic kidney disease in a subject, comprising a cell adhesion modifying substance incorporated within a vault complex, and at least one pharmaceutically acceptable excipient.

### Claim 10 (depends on 9)

The pharmaceutical composition of claim 9, wherein the cell adhesion modifying substance inhibits integrin binding.

### Claim 11 (depends on 9)

The pharmaceutical composition of claim 9, wherein the cell adhesion modifying substance is an RGD-containing peptide.

### Claim 12 (depends on 11)

The pharmaceutical composition of claim 11, wherein the RGD-containing peptide is cyclic.

### Claim 13 (depends on 12)

The pharmaceutical composition of claim 12, wherein the RGD-containing peptide is GRGDSP.

### Claim 14 (depends on 12)

The pharmaceutical composition of claim 12, wherein the cyclic RGD-containing peptide is attached to mINT.

### Claim 15 (depends on 9)

The pharmaceutical composition of claim 9, wherein the vault complex comprises MVP or modified MVP.

### Claim 16 (depends on 9)

The pharmaceutical composition of claim 9, wherein the vault complex further comprises VPARP or modified VPARP, or a portion of VARP or a modified portion of VPARP.

### Claim 17

A method of treating and/or preventing chronic kidney disease in a subject, comprising administering to the subject an effective amount of a cell adhesion modifying substance incorporated within a vault complex.

### Claim 18 (depends on 17)

The method of claim 17, wherein the cell adhesion modifying substance inhibits integrin binding.

### Claim 19 (depends on 17)

The method of claim 17, wherein the cell adhesion modifying substance is an RGD-containing peptide.

### Claim 20 (depends on 19)

The method of claim 19, wherein the RGD-containing peptide is cyclic.

### Claim 21 (depends on 20)

The method of claim 20, wherein the RGD-containing peptide is GRGDSP.

### Claim 22 (depends on 20)

The method of claim 20, wherein the cyclic RGD-containing peptide is attached to mINT.

### Claim 23 (depends on 17)

The method of claim 17, wherein the vault complex comprises MVP or modified MVP.

### Claim 24 (depends on 17)

The method of claim 17, wherein the vault complex further comprises VPARP or modified VPARP, or a portion of VARP or a modified portion of VPARP.

### Claim 25 (depends on 17)

The chronic kidney disease of claim 17, wherein the disease is caused by diabetic nephropathy.
