公开(公告)号：US20250057925A1

公开(公告)日：2025-02-20

申请号：US18943360

申请日：2024-11-11

Applicant: Amicus Therapeutics, Inc.

Inventor： Hung V. Do ,  Russell Gotschall ,  Richie Khanna ,  Yi Lun ,  Hing Char ,  Sergey Tesler ,  Wendy Sunderland ,  Enrique Diloné

IPC: A61K38/47 ,  A61P21/00

Abstract: Provided are a recombinant acid α-glucosidase and pharmaceutical composition comprising a recombinant acid α-glucosidase, wherein the recombinant acid α-glucosidase is expressed in Chinese hamster ovary (CHO) cells and comprises an increased content of N-glycan units bearing one or two mannose-6-phosphate residues when compared to a content of N-glycan units bearing one or two mannose-6-phosphate residues of alglucosidase alfa. Also provided herein are methods of producing, purifying, and formulating the recombinant acid α-glucosidase or pharmaceutical composition for administration to a subject and methods of treating a disease or disorder such as Pompe disease using the recombinant acid α-glucosidase or pharmaceutical composition.

公开(公告)号：US20250041445A1

公开(公告)日：2025-02-06

申请号：US18709415

申请日：2022-11-11

Applicant: The Trustees of the University of Pennsylvania ,  Amicus Therapeutics, Inc.

Inventor： Juliette Hordeaux ,  James M. Wilson ,  Leida Rassouli-Taylor ,  Hung Do ,  Steven Tuske

IPC: A61K48/00 ,  A61K38/46 ,  A61P3/00 ,  C12N15/86

Abstract: Provided herein is a recombinant AAV (rAAV) comprising an AAV capsid and a vector genome packaged therein, wherein the vector genome comprises an AAV 5′ inverted terminal repeat (ITR), an engineered nucleic acid sequence encoding a functional hSGSH, optionally comprising stabilizing amino acid changes, a regulatory sequence which direct expression of hSGSH in a target cell, and an AAV 3′ ITR. Also provided is a pharmaceutical composition comprising a rAAV as described herein in a formulation buffer, and a method of treating a human subject diagnosed with MPS IIIA.

公开(公告)号：US12109205B2

公开(公告)日：2024-10-08

申请号：US17078840

申请日：2020-10-23

Applicant: Amicus Therapeutics, Inc.

Inventor： Jeff Castelli ,  Elfrida Benjamin

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

CPC classification number: A61K31/445 ,  A61P13/12 ,  A61K9/48

Abstract: 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：US20240197706A1

公开(公告)日：2024-06-20

申请号：US18315928

申请日：2023-05-11

Applicant: Amicus Therapeutics, Inc.

Inventor： Franklin Johnson

IPC: A61K31/445

CPC classification number: A61K31/445

Abstract: Provided are methods of improving the pharmacokinetics of migalastat by limiting caffeine intake during migalastat therapy.

公开(公告)号：US20230321065A1

公开(公告)日：2023-10-12

申请号：US18199120

申请日：2023-05-18

Applicant: Amicus Therapeutics, Inc.

Inventor： Jeff Castelli

IPC: A61K31/45 ,  A61K31/445

CPC classification number: A61K31/445 ,  A61K31/45 ,  A61K31/7008

Abstract: Provided are dosing regimens for the treatment of Fabry disease in a patient. Certain methods relate to the treatment of ERT-experienced or ERT-naïve Fabry patients. Certain methods comprise administering to the patient about 123 mg free base equivalent of migalastat for improving left ventricular mass and/or improving podocyte globotriaosylceramide.

公开(公告)号：US20230203465A1

公开(公告)日：2023-06-29

申请号：US18111321

申请日：2023-02-17

Applicant: Amicus Therapeutics, Inc.

Inventor： Russell Gotschall ,  Hung V. Do

IPC: C12N9/40 ,  A61K48/00 ,  C12N15/09

CPC classification number: C12N9/2465 ,  C12Y302/0102 ,  A61K48/005 ,  C12N15/09 ,  C12N2510/02

Abstract: Recombinant human alpha glucosidase (rhGAA) composition derived from CHO cells that contains a more optimized glycan composition consisting of a higher amount of rhGAA containing N-glycans carrying mannose-6-phosphate (M6P) or bis-M6P than conventional rhGAAs, along with low amount of non-phosphorylated high mannose glycans, and low amount of terminal galactose on complex oligosaccharides. Compositions containing the rhGAA, and methods of use are described.

公开(公告)号：US20230158123A1

公开(公告)日：2023-05-25

申请号：US17838741

申请日：2022-06-13

Applicant: Amicus Therapeutics, Inc.

Inventor： Russell Gotschall

IPC: A61K38/47 ,  A61K31/445 ,  C12N9/40 ,  C12P21/00

CPC classification number: A61K38/47 ,  A61K31/445 ,  C12N9/2465 ,  C12P21/005 ,  C12Y302/01022

Abstract: Described are compositions comprising α-galactosidase A enzymes with unique carbohydrate profiles, as well as methods for manufacturing and purifying such enzymes. Also described methods of treating, preventing, and/or ameliorating Fabry Disease by administering such enzymes to a subject in need thereof. Also described are compositions comprising migalastat in combination with such α-galactosidase A enzymes.

公开(公告)号：US11612594B2

公开(公告)日：2023-03-28

申请号：US17077389

申请日：2020-10-22

Applicant: Amicus Therapeutics, Inc.

Inventor： Jeff Castelli ,  Elfrida Benjamin

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

Abstract: 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：US20220387406A1

公开(公告)日：2022-12-08

申请号：US17675536

申请日：2022-02-18

Applicant: Amicus Therapeutics, Inc.

Inventor： Jeff Castelli ,  Elfrida Benjamin

IPC: A61K31/445 ,  A61P13/12

Abstract: 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：US11491211B2

公开(公告)日：2022-11-08

申请号：US16654521

申请日：2019-10-16

Applicant: Amicus Therapeutics, Inc.

Inventor： Hing Char ,  Sergey Tesler ,  Wendy Sunderland ,  Enrique Diloné ,  Russell Gotschall ,  Hung Do

IPC: A61K38/47 ,  C12N9/26 ,  A61K9/00 ,  A61K9/19 ,  A61K47/02 ,  A61K47/12 ,  A61K47/26

Abstract: Provided are pharmaceutical formulations comprising a recombinant acid α-glucosidase, wherein the recombinant acid α-glucosidase is expressed in Chinese hamster ovary (CHO) cells and comprises an increased content of N-glycan units bearing one or two mannose-6-phosphate residues when compared to a content of N-glycan units bearing one or two mannose-6-phosphate residues of alglucosidase alfa; at least one buffer selected from the group consisting of a citrate, a phosphate and combinations thereof; and at least one excipient selected from the group consisting of mannitol, polysorbate 80, and combinations thereof, wherein the formulation has a pH of from about 5.0 to about 7.0. Also provided are methods of treating Pompe disease using these pharmaceutical formulations.