Patents Assigned to Amicus Therapeutics, Inc.
  • Publication number: 20210393603
    Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-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.
    Type: Application
    Filed: August 12, 2021
    Publication date: December 23, 2021
    Applicant: Amicus Therapeutics, Inc.
    Inventors: Jeff Castelli, Elfrida Benjamin
  • Publication number: 20210386723
    Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-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.
    Type: Application
    Filed: August 12, 2021
    Publication date: December 16, 2021
    Applicant: Amicus Therapeutics, Inc.
    Inventors: Jeff Castelli, Elfrida Benjamin
  • Publication number: 20210379034
    Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-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.
    Type: Application
    Filed: August 12, 2021
    Publication date: December 9, 2021
    Applicant: Amicus Therapeutics, Inc.
    Inventors: Jeff Castelli, Elfrida Benjamin
  • Publication number: 20210379035
    Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-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.
    Type: Application
    Filed: August 12, 2021
    Publication date: December 9, 2021
    Applicant: Amicus Therapeutics, Inc.
    Inventors: Jeff Castelli, Elfrida Benjamin
  • Publication number: 20210379036
    Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-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.
    Type: Application
    Filed: August 12, 2021
    Publication date: December 9, 2021
    Applicant: Amicus Therapeutics, Inc.
    Inventors: Jeff Castelli, Elfrida Benjamin
  • Publication number: 20210379037
    Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-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.
    Type: Application
    Filed: August 13, 2021
    Publication date: December 9, 2021
    Applicant: Amicus Therapeutics, Inc.
    Inventors: Jeff Castelli, Elfrida Benjamin
  • Publication number: 20210361633
    Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-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.
    Type: Application
    Filed: August 4, 2021
    Publication date: November 25, 2021
    Applicant: Amicus Therapeutics, Inc.
    Inventors: Jeff Castelli, Elfrida Benjamin
  • Publication number: 20210315875
    Abstract: Provided are methods of treating a patient diagnosed with Fabry disease and methods of enhancing ?-galactosidase A in a patient diagnosed with or suspected of having Fabry disease. Certain methods comprise administering to a patient a therapeutically effective dose of a pharmacological chaperone for ?-galactosidase A, wherein the patient has a mutation in the nucleic acid sequence encoding ?-galactosidase A. Also described are uses of pharmacological chaperones for the treatment of Fabry disease and compositions for use in the treatment of Fabry disease.
    Type: Application
    Filed: January 16, 2019
    Publication date: October 14, 2021
    Applicant: Amicus Therapeutics, Inc.
    Inventor: Elfrida Benjamin
  • Patent number: 11097015
    Abstract: Provided herein are polypeptides comprising one or more non-native cysteine residues that form a disulfide bridge between non-native cysteines within the protein or between non-native cysteines of two monomers of the protein. Such modified human polypeptides are useful in treatment of genetic diseases via enzyme replacement therapy and/or gene therapy.
    Type: Grant
    Filed: October 10, 2019
    Date of Patent: August 24, 2021
    Assignee: Amicus Therapeutics, Inc.
    Inventors: Hung Do, Ce Feng Liu
  • Publication number: 20210251971
    Abstract: The present invention provides methods to determine whether a patient with a lysosomal storage disorder will benefit from treatment with a specific pharmacological chaperone. The present invention exemplifies an in vitro method for determining ?-galactosidase A responsiveness to a pharmacological chaperone such as 1-deoxygalactonojirimycin in a cell line expressing a mutant from of ?-galactosidase A. The invention also provides a method for diagnosing Fabry disease in patients suspected of having Fabry disease.
    Type: Application
    Filed: October 26, 2020
    Publication date: August 19, 2021
    Applicant: AMICUS THERAPEUTICS, INC.
    Inventors: Elfrida Benjamin, Hung V. Do, Xiaoyang Wu, John Flanagan, Brandon Alan Wustman
  • Publication number: 20210251972
    Abstract: Provided are methods for the treatment of Fabry disease in a patient, such as reducing the risk of composite clinical outcomes. Also provided are methods for assessing various symptoms of Fabry disease such as gastrointestinal symptoms. Also provided are methods of evaluating treatment therapies for Fabry disease.
    Type: Application
    Filed: February 10, 2021
    Publication date: August 19, 2021
    Applicant: Amicus Therapeutics, Inc.
    Inventor: Nina Skuban
  • Publication number: 20210220344
    Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-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.
    Type: Application
    Filed: March 26, 2021
    Publication date: July 22, 2021
    Applicant: Amicus Therapeutics, Inc.
    Inventors: Jeff Castelli, Elfrida Benjamin
  • Patent number: 11033538
    Abstract: The present invention provides dosing regimens for administering pharmacological chaperones to a subject in need thereof. The dosing regimens can be used to treat disorders caused by improper protein misfolding, such as lysosomal storage disorders.
    Type: Grant
    Filed: June 18, 2018
    Date of Patent: June 15, 2021
    Assignee: Amicus Therapeutics, Inc.
    Inventors: Jeff Castelli, David J. Lockhart
  • Publication number: 20210162075
    Abstract: Provided herein are improved gene therapy vectors and methods of use, in some embodiments, comprising sequences for improved expression and cellular targeting of a therapeutic protein.
    Type: Application
    Filed: December 18, 2020
    Publication date: June 3, 2021
    Applicant: Amicus Therapeutics, Inc.
    Inventors: Hung Do, Steven Tuske, Russell Gotschall, Ce Feng Liu
  • Patent number: 10961522
    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.
    Type: Grant
    Filed: January 18, 2019
    Date of Patent: March 30, 2021
    Assignee: Amicus Therapeutics, Inc.
    Inventors: Russell Gotschall, Hung V. Do
  • Publication number: 20210085661
    Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-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.
    Type: Application
    Filed: October 21, 2020
    Publication date: March 25, 2021
    Applicant: Amicus Therapeutics, Inc.
    Inventors: Jeff Castelli, Elfrida Benjamin
  • Publication number: 20210085660
    Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-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.
    Type: Application
    Filed: October 21, 2020
    Publication date: March 25, 2021
    Applicant: Amicus Therapeutics, Inc.
    Inventors: Jeff Castelli, Elfrida Benjamin
  • Publication number: 20210069161
    Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-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.
    Type: Application
    Filed: October 22, 2020
    Publication date: March 11, 2021
    Applicant: Amicus Therapeutics, Inc.
    Inventors: Jeff Castelli, Elfrida Benjamin
  • Publication number: 20210069162
    Abstract: Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in ?-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.
    Type: Application
    Filed: October 22, 2020
    Publication date: March 11, 2021
    Applicant: Amicus Therapeutics, Inc.
    Inventors: Jeff Castelli, Elfrida Benjamin
  • Patent number: RE48608
    Abstract: The present invention provides methods to determine whether a patient with a lysosomal storage disorder will benefit from treatment with a specific pharmacological chaperone. The present invention exemplifies an in vitro method for determining ?-galactosidase A responsiveness to a pharmacological chaperone such as 1-deoxygalactonojirimycin in a cell line expressing a mutant from of ?-galactosidase A. The invention also provides a method for diagnosing Fabry disease in patients suspected of having Fabry disease.
    Type: Grant
    Filed: December 17, 2018
    Date of Patent: June 29, 2021
    Assignee: Amicus Therapeutics, Inc.
    Inventors: Elfrida Benjamin, Hung V. Do, John Flanagan, Xiaoyang Wu, Brandon Wustman