Title:
PHARMACEUTICAL COMBINATION FOR TREATMENT OF FABRY DISEASE AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2018/230628
Kind Code:
A1
Abstract:
Provided are: a pharmaceutical combination for treatment of Fabry disease; and a use thereof. The present invention pertains to a pharmaceutical combination for treatment of Fabry disease, comprising: a protein that has a mutation in the amino acid sequence of α-N-acetylgalactosaminidase, and that exhibits α-galactosidase activity; and an active-site-specific chaperone.
Inventors:
TSUKIMURA TAKAHIRO (JP)
TOGAWA TADAYASU (JP)
SAKURABA HITOSHI (JP)
TOGAWA TADAYASU (JP)
SAKURABA HITOSHI (JP)
Application Number:
PCT/JP2018/022679
Publication Date:
December 20, 2018
Filing Date:
June 14, 2018
Export Citation:
Assignee:
MEIJI PHARMACEUTICAL UNIV (JP)
International Classes:
A61K31/445; A61K31/55; A61K38/47; A61P3/06; A61P43/00; C12N9/24
Domestic Patent References:
| WO2008143354A1 | 2008-11-27 | |||
| WO2007058381A1 | 2007-05-24 | |||
| WO2006368925A1 | ||||
| WO2008143354A1 | 2008-11-27 |
Foreign References:
| JP2003528799A | 2003-09-30 | |||
| JP2012107020A | 2012-06-07 | |||
| JP2012102128A | 2012-05-31 | |||
| JP2014528901A | 2012-09-12 | |||
| US5507242A | 1996-04-16 |
Other References:
ISHII, S.: "Pharmacological chaperone therapy for Fabry disease", PROC. JPN. ACAD., vol. 88, no. 1, 2012, pages 18 - 30, XP055171870
CLARK, NE. ET AL.: "Pharmacological chaperones for human alpha-N-acetylgalactosaminidase", PNAS, vol. 109, no. 43, 2012, pages 17400 - 17405, XP055564877
WARNOCK D ET AL., PLOS ONE, vol. 10, 2015, pages e0134341
LENDERS M ET AL., J AM SOC NEPHROL., vol. 27, 2016, pages 256 - 64
TAJIMA Y ET AL., AM J HUM GENET., vol. 85, 2009, pages 569 - 80
"Molecular Cloning, A Laboratory Manual", 1989, COLD SPRING HARBOR LABORATORY PRESS
"Current Protocols in Molecular Biology", 1987, JOHN WILEY & SONS
See also references of EP 3639825A4
CLARK, NE. ET AL.: "Pharmacological chaperones for human alpha-N-acetylgalactosaminidase", PNAS, vol. 109, no. 43, 2012, pages 17400 - 17405, XP055564877
WARNOCK D ET AL., PLOS ONE, vol. 10, 2015, pages e0134341
LENDERS M ET AL., J AM SOC NEPHROL., vol. 27, 2016, pages 256 - 64
TAJIMA Y ET AL., AM J HUM GENET., vol. 85, 2009, pages 569 - 80
"Molecular Cloning, A Laboratory Manual", 1989, COLD SPRING HARBOR LABORATORY PRESS
"Current Protocols in Molecular Biology", 1987, JOHN WILEY & SONS
See also references of EP 3639825A4
Attorney, Agent or Firm:
HARAKENZO WORLD PATENT & TRADEMARK (JP)
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