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Amicus Therapeutics Inc patents


Recent patent applications related to Amicus Therapeutics Inc. Amicus Therapeutics Inc is listed as an Agent/Assignee. Note: Amicus Therapeutics Inc may have other listings under different names/spellings. We're not affiliated with Amicus Therapeutics Inc, we're just tracking patents.

ARCHIVE: New 2018 2017 2016 2015 2014 2013 2012 2011 2010 2009 | Company Directory "A" | Amicus Therapeutics Inc-related inventors


Methods for treatment of fabry disease

Provided are in vitro and in vivo methods for determining whether a patient with fabry disease will respond to treatment with a specific pharmacological chaperone.. . ... Amicus Therapeutics Inc

Augmented acid alpha-glucosidase for the treatment of pompe disease

A method for treating pompe disease including administration of recombinant human acid α-glucosidase having optimal glycosylation with mannose-6-phosphate residues in combination with an amount of miglustat effective to maximize tissue uptake of recombinant human acid α-glucosidase while minimizing inhibition of the enzymatic activity of the recombinant human acid α-glucosidase is provided.. . ... Amicus Therapeutics Inc

Method for the treatment of pompe disease using 1-deoxynojirimycin derivatives

The present invention provides a method for increasing the activity of a mutant or wild-type α-glucosidase enzyme in vitro and in vivo by contacting the enzyme with a specific pharmacological chaperone which is a derivative of 1-deoxynojirimycin. The invention also provides a method for the treatment of pompe disease by administration of chaperone small molecule compound which is a derivative of 1-deoxynojirimycin. ... Amicus Therapeutics Inc

Chemical crosslinkers

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.. ... Amicus Therapeutics Inc

Dosing regimens for the treatment of fabry disease

The presently disclosed subject matter provides a dosing regimen and administration schedule for the use of 1-deoxygalactonojirimycin and enzyme replacement therapy for the treatment of fabry disease. The presently disclosed subject matter further provides a dosing regimen and administration schedule for the use of migalastat hydrochloride and agalsidase for the treatment of fabry disease.. ... Amicus Therapeutics Inc

Treatment of cns disorders associated with mutations in genes encoding lysosomal enzymes

Described is a method for treating an individual having a neurological disorder with an associated mutation or mutations in a gene encoding a lysosomal enzyme. Specifically, the individual is administered a specific pharmacological chaperone for the lysosomal enzyme which increases trafficking of the protein from the er to the lysosome in neural cells, with or without concomitantly increasing enzyme activity in neural cells. ... Amicus Therapeutics Inc

Stable parenteral dnj compositions

A stable pharmaceutical composition that includes an active agent selected from 1-deoxynojirimycin, a pharmaceutically acceptable salt thereof, or a derivative thereof, and a buffer, wherein the stable pharmaceutical composition is capable of being parenterally administered to a human without deleterious health effects. Pompe disease is an example of a lysosomal storage disorder. ... Amicus Therapeutics Inc

Novel compounds for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system

Described are novel salts of the compound (3r,4r,5s)-5-(difluoromethyl) piperidine-3,4-diol, as well as methods of using the same for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system. In particular, the present invention provides methods for preventing and/or treating gaucher's disease and/or parkinson's disease.. ... Amicus Therapeutics Inc

Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation

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.. ... Amicus Therapeutics Inc

Method for selection of high m6p recombinant proteins

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. ... Amicus Therapeutics Inc

Formulations comprising recombinant acid alpha-glucosidase

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.. ... Amicus Therapeutics Inc

Methods for coupling targeting peptides onto recombinant lysosomal enzymes for improved treatments of lysosomal storage diseases

Described herein are methods of making targeting peptides conjugated to a recombinant lysosomal enzyme by modifying the amino (n)-terminus and one or more lysine residues on a recombinant human lysosomal enzyme using a first crosslinking agent to give rise to a first crosslinking agent modified recombinant human lysosomal enzyme, modifying a lysine or cysteine within a short extension linker at the carboxyl (c)-terminus on a variant igf-2 peptide having a short extension linker using a second crosslinking agent to give rise to a second crosslinking agent modified variant igf-2 peptide, and then conjugating the first crosslinking agent modified recombinant human lysosomal enzyme to the second crosslinking agent modified variant igf-2 peptide containing a short extension linker. Also described herein are conjugates synthesized using the methods disclosed herein. ... Amicus Therapeutics Inc

Dosing regimens for treating and/or preventing cerebral amyloidoses

Described herein are dosing regimens and kits for the treatment and/or prevention of cerebral amyloidoses such as alzheimer's disease (ad) and/or cerebral amyloid angiopathy (caa).. . ... Amicus Therapeutics Inc

Methods of treating fabry disease in patients having the g9331a mutation in the gla gene

Provided are methods of treating a patient diagnosed with fabry disease and methods of enhancing α-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 α-galactosidase a, wherein the patient has a splice site mutation in intron 4 of the nucleic acid sequence encoding α-galactosidase a. ... Amicus Therapeutics Inc

07/27/17 / #20170209548

Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation for the treatment of fabry disease

The present application provides 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. ... Amicus Therapeutics Inc

07/06/17 / #20170190665

Sugar derivatives comprising sulfur-containing moieties and methods of making same and methods of using the same for the treatment of mps iiic

Described herein are modified sugar, iminosugar and azasugar compounds and methods of making same. One or more of these modified compounds contain sulfates, sulfites, sulfamates and/or sulfonamides. ... Amicus Therapeutics Inc

07/06/17 / #20170189497

Augmented acid alpha-glucosidase for the treatment of pompe disease

A method for treating pompe disease including administration of recombinant human acid α-glucosidase having optimal glycosylation with mannose-6-phosphate residues in combination with an amount of miglustat effective to maximize tissue uptake of recombinant human acid α-glucosidase while minimizing inhibition of the enzymatic activity of the recombinant human acid α-glucosidase is provided.. . ... Amicus Therapeutics Inc

06/15/17 / #20170166588

Compounds and methods for the treatment of alzheimer's disease and/or cerebral amyloid angiopathy

Described herein are novel compounds and methods for the treatment and/or prevention of cerebral amyloidoses such as alzheimer's disease (ad) and/or cerebral amyloid angiopathy (caa).. . ... Amicus Therapeutics Inc

06/08/17 / #20170157220

Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity

Described herein are variant, recombinant β-glucocerebrosidase proteins characterized as having increased stability relative to recombinant wild-type β-glucocerebrosidase. Also provided herein are variant, recombinant β-glucocerebrosidase proteins characterized as retaining more catalytic activity relative to recombinant wild-type β-glucocerebrosidase. ... Amicus Therapeutics Inc

03/02/17 / #20170056483

High concentration alpha-glucosidase compositions for the treatment of pompe disease

The present application provides for compositions comprising high concentrations of acid α-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.. ... Amicus Therapeutics Inc

03/02/17 / #20170056384

Regimens for treating and preventing lysosomal disorders and degenerative disorders of the central nervous system

Described herein are dosing regimens and kits for the treatment and/or prevention of lysosomal storage disorders such as gaucher disease. Also described are dosing regimens and kits for the treatment and/or prevention of degenerative disorders of the central nervous system, such as the synucleinopathies parkinson's disease or lewy body dementia.. ... Amicus Therapeutics Inc

02/16/17 / #20170044102

New method for preparing isofagomine and its derivatives

A method for preparing isofagomine, its derivatives, intermediates and salts thereof using novel processes to make isofagomine from d-(−)-arabinose and l-(−)-xylose.. . ... Amicus Therapeutics Inc

02/16/17 / #20170042868

Methods for treatment of fabry disease

Provided are in vitro and in vivo methods for determining whether a patient with fabry disease will respond to treatment with a specific pharmacological chaperone.. . ... Amicus Therapeutics Inc

02/02/17 / #20170027919

Method for the treatment of neurological disorders by enhancing the activity of beta-glucocerebrosidase

Provided is a method of increasing the stability of wild-type β-glucocerebrosidase. Also provided are methods of treating and/or preventing an individual having a neurological disease in which increased expression or activity of β-glucocerebrosidase in the central nervous system would be beneficial. ... Amicus Therapeutics Inc

01/26/17 / #20170020853

Novel compounds for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system

Described are novel salts of the compound (3r,4r,5s)-5-(difluoromethyl) piperidine-3,4-diol, as well as methods of using the same for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system. In particular, the present invention provides methods for preventing and/or treating gaucher's disease and/or parkinson's disease.. ... Amicus Therapeutics Inc

01/05/17 / #20170003301

Method to predict response to pharmacological chaperone treatment of diseases

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 α-galactosidase a responsiveness to a pharmacological chaperone such as 1-deoxygalactonojirimycin in a cell line expressing a mutant from of α-galactosidase a. ... Amicus Therapeutics Inc








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