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1.发明申请公开(公告)号：US20190030038A1
公开(公告)日：2019-01-31
申请号：US16147153
申请日：2018-09-28
IPC分类号： A61K31/522 , A61K31/00
摘要： The present invention relates generally to the field of using udenafil or a pharmaceutically acceptable salt thereof in patients who have undergone the Fontan operation.
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公开(公告)号：US12123020B2
公开(公告)日：2024-10-22
申请号：US18330167
申请日：2023-06-06
发明人： Stella Chou , Connie Westhoff
IPC分类号： A61K35/14 , A61K35/17 , A61K39/00 , A61K39/12 , A61K39/145 , C12N5/078 , C12N9/22 , C12N15/11 , G01N33/68 , G01N33/80
CPC分类号： C12N5/0634 , A61K35/14 , C12N9/22 , C12N15/111 , G01N33/6854 , C12N2310/20 , C12N2510/00 , G01N33/80
摘要： Provided herein are engineered red blood cells expressing rare blood antigen group profiles, and methods of making use the same, are described. Also provided are recombinant reagent red blood cells that express or lack the expression of at least one protein (e.g., a blood group antigen) on its surface and uses thereof.
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公开(公告)号：US12082845B2
公开(公告)日：2024-09-10
申请号：US18162036
申请日：2023-01-31
申请人： The Trustees of the University of Pennsylvania , The Children's Hospital of Philadelphia , Onocor LLC
CPC分类号： A61B17/50 , A61B17/22031 , A61B17/221 , A61F2/962 , A61B2017/00367 , A61B2017/00778 , A61B2017/22034 , A61B2017/22035 , A61B2017/2215 , A61F2/2427 , A61F2002/9528 , A61F2002/9623
摘要： Disclosed are systems, methods, and devices for percutaneous retrieval of objects, such as endovascular devices, from an internal body space. The present inventions have vascular, non-vascular (gastrointestinal), and surgical (laproscopic) applications. The inventions include a a wire woven distal end that is formed from wires and includes a substantially cylindrical main body section comprising a leading end, a trailing end, and a middle portion extending between the leading end and the trailing end and defining a longitudinal axis of the main body section, and a flare defining a mouth of the wire woven distal end that projects circumferentially from the leading end of the main body section, wherein when at least a portion of the retrieval end is transitioned to a compressed state following capture of an object, the retrieval end exerts an inward force that at least partially collapses or compresses the object.
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公开(公告)号：US20240197920A1
公开(公告)日：2024-06-20
申请号：US18557033
申请日：2022-04-27
发明人： Beverly DAVIDSON , Paul RANUM
CPC分类号： A61K48/0058 , C12N15/86 , C12N2750/14122 , C12N2750/14143 , C12N2750/14145 , C12N2830/008
摘要： Provided herein are compositions and methods for delivering a molecular therapeutic to the cochlea of a subject. The methods comprise administering an adeno-associated virus (AAV) to the cerebrospinal fluid of the subject. The AAVs encode a therapeutic transgene for molecular therapy. Optionally, the therapeutic transgene may be operably linked to a cochlea-specific promoter.
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公开(公告)号：US12011452B2
公开(公告)日：2024-06-18
申请号：US17256406
申请日：2019-06-27
发明人： Marni J. Falk , Eiko Nakamaru-Ogiso
IPC分类号： A61K31/7004 , A61K31/10 , A61K31/145 , A61K31/198 , A61K31/455 , G01N33/50
CPC分类号： A61K31/7004 , A61K31/10 , A61K31/145 , A61K31/198 , A61K31/455 , G01N33/5044 , G01N2333/43534
摘要： Compositions and methods for treatment of mitochondrial respiratory chain dysfunction and other mitochondrial disorders are provided. Also disclosed are a number of screening assays having utility for the identification of agents which modulate the phenotype associated with mitochondrial respiratory chain dysfunction.
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6.发明公开公开(公告)号：US20240131126A1
公开(公告)日：2024-04-25
申请号：US18389662
申请日：2023-12-19
发明人： Katherine A. HIGH , Xavier ANGUELA
CPC分类号： A61K38/4846 , C12N9/644 , C12N15/86 , C12Y304/21022 , C12N2750/14143
摘要： The invention relates to modified Factor IX coding sequence, expression cassette, vectors such as viral (e.g., lenti- or adeno-associated viral) vectors, and gene transfer methods and uses. In particular, to target Factor IX nucleic acid to cells, tissues or organs for expression (transcription) of Factor IX.
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公开(公告)号：US20240075164A1
公开(公告)日：2024-03-07
申请号：US17052170
申请日：2019-04-30
CPC分类号： A61K48/005 , A61P3/00 , C12N9/0069 , C12N9/22 , C12N15/113 , C12N15/86 , C12N2310/11 , C12N2310/20 , C12N2710/10343 , C12N2750/14143 , C12Y113/11027
摘要： A method for in utero genome editing, the method comprising administering to a subject an adenoviral vector, wherein the subject is a fetus, the adenoviral vector comprising CRISPR-mediated base editor and a guide RNA (gRNA), the gRNA targeting a mutation in a therapeutic gene; and introducing a modified codon in the therapeutic gene by base editing the therapeutic gene, wherein the base editing is performed by the adenoviral vector, an adeno-associated viral vector or lipid based nanoparticle.
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公开(公告)号：US11878056B2
公开(公告)日：2024-01-23
申请号：US17016993
申请日：2020-09-10
发明人： John Fraser Wright , Guang Qu , Bernd Hauck , Katherine A. High
IPC分类号： A61K39/23 , C12N15/864 , A61K48/00 , C12N7/00 , C12N15/00 , C07K2/00 , C12N15/64 , A61K38/46 , C12N15/86
CPC分类号： A61K39/23 , A61K38/465 , A61K48/0091 , C07K2/00 , C12N7/00 , C12N15/64 , C12N15/86 , C12N15/864 , A61K48/00 , C12N15/8645 , C12N2750/14011 , C12N2750/14051 , C12N2750/14111 , C12N2750/14143 , C12N2750/14151 , C12Y301/01064
摘要： Methods for preparing highly purified AAV vector formulations are provided. The highly pure AAV formulations described herein are superior for clinical use.
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公开(公告)号：US11807865B2
公开(公告)日：2023-11-07
申请号：US16113908
申请日：2018-08-27
发明人： Katherine A. High , John Fraser Wright , Bernd Hauck , Guang Qu
IPC分类号： C12N15/867 , C12N7/00 , C12N7/01 , C12N7/02 , C12N15/113 , C12N15/86 , A61K48/00
CPC分类号： C12N15/86 , C12N7/00 , A61K48/00 , C12N2740/15041 , C12N2740/15051 , C12N2740/15052 , C12N2740/16041 , C12N2740/16051 , C12N2740/16052 , C12N2800/50
摘要： In accordance with the present invention, a method for increasing the yield of rLV vector particles comprising a trans gene encoding a therapeutic protein or fragment thereof is disclosed. In one approach, cells are transfected with plasmids encoding the necessary components for rLV production using a calcium chloride transfection mix at pH 7.1 wherein the calcium chloride and plasmids form a complex which is added to the cells at a constant speed. The cells are then incubated for a suitable time period wherein virus particle media is collected at least twice during the incubation period and stored in a cold storage unit, thereby reducing virus inactivation.
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10.发明公开公开(公告)号：US20230340498A1
公开(公告)日：2023-10-26
申请号：US17798851
申请日：2021-02-12
IPC分类号： C12N15/62 , A61K48/00 , C12N15/86 , C07K14/015
CPC分类号： C12N15/625 , A61K48/005 , C07K14/015 , C12N15/86 , C07K2319/02 , C12N2320/33 , C12N2750/14143
摘要： Provided herein are chimeric minigenes, where the alternative splicing of the minigene determines whether an encoded gene is expressed. In particular, the minigenes are alternatively spliced in response to splicing modulator dmgs, such that the encoded gene is only expressed in the present of the splicing modulator dmg. The encoded gene may encode an inhibitory RNA, a CRISPR-Cas9 protein, a transactivator, or a therapeutic protein.
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