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公开(公告)号：US12043665B2
公开(公告)日：2024-07-23
申请号：US17306509
申请日：2021-05-03
Applicant: Genzyme Corporation
Inventor： Yunxiang Zhu , Jozsef Karman , Ronnie Wei , Canwen Jiang , Seng Cheng
IPC: C07K16/28 , A61K38/17 , A61K39/395 , A61P37/06 , C07K14/47 , C07K14/705 , C07K14/74
CPC classification number: C07K16/2803 , C07K14/47 , C07K14/70532 , C07K14/70539 , C07K2319/00
Abstract: The present invention provides a bispecific biologic comprising a ligand specific for CTLA-4 and a ligand specific for a pMHC complex.
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公开(公告)号：US11028170B2
公开(公告)日：2021-06-08
申请号：US15788433
申请日：2017-10-19
Applicant: Genzyme Corporation
Inventor： Yunxiang Zhu , Jozsef Karman , Ronnie Wei , Canwen Jiang , Seng Cheng
IPC: C07K14/47 , C07K14/705 , C07K19/00 , A61K38/17 , A61K39/395 , C07K16/28 , C07K14/74
Abstract: The present invention provides a bispecific biologic comprising a ligand specific for CTLA-4 and a ligand specific for a pMHC complex.
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公开(公告)号：US11369693B2
公开(公告)日：2022-06-28
申请号：US14328621
申请日：2014-07-10
Applicant: Genzyme Corporation
Inventor： James Dodge , Seng Cheng
Abstract: This disclosure provides methods and compositions for treating lysosomal storage diseases in a subject. In one aspect of the invention, a transgene product is delivered to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain. The transgene product may be any that is deficient in a lysosomal storage disease.
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公开(公告)号：US20210380686A1
公开(公告)日：2021-12-09
申请号：US17306509
申请日：2021-05-03
Applicant: Genzyme Corporation
Inventor： Yunxiang Zhu , Jozsef Karman , Ronnie Wei , Canwen Jiang , Seng Cheng
IPC: C07K16/28 , C07K14/74 , C07K14/47 , C07K14/705
Abstract: The present invention provides a bispecific biologic comprising a ligand specific for CTLA-4 and a ligand specific for a pMHC complex.
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公开(公告)号：US10111962B2
公开(公告)日：2018-10-30
申请号：US14431115
申请日：2013-09-24
Applicant: Genzyme Corporation
Inventor： Andrew Leger , Bruce Wentworth , Carol A. Nelson , Timothy E. Weeden , Nicholas Clayton , Seng Cheng
IPC: A61K48/00 , A61K47/48 , C12N15/113 , A61K31/712
Abstract: Provided herein are peptide-linked morpholino (PPMO) antisense oligonucleotides that target the poly CUG repeat tract in the 3′ untranslated region of the gene encoding dystrophia myotonica-protein kinase (DMPK) and methods for systemic administration of the same for the treatment of mytonic dystrophy type I (DM1).
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Patent Agency Ranking
公开(公告)号：US20240384271A1
公开(公告)日：2024-11-21
申请号：US18626784
申请日：2024-04-04
Applicant: Genzyme Corporation
Inventor： Seng Cheng , Sarah Melissa Jacobo , Takako Moriguchi , Catherine O'Riordan , Guoxiang Ruan
IPC: C12N15/113 , A61K45/06 , A61P21/00 , C12N15/86
Abstract: Provided herein are RNAi molecules for treating myotonic dystrophy type 1 (DM1). Further provided herein are expression cassettes, vectors (e.g., rAAV), viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat DM1.
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公开(公告)号：US20230365968A1
公开(公告)日：2023-11-16
申请号：US18296283
申请日：2023-04-05
Applicant: Genzyme Corporation
Inventor： Seng Cheng , Sarah Melissa Jacobo , Takako Moriguchi , Catherine O'Riordan , Guoxiang Ruan
IPC: C12N15/113 , C12N15/86 , A61P21/00
CPC classification number: C12N15/113 , C12N15/86 , A61P21/00 , C12N2750/14143
Abstract: Provided herein are RNAi molecules for treating myotonic dystrophy type 1 (DM1). Further provided herein are expression cassettes, vectors (e.g., rAAV), viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat DM1.
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8.发明申请公开(公告)号：US20160120842A1
公开(公告)日：2016-05-05
申请号：US14994489
申请日：2016-01-13
Applicant: Genzyme Corporation
Inventor： Hanlan Liu , Chris Willis , Renu Bhardwaj , Diane P. Copeland , Abizer Harianawala , Jeffrey Skell , John Marshall , Jianmei Kochling , Gerard Palace , Judith Peterschmitt , Craig Siegel , Seng Cheng
IPC: A61K31/4025
CPC classification number: A61K31/4025 , A61K45/06 , C07D319/16 , C07D405/06
Abstract: The hemitartrate salt of a compound represented by the following structural formula: (Formula I Hemitartrate), which may be used in pharmaceutical applications, are disclosed. Particular single crystalline forms of the Formula (I) Hemitartrate are characterized by a variety of properties and physical measurements. As well, methods of producing crystalline Formula (I) Hemitartrate, and using it to inhibit glucosylceramide synthase or lowering glycosphingolipid concentrations in subjects to treat a number of diseases, are also discussed. Pharmaceutical compositions are also described.
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公开(公告)号：US20250002578A1
公开(公告)日：2025-01-02
申请号：US18741190
申请日：2024-06-12
Applicant: GENZYME CORPORATION
Inventor： Yunxiang Zhu , Jozsef Karman , Ronnie Wei , Canwen Jiang , Seng Cheng
IPC: C07K16/28 , C07K14/47 , C07K14/705 , C07K14/74
Abstract: The present invention provides a bispecific biologic comprising a ligand specific for CTLA-4 and a ligand specific for a pMHC complex.
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公开(公告)号：US20180030134A1
公开(公告)日：2018-02-01
申请号：US15788433
申请日：2017-10-19
Applicant: Genzyme Corporation
Inventor： Yunxiang Zhu , Jozsef Karman , Ronnie Wei , Canwen Jiang , Seng Cheng
IPC: C07K16/28 , C07K14/47 , C07K14/705 , C07K14/74
Abstract: The present invention provides a bispecific biologic comprising a ligand specific for CTLA-4 and a ligand specific for a pMHC complex.
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