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公开(公告)号:US11097015B2
公开(公告)日:2021-08-24
申请号:US16598960
申请日:2019-10-10
发明人: Hung Do , Ce Feng Liu
摘要: 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.
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公开(公告)号:US20220023443A1
公开(公告)日:2022-01-27
申请号:US17363333
申请日:2021-06-30
发明人: Hung Do , Ce Feng Liu
摘要: 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.
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公开(公告)号:US10660972B2
公开(公告)日:2020-05-26
申请号:US15347006
申请日:2016-11-09
发明人: Hung Do
摘要: 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. Also described herein are treatment methods using the disclosed conjugates.
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公开(公告)号:US12064485B2
公开(公告)日:2024-08-20
申请号:US17363333
申请日:2021-06-30
发明人: Hung Do , Ce Feng Liu
CPC分类号: A61K48/0058 , A61K38/465 , A61K38/47 , A61K48/0033 , A61P43/00 , C12N15/86 , C07H21/04 , C12N15/79 , C12N2750/14143 , C12N2800/00 , C12Y301/02022 , C12Y302/01022
摘要: 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.
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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:US10814008B2
公开(公告)日:2020-10-27
申请号:US15891397
申请日:2018-02-08
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.
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公开(公告)号:US20210162075A1
公开(公告)日:2021-06-03
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:US10512676B2
公开(公告)日:2019-12-24
申请号:US15473999
申请日:2017-03-30
发明人: Hing Char , Sergey Tesler , Wendy Sunderland , Enrique Diloné , Russell Gotschall , Hung Do
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:US11491211B2
公开(公告)日:2022-11-08
申请号:US16654521
申请日:2019-10-16
发明人: Hing Char , Sergey Tesler , Wendy Sunderland , Enrique Diloné , Russell Gotschall , Hung Do
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:US20200171133A1
公开(公告)日:2020-06-04
申请号:US16654521
申请日:2019-10-16
发明人: Hing Char , Sergey Tesler , Wendy Sunderland , Enrique Diloné , Russell Gotschall , Hung Do
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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