US 8,569,032 B2Granted

Proteins having acquired A-galactosidase activity

AssigneesTokyo Metropolitan Institute of Medical Science, Altif Laboratories

InventorsHitoshi Sakuraba, Youichi Tajima, Ikuo Kawashima, Seiichi Aikawa, Fumiko Aikawa

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Abstract

The present invention provides a pharmaceutical composition comprising a protein having α-galactosidase activity for treating Fabry disease, which causes no allergic side effect, which is highly stable in blood (plasma) and which can readily be taken up by a cell of an affected organ. The pharmaceutical composition for treating Fabry disease of the invention comprises, for example, a protein which acquires an α-galactosidase activity through alteration of the structure of the active site of wild-type human α-N-acetylgalactosaminidase.

Claims4

Dependent claims

An isolated protein comprising a variant of SEQ ID NO:

6 having α-galactosidase activity, said variant selected from:
(i) the variant of SEQ ID NO:
6 consisting of a substitution of serine 202 by a different amino acid;
(ii) the variant of SEQ ID NO:
6 consisting of a substitution of alanine 205 by a different amino acid;
(iii) a variant of SEQ ID NO:
6 consisting of a substitution of serine 202 by a different amino acid and a substitution of serine 205 by a different amino acid;
and
(iv) a variant of SEQ ID NO:
6 consisting of a substitution of serine 202 by a different amino acid and/or a substitution of serine 205 by a different amino acid and wherein one to ten additional amino acids are deleted, substituted or added.

7 more elements

Dependencies are inferred from the claim text.

Facts

Application: 12/600,497Filed 19 May 2008
Priority: JP 2007-13353618 May 2007
Published: US 2010/0291059 A118 Nov 2010
Granted: 29 Oct 2013
Term ends: Not estimatedThe source records don’t support an estimate.

Classification13 codes

C12N 9/2465
Hydrolysing O- and S- glycosyl compounds: acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase
A61K 38/47
Hydrolases: acting on glycosyl compounds , e.g. cellulases, lactases
A61K 48/005
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy:…
A61P 3/00
Drugs for disorders of the metabolism

Family

US 2010/0291059 A1Pre-grant publication · published 18 Nov 2010
US 8,569,032 B2This patent

From the US filing history. Foreign family members aren’t listed.

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