公开(公告)号：US10814008B2

公开(公告)日：2020-10-27

申请号：US15891397

申请日：2018-02-08

申请人： AMICUS THERAPEUTICS, INC.

发明人： Hung Do

IPC分类号： A61K47/64 ,  C12N9/16 ,  C12N9/20 ,  C12N9/24 ,  C12N9/26 ,  C12N9/42 ,  C12N9/40 ,  C07K14/65 ,  A61K38/30 ,  A61K38/47 ,  C12N9/96 ,  A61K47/60

摘要： Disclosed herein are methods of chemical conjugation comprising contacting a lysosomal enzyme with a first crosslinking agent to introduce aldehyde groups; contacting a lysosomal targeting peptide with a second crosslinking agent to introduce a hydrazide group at the N-terminal residue; contacting the lysosomal enzyme with aldehyde groups of step a. with the lysosomal targeting peptide with a hydrazide group at the N-terminal residue of step b; and forming a lysosomal enzyme-lysosomal targeting peptide conjugate.

公开(公告)号：US10792279B2

公开(公告)日：2020-10-06

申请号：US16817927

申请日：2020-03-13

申请人： Amicus Therapeutics, Inc.

发明人： Jeff Castelli ,  Elfrida Benjamin

IPC分类号： A61K31/445 ,  A61P13/12 ,  A61K9/48

摘要： Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

公开(公告)号：US20200206210A1

公开(公告)日：2020-07-02

申请号：US16817927

申请日：2020-03-13

申请人： Amicus Therapeutics, Inc.

发明人： Jeff Castelli ,  Elfrida Benjamin

IPC分类号： A61K31/445 ,  A61P13/12

摘要： Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

公开(公告)号：US20200206204A1

公开(公告)日：2020-07-02

申请号：US16817895

申请日：2020-03-13

申请人： AMICUS THERAPEUTICS, INC.

发明人： JEFF CASTELLI ,  ELFRIDA BENJAMIN

IPC分类号： A61K31/445 ,  A61P13/12

摘要： Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

公开(公告)号：US20200206203A1

公开(公告)日：2020-07-02

申请号：US16817881

申请日：2020-03-13

申请人： Amicus Therapeutics, Inc.

发明人： Jeff Castelli ,  Elfrida Benjamin

IPC分类号： A61K31/445 ,  A61P13/12

摘要： Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

公开(公告)号：US20200206202A1

公开(公告)日：2020-07-02

申请号：US16817877

申请日：2020-03-13

申请人： Amicus Therapeutics, Inc.

发明人： Jeff Castelli ,  Elfrida Benjamin

IPC分类号： A61K31/445 ,  A61P13/12

摘要： Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day.

公开(公告)号：US10512677B2

公开(公告)日：2019-12-24

申请号：US16015556

申请日：2018-06-22

申请人： Amicus Therapeutics, Inc.

发明人： Kenneth Valenzano ,  John Crowley ,  Richie Khanna ,  John Flanagan

IPC分类号： A61K47/22 ,  A61K38/47 ,  C12N9/96 ,  A61K9/00 ,  A61K31/7008 ,  A61K31/445 ,  C12N9/26

摘要： The present application provides for compositions comprising high concentrations of acid a-glucosidase in combination with an active site-specific chaperone for the acid α-glucosidase, and methods for treating Pompe disease in a subject in need thereof, that includes a method of administering to the subject such compositions. The present application also provides methods for increasing the in vitro and in vivo stability of an acid α-glucosidase enzyme formulation.

公开(公告)号：US10357548B2

公开(公告)日：2019-07-23

申请号：US15639290

申请日：2017-06-30

申请人： Amicus Therapeutics, Inc.

发明人： Richie Khanna ,  Kenneth Valenzano ,  Susan Elizabeth Fowles

IPC分类号： A61K38/47 ,  C12N9/40 ,  A61K31/445

摘要： The present application provides for compositions comprising α-galactosidase A in combination with an active site-specific chaperone for the α-galactosidase A, and methods for treating Fabry disease in a subject in need thereof, that includes a method of administering to the subject such compositions. The present application also provides methods for increasing the in vitro and in vivo stability of an α-galactosidase A enzyme formulation.

公开(公告)号：USD843850S1

公开(公告)日：2019-03-26

申请号：US29585388

申请日：2016-11-23

申请人： Amicus Therapeutics, Inc.

设计人： Jeffrey LoPrete ,  Muzammil Mustufa ,  Jayne Gershkowitz

公开(公告)号：US10227577B2

公开(公告)日：2019-03-12

申请号：US15473994

申请日：2017-03-30

申请人： Amicus Therapeutics, Inc.

发明人： Hung V. Do ,  Russell Gotschall

IPC分类号： C12N9/26 ,  A61K38/00

摘要： Methods for the production, capturing and purification of recombinant human lysosomal proteins are described. Such recombinant human lysosomal proteins can have high content of mannose-6-phosphate residues. Also described are pharmaceutical compositions comprising such recombinant human lysosomal proteins, as well as methods of treatment and uses of such recombinant human lysosomal proteins.