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公开(公告)号：US12042488B2
公开(公告)日：2024-07-23
申请号：US18326274
申请日：2023-05-31
发明人： Jeff Castelli , Elfrida Benjamin
IPC分类号： A61K31/445 , A61P13/12 , A61K9/48
CPC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.
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公开(公告)号：US20240066104A1
公开(公告)日：2024-02-29
申请号：US18053151
申请日：2022-11-07
发明人： Hing Char , Sergey Tesler , Wendy Sunderland , Enrique Diloné , Russell Gotschall , Hung V. Do
CPC分类号： A61K38/47 , A61K9/0095 , A61K9/19 , A61K47/02 , A61K47/12 , A61K47/26 , C12N9/2408 , C12Y302/0102
摘要： 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.
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公开(公告)号：US20240043494A1
公开(公告)日：2024-02-08
申请号：US18040866
申请日：2021-08-06
发明人： Joseph Bauman , Xiaoyang Wu , Yuliya McAnany
IPC分类号： C07K14/705 , C07K14/47
CPC分类号： C07K14/705 , C07K14/47 , C07K2319/02 , C07K2319/03
摘要： The disclosure provides a novel method for treating genetic disorders where a peptide sequence targets proteins produced via gene therapy into exosomes. These protein-loaded exosomes can enter into non-transduced cells and correct pathology. Also, gene therapy compositions, protein replacement therapy composition, pharmaceutical compositions, methods of treatment, and uses of the gene therapy compositions and the recombinant proteins are also disclosed. The method can also be used to improve in vitro recombinant protein yield.
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54.发明公开公开(公告)号：US20230348388A1
公开(公告)日：2023-11-02
申请号：US18214002
申请日：2023-06-26
发明人： Kamlesh Sheth , Sergey Tesler , James Cartwright , Clive King , Wendy Cross
IPC分类号： C07D211/46
CPC分类号： C07D211/46 , C07B2200/13
摘要： Provided are methods of producing Active Pharmaceutical Ingredient (API) grade migalastat hydrochloride, and for purifying intermediate grade migalastat hydrochloride. Further provided are methods of producing [(2R,3S,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol hydrochloride (lucerastat hydrochloride) and other 1-deoxygalactonojirimycin compounds, as well as methods of purifying intermediate grade lucerastat hydrochloride and other 1-deoxygalactonojirimycin compounds.
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公开(公告)号：US20230220320A1
公开(公告)日：2023-07-13
申请号：US17640456
申请日：2020-09-04
摘要： Methods for the continuous production, capturing and purification of biologics such as recombinant proteins are described. Also described are pharmaceutical compositions comprising such biologics, as well as methods of treatment and uses of such biologics.
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公开(公告)号：US20230218598A1
公开(公告)日：2023-07-13
申请号：US18069720
申请日：2022-12-21
发明人： Jeff Castelli , Elfrida Benjamin
IPC分类号： A61K31/445 , A61P13/12 , A61K9/48
CPC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.
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公开(公告)号：US11622962B2
公开(公告)日：2023-04-11
申请号：US17077402
申请日：2020-10-22
发明人： Jeff Castelli , Elfrida Benjamin
IPC分类号： A61K38/16 , 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.
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公开(公告)号：US20220370571A1
公开(公告)日：2022-11-24
申请号：US17699927
申请日：2022-03-21
发明人： Hung V. DO , Richie KHANNA , Russell GOTSCHALL
IPC分类号： A61K38/47 , A61K31/445 , A61P3/00 , A61P21/00
摘要： 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.
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公开(公告)号：US11491243B2
公开(公告)日：2022-11-08
申请号：US17127001
申请日：2020-12-18
发明人： Hung Do , Steven Tuske , Russell Gotschall , Ce Feng Liu
摘要： 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.
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公开(公告)号：US20220313670A1
公开(公告)日：2022-10-06
申请号：US17618277
申请日：2020-06-11
发明人： Franklin Johnson
IPC分类号： A61K31/445
摘要： Provided are methods for treatment of Fabry disease in a patient having renal impairment. Certain methods comprise administering to the patient about 100 mg to about 300 mg free base equivalent of migalastat or salt thereof at a frequency of greater than once every other day, such as once every four or seven days. Certain methods comprise measuring lyso-Gb3 and/or migalastat in one or more plasma samples from the patient.
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