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公开(公告)号：US20240318156A1
公开(公告)日：2024-09-26
申请号：US18573298
申请日：2022-06-24
CPC分类号： C12N9/2402 , C12Y302/01052 , A61K38/00
摘要： Disclosed herein are recombinant β-hexosaminidase variant a subunits that form a β-hexosaminidase variant a subunit homodimer that have optimized properties for use in treating Tay-Sachs disease or Sandhoff Disease.
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公开(公告)号：US20240200096A1
公开(公告)日：2024-06-20
申请号：US18479761
申请日：2023-10-02
发明人： Peter COLOSI , Justin ISHIDA , Silvia RAMIREZ
CPC分类号： C12N15/86 , A61P7/00 , A61K45/06 , C12N2750/14121 , C12N2750/14122 , C12N2750/14142 , C12N2750/14143 , C12N2830/008 , C12N2830/48 , C12N2830/85
摘要： The present disclosure relates, in general, to methods for readministering, or redosing, a subject having undergone a first gene therapy regimen with a second, or subsequent, administration of a gene therapy regimen, wherein the first gene therapy vector and second gene therapy vector comprise different AAV capsids but carry a transgene or polynucleotide useful to treat the same disease or disorder.
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公开(公告)号：US20240124849A1
公开(公告)日：2024-04-18
申请号：US18273430
申请日：2022-01-27
IPC分类号： C12N7/00 , C07K14/015 , C12N15/86
CPC分类号： C12N7/00 , C07K14/015 , C12N15/86 , C12N2750/14151
摘要： The present invention provides modified alphaviruses and compositions, methods, and kits for preparing and using, in particular AAV viral particles pseudotyped with capsids, in particular for use in gene therapy and/or diagnostics.
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4.发明授权公开(公告)号：US11918633B2
公开(公告)日：2024-03-05
申请号：US17747697
申请日：2022-05-18
发明人： Debra Lounsbury
CPC分类号： A61K38/51 , A61P3/00 , C12Y403/01024
摘要： Phenylalanine ammonia-lyase (PAL) variants with a greater phenylalanine-converting activity and/or a reduced immunogenicity as compared to a wild-type PAL for therapeutic uses, including the treatment of adolescent subjects having PKU.
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公开(公告)号：US20230322864A1
公开(公告)日：2023-10-12
申请号：US18063589
申请日：2022-12-08
发明人： Peter Cameron COLOSI , Michael LOCHRIE , Robert NG
IPC分类号： C07K14/005 , C12N15/86 , A61P21/00
CPC分类号： C07K14/005 , C12N15/86 , A61P21/00 , C12N2750/14122 , C12N2750/14143
摘要： The invention relates to novel adeno-associated virus (AAV) capsid proteins, AAV particles comprising a novel capsid protein, polynucleotides encoding these capsid proteins and AAV vectors expressing these capsid proteins. The invention also relates to methods of making the herein described AAV vectors expressing the novel capsid proteins of the invention and associated therapeutic uses of thereof.
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公开(公告)号：US20230192799A1
公开(公告)日：2023-06-22
申请号：US17642150
申请日：2020-09-16
CPC分类号： C07K14/58 , A61K47/542 , A61P19/08 , A61K38/00
摘要： The present disclosure, relates, in general, to stable variants of C-type natriuretic peptide (CNP) and uses thereof to treat bone-related disorders.
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公开(公告)号：US20230089374A1
公开(公告)日：2023-03-23
申请号：US17751144
申请日：2022-05-23
IPC分类号： C07D249/04 , C07D401/12 , C07D401/14 , C07D403/12 , C07D405/12 , C07D409/12 , C07D417/12
摘要： Described herein are compounds, methods of making such compounds, pharmaceutical compositions and medicaments containing such compounds, and methods of using such compounds to treat or prevent diseases or disorders associated with the enzyme glycolate oxidase (GO). Such diseases or disorders include, for example, disorders of glyoxylate metabolism, including primary hyperoxaluria, that are associated with production of excessive amounts of oxalate.
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公开(公告)号：US20220402974A1
公开(公告)日：2022-12-22
申请号：US16319877
申请日：2017-07-25
发明人： Peter Cameron Colosi , Michael LOCHRIE , Robert NG
IPC分类号： C07K14/005 , C12N15/86
摘要： The invention relates to novel adeno-associated virus (AAV) capsid proteins, AAV particles comprising a novel capsid protein, polynucleotides encoding these capsid proteins and AAV vectors expressing these capsid proteins. The invention also relates to methods of making the herein described AAV vectors expressing the novel capsid proteins of the invention and associated therapeutic uses of thereof.
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9.发明授权公开(公告)号：US11345904B2
公开(公告)日：2022-05-31
申请号：US16078546
申请日：2017-02-24
摘要： The present disclosure relates in general to therapeutic lysosomal enzyme fusion proteins useful for treating lysosomal storage diseases, liquid formulations comprising such fusion proteins and associated methods useful for treating lysosomal storage diseases in mammals.
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公开(公告)号：US11254725B2
公开(公告)日：2022-02-22
申请号：US16378163
申请日：2019-04-08
发明人： Mika Aoyagi-Scharber , Teresa Margaret Christianson , Melita Dvorak-Ewell , Daniel J. Wendt , Shinong Long , Jonathan LeBowitz , Daniel Solomon Gold
摘要： The present invention relates in general to therapeutic fusion proteins useful to treat lysosomal storage diseases and methods for treating such diseases. Exemplary therapeutic fusion proteins comprise a lysosomal enzyme, a lysosomal targeting moiety, e.g., an IGF-II peptide, and a spacer peptide. Also provided are compositions and methods for treating Mucopolysaccharidosis Type IIIB (Sanfilippo B Syndrome), comprising a targeted therapeutic fusion protein comprising alpha-N-acetylglucosaminidase (Naglu), a lysosomal targeting moiety, e.g., an IGF-II peptide, and a spacer peptide.
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