公开(公告)号：US20240301380A1

公开(公告)日：2024-09-12

申请号：US18261285

申请日：2022-01-13

申请人： Spark Therapeutics, Inc.

发明人： Sean Armour ,  Daniel Cohen ,  Christopher Riling

IPC分类号： C12N9/40 ,  A61K48/00 ,  A61P3/00 ,  C12N15/86

CPC分类号： C12N9/2465 ,  A61K48/0058 ,  A61P3/00 ,  C12N15/86 ,  C07K2319/02 ,  C12N2750/14143 ,  C12N2830/008 ,  C12N2830/15 ,  C12N2830/42 ,  C12N2830/50 ,  C12Y302/01022

摘要： Polynucleotides including nucleic acids encoding α-galactosidase A (GLA) are described. Also described are expression cassettes, vectors, cells, and cell lines containing the polynucleotides, as well as methods of using the polynucleotides to treat lysosomal storage disorders such as Fabry disease.

公开(公告)号：US20240287543A1

公开(公告)日：2024-08-29

申请号：US18355575

申请日：2023-07-20

申请人： MEMORIAL SLOAN-KETTERING CANCER CENTER

发明人： Michel Sadelain ,  Annalisa Cabriolu

IPC分类号： C12N15/86 ,  A61P7/00 ,  C12N9/22

CPC分类号： C12N15/86 ,  A61P7/00 ,  C12N9/22 ,  C12N2310/20 ,  C12N2830/008 ,  C12N2830/15 ,  C12N2830/30

摘要： The presently disclosed subject matter provides for expression cassettes that allow for expression of a globin gene or a functional portion thereof, vectors comprising thereof, and cells transduced with such expression cassettes and vectors. The presently disclosed subject matter further provides methods for treating a hemoglobinopathy in a subject comprising administering an effective amount of such transduced cells to the subject.

公开(公告)号：US12029784B2

公开(公告)日：2024-07-09

申请号：US17079006

申请日：2020-10-23

申请人： 2seventy bio, Inc.

发明人： Richard Morgan ,  Kevin Friedman

IPC分类号： A61K39/00 ,  C07K14/705 ,  C07K14/725 ,  C07K16/28 ,  C12N5/0783 ,  C12N15/86

CPC分类号： A61K39/0011 ,  C07K14/7051 ,  C07K14/70517 ,  C07K14/70578 ,  C07K16/2878 ,  C12N5/0636 ,  C12N15/86 ,  A61K2039/5156 ,  A61K2039/5158 ,  A61K2039/585 ,  C07K2317/622 ,  C07K2319/32 ,  C12N2740/15043 ,  C12N2830/15

摘要： The invention provides improved compositions for adoptive T cell therapies for B cell related conditions.

公开(公告)号：US20240175006A1

公开(公告)日：2024-05-30

申请号：US18285370

申请日：2022-03-31

申请人： HUNTERIAN MEDICINE LLC

发明人： Vinod Jaskula-Ranga

IPC分类号： C12N15/10 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86

CPC分类号： C12N15/1024 ,  C12N9/22 ,  C12N15/111 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2800/22 ,  C12N2830/15 ,  C12N2830/42

摘要： The invention relates generally to compact promoters and their use in gene editing e.g., for treating disease. The disclosure is based, in part, upon the discovery of compact, bidirectional promoters that can be used to express both a nuclease (e.g., a Cas9 nuclease) and a guide RNA (gRNA). For example, in certain embodiments disclosed herein, a compact, bidirectional promoter can comprise at least one regulatory element that directs expression of a gRNA in one direction and at least one regulatory element that directs expression of a nuclease in the other direction. Accordingly, the promoters disclosed herein use less space than prior art promoters, allowing both a nuclease and a gRNA to be packaged in a single vector (e.g., a plasmid or an AAV).

公开(公告)号：US20240173436A1

公开(公告)日：2024-05-30

申请号：US18285369

申请日：2022-03-31

申请人： HUNTERIAN MEDICINE LLC

发明人： Vinod Jaskula-Ranga

IPC分类号： A61K48/00 ,  A61P1/00 ,  A61P11/00 ,  C12N15/86

CPC分类号： A61K48/0066 ,  A61P1/00 ,  A61P11/00 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2800/22 ,  C12N2830/15 ,  C12N2830/42

摘要： The invention relates generally to compact promoters and their use in expressing genes, e.g., for treating disease.

公开(公告)号：US11975078B2

公开(公告)日：2024-05-07

申请号：US16924939

申请日：2020-07-09

申请人： VRIJE UNIVERSITEIT BRUSSEL ,  VIB VZW ,  UNIVERSITEIT GENT

发明人： Thierry Vandendriessche ,  Marinee Chuah ,  Pieter De Bleser

IPC分类号： A61K48/00 ,  C12N9/26 ,  C12N15/85 ,  C12N15/86 ,  C12N15/864

CPC分类号： A61K48/0058 ,  A61K48/0075 ,  C12N9/2408 ,  C12N15/85 ,  C12N15/86 ,  C12N15/8645 ,  C12N2710/10043 ,  C12N2750/14143 ,  C12N2800/22 ,  C12N2830/008 ,  C12N2830/15 ,  C12N2830/85

摘要： The present invention relates to nucleic acid regulatory elements that are able to enhance muscle-specific expression of genes, in particular expression in cardiac muscle and/or skeletal muscle, methods employing these regulatory elements and uses of these elements. Expression cassettes and vectors containing these nucleic acid regulatory elements are also disclosed. The present invention is particularly useful for applications using gene therapy, more particularly muscle-directed gene therapy, and for vaccination purposes.

公开(公告)号：US20240076693A1

公开(公告)日：2024-03-07

申请号：US18354444

申请日：2023-07-18

申请人： Aavocyte, Inc.

发明人： Aiquan Chang ,  Chuanxin Liu ,  Yong Liu

IPC分类号： C12N15/86 ,  A61K35/17 ,  C12N5/0783 ,  C12N5/0784 ,  C12N15/864

CPC分类号： C12N15/86 ,  A61K35/17 ,  C12N5/0638 ,  C12N5/0639 ,  C12N15/8645 ,  C12N2501/22 ,  C12N2501/2302 ,  C12N2501/2304 ,  C12N2501/2307 ,  C12N2501/25 ,  C12N2750/14134 ,  C12N2750/14143 ,  C12N2800/107 ,  C12N2830/00 ,  C12N2830/15 ,  C12N2830/50

摘要： The present disclosure provides rAAV vectors and rAAV virions that specifically express exogenous nucleic acid sequences in CD14+ cells. The rAAV vectors or virions are useful for specifically expressing exogenous nucleic acid sequences encoding, for example, cancer antigens, viral antigens, and/or bacterial antigens in monocytes and dendritic cells. The rAAV transduced CD14+ cells can be used as antigen presenting cells that induce antigen-specific T cell responses. The present disclosure further provides methods producing rAAV virions and methods of immunotherapy.

公开(公告)号：US20240016132A1

公开(公告)日：2024-01-18

申请号：US18353012

申请日：2023-07-14

申请人： Oxitec Limited

发明人： Martha Koukidou ,  Luke Alphey ,  Simon Warner

IPC分类号： A01K67/033 ,  C12N15/85 ,  C12Q1/6888

CPC分类号： A01K67/0339 ,  C12N15/8509 ,  C12Q1/6888 ,  A01K2217/072 ,  A01K2217/15 ,  A01K2217/203 ,  A01K2217/206 ,  A01K2217/30 ,  A01K2227/706 ,  A01K2267/02 ,  C12N2800/90 ,  C12N2830/006 ,  C12N2830/008 ,  C12N2830/15 ,  C12N2840/007 ,  C12N2840/44 ,  C12N2840/75 ,  C12N2999/007 ,  C12Q2600/158

摘要： Two or more conditional, dominant, lethal gene expression systems provide high levels of penetrance in insects. Lethality is induced at an earlier stage of development and the risk of biochemical resistance is reduced, as compared to a single insect conditional, dominant, lethal gene expression system. The invention is useful for the control of insect populations.

公开(公告)号：US20230257777A1

公开(公告)日：2023-08-17

申请号：US18307256

申请日：2023-04-26

申请人： Adverum Biotechnologies, Inc.

发明人： Annahita Keravala

IPC分类号： C12N15/86 ,  A61K48/00 ,  C07K14/71

CPC分类号： C12N15/86 ,  A61K48/0066 ,  C07K14/71 ,  C12N2750/14143 ,  C12N2800/22 ,  C12N2830/15 ,  C12N2830/42 ,  C12N2830/50

摘要： The present disclosure provides polynucleotide cassettes, expression vectors and methods for the expression of a gene in mammalian cells.

公开(公告)号：US11717579B2

公开(公告)日：2023-08-08

申请号：US15449416

申请日：2017-03-03

申请人： MEMORIAL SLOAN-KETTERING CANCER CENTER ,  UNIVERSITY OF WASHINGTON

发明人： Michel Sadelain ,  Isabelle Riviere ,  Jorge Mansilla-Soto ,  Xiuyan Wang ,  George Stamatoyannopoulos ,  John Stamatoyannopoulos ,  Mingdong Liu

IPC分类号： C12N15/86 ,  C12N15/63 ,  A61K48/00 ,  C12N15/11 ,  C07K14/805 ,  C12N9/22 ,  C12N15/85

CPC分类号： A61K48/0066 ,  A61K48/0058 ,  C07K14/805 ,  C12N9/22 ,  C12N15/85 ,  C12N15/86 ,  C12N2740/10043 ,  C12N2740/15043 ,  C12N2740/16043 ,  C12N2830/008 ,  C12N2830/15 ,  C12N2830/30 ,  C12N2830/40 ,  C12N2830/46 ,  C12N2830/48 ,  C12N2830/50

摘要： The presently disclosed subject matter provides for expression cassettes that allow for expression of a globin gene or a functional portion thereof, vectors comprising thereof, and cells transduced with such expression cassettes and vectors. The presently disclosed subject matter further provides methods for treating a hemoglobinopathy in a subject comprising administering an effective amount of such transduced cells to the subject.