Altif Laboratories

Tokyo Metropolitan Institute of Medical Science

Tokyo Metropolitan Organization for Medical Research

National Institute of Advanced Industrial Science and Technology

Jcr Pharmaceuticals Co., Ltd.

Hitoshi Sakuraba

Youichi Tajima

Seiichi Aikawa

Ikuo Kawashima

Kazuo Kobayashi

Shinji Takeoka

Mai Ito

Yasunori Chiba

Makoto Takeuchi

Atsushi Mizuno

Fumiko Aikawa

Yoshifumi Jigami

Yoriko Takeuchi, Legal Representative

Katsuji Ohta

Manabu Ito

Yoshifumi c/o Tsukuba Center Jigami

Yasunori c/o Tsukuba Center Chiba

Kazuo c/o Bio Process R&D Lab Kobayashi

The present invention provides a pharmaceutical composition comprising a protein having &#x3b1;-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 &#x3b1;-galactosidase activity through alteration of the structure of the active site of wild-type human &#x3b1;-N-acetylgalactosaminidase.

Polynucleotides encoding proteins having acquired &#x3b1;-galactosidase activity

The present invention provides, as an enzyme which can be used for enzyme replacement therapy for Fabry disease, a protein having &#x3b1;-galactosidase activity, which shows no allergic adverse side effect, shows a high stability in blood, and can be easily incorporated into a cell of an affected organ. The protein of the present invention is a protein which has acquired &#x3b1;-galactosidase activity by changing the structure of the active site of wild-type human &#x3b1;-N-acetylgalactosaminidase.

Highly functional enzyme having alpha-galactosidase activity

Highly functional enzyme having &#x3b1;-galactosidase activity

Proteins having acquired A-galactosidase activity

A process for producing a lysosomal enzyme having a mannose-6-phosphate-containing acidic sugar chain, wherein the process comprising: culturing in a medium yeast cells obtained by introducing a lysosomal enzyme gene into a sugar chain biosynthetic enzyme gene mutant strain of yeast, collecting a lysosomal enzyme having a phosphate-containing sugar chain from the culture, and then treating the enzyme with &#x3b1;-mannosidase; and pharmaceutical compositions for treatment of human lysosomal enzyme deficiencies produced by the process. The genetic engineering technique using the yeast according to the present invention allows large-amount and high-purity production of a glycoprotein having a phosphate-containing acidic sugar chain which can serve as a labeling marker for transporting into lysosomes in cells of mammals such as human. The glycoprotein having a phosphate-containing acidic sugar chain according to the invention may be utilized as a drug effective in treatment of human lysosomal enzyme deficiencies, etc.

Glycoprotein and process for producing the same

Growing community of inventors

Chiba, Japan

Hitoshi Sakuraba