William S Sly

US Patent:

7943126, May 17, 2011

Filed:

Mar 17, 2009

Appl. No.:

12/405920

Inventors:

Shunji Tomatsu - Saint Louis MO, US
William S Sly - Saint Louis MO, US
Jeffrey H Grubb - Saint Louis MO, US
Tatsuo Nishioka - Kanazawa, JP
Ken-ichi Miyamoto - Kanazawa, JP
Seiji Yamaguchi - Izumo, JP

Assignee:

Saint Louis University - Saint Louis MO

International Classification:

A61K 38/43
A61P 19/08
C12N 9/00
C12P 21/02

US Classification:

424 941, 514 12, 530350, 435 691, 435183

Abstract:

The present invention provides compositions and methods for use in enzyme replacement therapy. The inventors disclose a method of producing membrane bound enzymes in an active soluble form by eliminating the glycosylphosphatidylinositol (GPI) membrane anchor. In particular the inventors disclose a soluble active form of the membrane bound enzyme TNSALP which they produced by deleting the GPI anchor single peptide sequence. They have further shown that this composition is useful for treatment of hypophosphatasia. The inventors also disclose oligo acid amino acid variants thereof which specifically target bone tissue.

US Patent:

20110250187, Oct 13, 2011

Filed:

Mar 24, 2011

Appl. No.:

13/071445

Inventors:

Shunji Tomatsu - Saint Louis MO, US
William Sly - Saint Louis MO, US
Jeffrey Grubb - Saint Louis MO, US
Tatsuo Nishioka - Kanazawa, JP
Ken-ichi Miyamoto - Kanazawa, JP
Seiji Yamaguchi - Izumo, JP

Assignee:

Saint Louis University - Saint Louis MO

International Classification:

A61K 35/54
A61P 3/00
A61K 48/00
C12N 9/16
A61K 38/46

US Classification:

424 9321, 435196, 424 946, 514 44 R

Abstract:

The present invention provides compositions and methods for use in enzyme replacement therapy. The inventors disclose a method of producing membrane bound enzymes in an active soluble form by eliminating the glycosylphosphatidylinositol (GPI) membrane anchor. In particular the inventors disclose a soluble active form of the membrane bound enzyme TNSALP which they produced by deleting the GPI anchor single peptide sequence. They have further shown that this composition is useful for treatment of hypophosphatasia. The inventors also disclose oligo acid amino acid variants thereof which specifically target bone tissue.

US Patent:

20140377246, Dec 25, 2014

Filed:

Jun 11, 2014

Appl. No.:

14/302016

Inventors:

- Tucson AZ, US
Jeffrey Grubb - Petaluma CA, US
Daniel Rowan - Wauwatosa WI, US
William Sly - Saint Louis MO, US

Assignee:

Carol Ann Foundation and International Morquio Organization - Tucson AZ

International Classification:

A61K 38/47

US Classification:

424 9461, 424 946

Abstract:

The invention relates to a method of treating mucopolysaccharidoses using enzyme replacement therapy with chemically modified lysosomal enzymes. More specifically the method relates to administering chemically modified lysosomal enzymes intraperitoneal injection. In addition, the invention relates to treating type VII mucopolysaccharidoses or mucopolysaccharidoses type VII related bone lesions with a chemical modified β-glucuronidase, wherein the modified β-glucuronidase may be administered 5 weeks after birth, and or may be administered intraperitoneally.