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公开(公告)号：US20190022192A1
公开(公告)日：2019-01-24
申请号：US15574420
申请日：2016-04-15
申请人： Genzyme Corporation
发明人： Guoxiang RUAN , Abraham SCARIA
IPC分类号： A61K38/46 , C12N9/22 , C12N15/11 , C12N15/90 , A61K31/7105 , A61K35/761
摘要： Provided herein are compositions, methods, kits, and viral particles for treating a disease or disorder associated with a deep intronic mutation using an engineered, non-naturally occurring Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-CRISPR associated (Cas) (CRISPR-Cas) system. In some aspects, provided herein is a self-limiting CRISPER-Cas system.
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公开(公告)号：US20180180525A1
公开(公告)日：2018-06-28
申请号：US15544498
申请日：2016-01-19
申请人： Genzyme Corporation
CPC分类号： G01N15/042 , C12N7/00 , C12N2710/10351 , C12N2750/14151 , G01N1/405 , G01N1/4077 , G01N2001/4083 , G01N2015/045
摘要： Provided herein are methods to characterize preparations of recombinant viral particles using analytical ultracentrifugation. Recombinant viral particles include recombinant adeno-associated viral particles, recombinant adenoviral particles, recombinant lentiviral particles and recombinant herpes simplex virus particles. Variant species of recombinant viral particles including empty capsids and recombinant viral particles with variant genomes (e.g., truncated genomes, aggregates, recombinants) can be identified and quantitated. The methods can be used to characterize preparations of recombinant viral particles regardless of the sequence of the recombinant viral genome or the serotype of the recombinant viral capsid.
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公开(公告)号：US20190111157A1
公开(公告)日：2019-04-18
申请号：US15549962
申请日：2016-02-09
申请人： Genzyme Corporation
发明人： Lisa M. STANEK , Lamya SHIHABUDDIN
摘要： Provided herein are novel methods for delivering recombinant adeno-associated viral (rAAV) particles to the central nervous system of a mammal (e.g., a human). In aspects, the methods involve administering rAAV particles containing a heterologous nucleic acid to the striatum and causing expression of the heterologous nucleic acid in at least the cerebral cortex and the striatum of the mammal.
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4.发明公开公开(公告)号：US20240343790A1
公开(公告)日：2024-10-17
申请号：US18627131
申请日：2024-04-04
申请人： Genzyme Corporation
发明人： Huawei Qiu , Clark Pan , Julie Bird
CPC分类号： C07K16/22 , A61K39/395 , A61K39/3955 , C07K16/00 , C07K16/46 , C12N15/11 , C12N15/62 , A61K2039/505 , C07K2317/21 , C07K2317/35 , C07K2317/52 , C07K2317/53 , C07K2317/56 , C07K2317/565 , C07K2317/622 , C07K2317/626 , C07K2317/64 , C07K2317/70 , C07K2317/72 , C07K2317/76 , C07K2317/92 , C07K2317/94 , C07K2319/00 , C07K2319/23 , C07K2319/73
摘要： An scFv-Fc dimer binds and neutralizes TGFβ1 selectively and with high affinity and avidity. The scFv region may comprise the same VH and VL domains or CDR regions as metelimumab. The unique combination of their smaller size, high selectivity, potency against TGFβ1, and long in vivo half-life makes the scFv-Fc dimers ideal candidates for therapeutic applications.
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公开(公告)号：US20240288454A1
公开(公告)日：2024-08-29
申请号：US18428535
申请日：2024-01-31
申请人： GENZYME CORPORATION
发明人： Ryan Grabert , Susan Richards , Valerie Theobald , Yuanxin Xu , Jad Zoghbi
IPC分类号： G01N33/94 , G01N33/53 , G01N33/539 , G01N33/68
CPC分类号： G01N33/94 , G01N33/5306 , G01N33/539 , G01N33/6854 , G01N2430/00
摘要： Methods and kits for detecting antibodies (e.g., anti-drug antibodies). Such methods and kits permit the detection of, for example, anti-drug antibodies in human body fluids, such as blood, plasma and scrum.
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6.发明公开公开(公告)号：US20240254556A1
公开(公告)日：2024-08-01
申请号：US18560569
申请日：2022-05-13
申请人： GENZYME CORPORATION
发明人： Darren Phillip BAKER , Emanuele DE RINALDIS , Richa HANAMSAGAR , Evis HAVARI , Virginia SAVOVA , Srinivas SHANKARA
IPC分类号： C12Q1/6883 , C12Q1/6874 , G01N33/564
CPC分类号： C12Q1/6883 , C12Q1/6874 , G01N33/564 , C12Q2600/158 , G01N2800/046 , G01N2800/285 , G01N2800/50 , G01N2800/52
摘要： The invention provides methods of assessing the risk of secondary autoimmunity in a patient with a primary autoimmune disease (e.g., MS) following lymphocyte depleting therapy (e.g., anti-CD52 antibody therapy) based on the fraction of a novel cell type termed platelet lineage cells (PLCs) among total cells, and/or the Immature Platelet Fraction (IPF) value, in a biological sample from the patient.
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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
申请人： Genzyme Corporation
发明人： Yunxiang Zhu , Jozsef Karman , Ronnie Wei , Canwen Jiang , Seng Cheng
IPC分类号： C07K16/28 , A61K38/17 , A61K39/395 , A61P37/06 , C07K14/47 , C07K14/705 , C07K14/74
CPC分类号： C07K16/2803 , C07K14/47 , C07K14/70532 , C07K14/70539 , C07K2319/00
摘要： 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：US20240218338A1
公开(公告)日：2024-07-04
申请号：US18409535
申请日：2024-01-10
申请人： Genzyme Corporation
CPC分类号： C12N9/16 , A61P19/08 , C12Y301/04
摘要： The invention provides methods of using a human acid sphingomyelinase (e.g., olipudase alfa) in treating an abnormal bone condition in acid sphingomyelinase deficiency patients such as low bone density, high bone marrow burden, and other skeletal abnormalities presented in acid sphingomyelinase deficiency patients.
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公开(公告)号：US11999780B2
公开(公告)日：2024-06-04
申请号：US17557318
申请日：2021-12-21
申请人： Genzyme Corporation
发明人： Graham Parry , Stephen Moore , Michael Storek , Nina C. Leksa
CPC分类号： C07K16/18 , A61K47/6843 , C07K2317/24 , C07K2317/31 , C07K2317/52 , C07K2317/92
摘要： Provided herein are humanized anti-factor Bb antibodies, methods of producing the antibodies and methods of using the antibodies.
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公开(公告)号：US20240158761A1
公开(公告)日：2024-05-16
申请号：US18511501
申请日：2023-11-16
申请人： Genzyme Corporation
发明人： Bruno Figueroa , Qingxuan Li , Junfen Ma , Yuanli Song
IPC分类号： C12N7/00
CPC分类号： C12N7/00 , C12N2750/14151
摘要： The disclosure provides methods of purifying adeno-associated virus (AAV) particles by introducing a flocculant step into a large scale a manufacturing process, thereby improving AAV particle purity and yield.
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