# US 8,540,985 B2 · Use of Hsp70 as a regulator of enzymatic activity

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- Number: US 8,540,985 B2
- Document ID: US8540985B2
- Status: Granted
- Kind: B2
- Application number: 13/001,316
- Filed: 2009-06-26
- Published: 2011-11-24
- Granted: 2013-09-24
- Earliest claimed: 2008-06-26
- Assignees: Orphazyme ApS
- Applicants: Thomas Kirkegaard Jensen; Marja Helena Jaattela
- Inventors: Thomas Kirkegaard Jensen; Marja Helena Jaattela
- Web page: https://patentgrep.com/patent/US8540985B2

## CPC

- A61K 31/4545: containing a six-membered ring with nitrogen as a ring hetero atom, e.g. pipamperone, anabasine
- A61K 31/445: Non condensed piperidines, e.g. piperocaine
- A61K 31/56: Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
- A61K 33/30: Zinc; Compounds thereof
- A61K 38/16: Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof {(enzyme inhibitors A61K38/005)}
- A61K 38/17: from animals; from humans {(enzyme inhibitors A61K38/005)}
- A61K 38/1709: {from mammals}
- A61K 38/43: Enzymes; Proenzymes; Derivatives thereof
- A61K 38/46: Hydrolases (3)
- A61K 38/47: acting on glycosyl compounds (3.2), e.g. cellulases, lactases
- A61K 45/06: Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
- A61P 3/00: Drugs for disorders of the metabolism (of the blood or the extracellular fluid A61P7/00)
- A61P 43/00: Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
- C12Y 306/01003: Adenosine triphosphatase (3.6.1.3)
- 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
- C12Y: ENZYMES

## Abstract

The present invention concerns a method for modulating the enzymatic activity of an enzyme, wherein said enzyme interacts with BMP, said method comprising the step of administering or inducing Hsp70, or a functional fragment or variant thereof, in a form suitable for allowing interaction between BMP and Hsp70, or said functional fragment or variant thereof, and thereby modulating the enzymatic activity of an enzyme interacting with BMP.

## Claims

### Claim 1

A method for treatment of a lysosomal storage disorder selected from the group consisting of Niemann-Pick type A, Niemann-Pick type B, Niemann-Pick type C and Niemann-Pick type D, comprising the administration of a bioactive agent selected from Hsp70, a functional fragment of Hsp70 or variant thereof having at least 95% sequence identity to Hsp70, to an individual in need thereof.

### Claim 2 (depends on 1)

The method according to claim 1, wherein said bioactive agent is formulated as a pharmaceutical composition.

### Claim 3 (depends on 1)

The method according to claim 1, wherein said lysosomal storage disorder is characterised as having residual enzymatic activity of the defective enzyme involved in the disease pathology.

### Claim 4 (depends on 1)

The method according to claim 1, wherein said Hsp70, or functional fragment or variant thereof, has 100% identity to wild-type Hsp70 protein.

### Claim 5 (depends on 1)

The method according to claim 1, wherein said bioactive agent is Hsp70.

### Claim 6 (depends on 1)

The method according to claim 1, wherein said Hsp70 is full length Hsp70.

### Claim 7 (depends on 1)

The method according to claim 1, wherein said bioactive agent is a functional fragment or variant of Hsp70 having at least 95% sequence identity to Hsp70 .

### Claim 8 (depends on 1)

The method according to claim 1, wherein said functional fragment or variant of Hsp70 comprises all or part of the ATPase domain of Hsp70.

### Claim 9 (depends on 1)

The method according to claim 1, wherein said functional fragment or variant of Hsp70 comprises tryptophan at amino acid position 90 of the Hsp70 ATPase domain.

### Claim 10 (depends on 1)

The method according to claim 1, wherein said Hsp70, or a functional fragment or variant thereof, is recombinant Hsp70 (rHsp70).

### Claim 11 (depends on 1)

The method according to claim 1, wherein said Hsp70 is derived from a mammal selected from the group consisting of human (homo sapiens), mouse (mus musculus), cow, dog, rat, ferret, pig, sheep, and monkey.