Pharmaceutical composition for treating lysosomal storage disease
AssigneeOsaka University
InventorsKeiichi Ozono, Takanobu Otomo, Norio Sakai
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Abstract
The purpose of the present invention is to provide, in a simple and also inexpensive manner, a pharmaceutical composition which comprises a plurality of lysosomal enzymes and is effective in treating lysosomal storage disease caused by a deficiency in a plurality of lysosomal enzymes. Provided is a pharmaceutical composition for treating lysosomal storage disease, the composition comprising as an active ingredient a lysosomal enzyme group obtained from cells derived from a subject who does not suffer from lysosomal storage disease.
Claims3
A process for producing a pharmaceutical composition for treating mucolipidosis type II or type III, comprising a group of purified lysosomal enzymes as an active ingredient, the process comprising obtaining the group of lysosomal enzymes by performing the following steps:
- adding to a cell one or more reagents selected from the group consisting of amphiphilic amines, lysosome-tropic amines, ionophores, and V-ATPase inhibitors, followed by culturing;
- collecting a culture supernatant;
- and
- purifying the obtained culture supernatant,
- wherein the group of purified lysosomal enzymes comprises at least α-mannosidase, α-fucosidase, α-galactosidase, α-glucosidase, β-galactosidase, β-glucosidase, β-hexosaminidase, β-glucuronidase, galactocerebrosidase, and cathepsin, and wherein the purified lysosomal enzymes are not recombinant enzymes.
A process for producing a pharmaceutical composition for treating mucolipidosis type II or type III, comprising a group of purified lysosomal enzymes as an active ingredient, the process comprising obtaining the group of lysosomal enzymes by performing the following steps:
- adding to a cell one or more reagents selected from the group consisting of amphiphilic amines, lysosome-tropic amines, ionophores, and V-ATPase inhibitors, followed by culturing, wherein the cell is not a cell into which a gene encoding a lysosomal enzyme has been artificially integrated;
- collecting a culture supernatant;
- and
- purifying the obtained culture supernatant,
- wherein the group of purified lysosomal enzymes comprises at least α-mannosidase, α-fucosidase, α-galactosidase, α-glucosidase, β-galactosidase, β-glucosidase, β-hexosaminidase, β-glucuronidase, galactocerebrosidase, and cathepsin.
Dependencies are inferred from the claim text.