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公开(公告)号：US20240115703A1
公开(公告)日：2024-04-11
申请号：US18482752
申请日：2023-10-06
发明人： Ziliang LI , Annie Yang WEAVER , Sarah COHEN , Ewelina MORAWA
CPC分类号： A61K39/4611 , A61K39/4631 , A61K39/464412 , A61P35/00 , A61P35/02 , A61K2239/38 , A61K2239/39 , A61K2239/48
摘要： Methods for treating B-cell malignancies such as relapsed and/or refractory B-cell malignancies with a population of genetically engineered T cells expressing a chimeric antigen receptor (CAR) targeting CD19 and having multiple genetic edits, including a disrupted TRAC gene, a disrupted β2M gene, a disrupted Regnase 1 gene, and/or a disrupted TGFBRII gene.
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公开(公告)号：US11926676B2
公开(公告)日：2024-03-12
申请号：US17313121
申请日：2021-05-06
IPC分类号： C07K16/40 , A61K35/17 , A61K38/00 , A61K39/00 , A61P35/00 , C07K7/08 , C07K14/47 , C07K14/705 , C07K14/725 , C12N5/0783
CPC分类号： C07K16/40 , A61K35/17 , A61P35/00 , C07K7/08 , C07K14/47 , C07K14/7051 , C07K14/70517 , C07K14/70521 , C07K14/70578 , C12N5/0636 , A61K38/00 , A61K2039/505 , A61K2039/5156 , A61K2039/5158 , C07K2317/622 , C07K2317/72 , C07K2317/73 , C07K2317/76 , C07K2317/92 , C07K2319/02 , C07K2319/03 , C07K2319/30 , C07K2319/33 , C07K2319/50 , C12N2510/00
摘要： Masked chimeric antigen receptor (CAR) constructs comprising an extracellular antigen binding domain specific tyrosine-protein kinase-like 7 (PTK7), which is linked to a mask peptide that blocks binding of masked CAR from binding to PTK7. Also provided herein are genetically engineered T cells expressing a masked CAR specific to PTK7 and therapeutic uses thereof.
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公开(公告)号：US11866727B2
公开(公告)日：2024-01-09
申请号：US15759202
申请日：2016-11-07
CPC分类号： C12N15/907 , C12N9/16 , C12N9/22 , C12N15/11 , C12Y301/03009 , A61K38/00 , C12N2310/20 , C12N2800/80
摘要： The present application provides materials and methods for treating a patient with Glycogen Storage Disease type 1a (GSD1a) both ex vivo and in vivo. In addition, the present application provides materials and methods for modulating the expression, function, and/or activity of the glucose-6-phosphatase, catalytic (G6PC) and/or the glucose-6-phosphatase (G6Pase) protein in a cell by genome editing.
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公开(公告)号：US11851653B2
公开(公告)日：2023-12-26
申请号：US15779836
申请日：2016-12-01
发明人： Chad Albert Cowan , Roman Lvovitch Bogorad , Jeffrey Li , Ante Sven Lundberg , Matthias Johannes John , Jeffrey William Stebbins , Thao Thi Nguyen
CPC分类号： C12N15/111 , A61K9/0019 , A61K31/7088 , C12N9/22 , C12N15/102 , A61K48/00 , C12N2310/20 , C12N2320/30 , C12N2320/32 , C12N2800/80 , C12N15/102 , C12Q2521/301
摘要： The present application provides materials and methods for treating a patient with Alpha-1 antitrypsin deficiency (AATD) both ex vivo and in vivo. In addition, the present application provides materials and methods for editing the SERPINA1 gene in a cell by genome editing.
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公开(公告)号：US20230392134A1
公开(公告)日：2023-12-07
申请号：US18246416
申请日：2021-09-29
CPC分类号： C12N9/22 , C12N15/86 , C12N2310/20 , C12N2750/14143
摘要： The present application provides materials and methods for treating a patient with Amyotrophic Lateral Sclerosis (ALS). In addition, the present application provides materials and methods for (1) modifying the transcription start site of exon1a to render the transcription start site non-functioning, (2) deleting the transcription site of exon1a, (3) deleting exon1a, or (4) deleting of the expanded hexanucleotide repeat within or near the C9ORF72 gene, or any combinations of (1)-(4), above in a cell by genome editing.
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6.发明公开公开(公告)号：US20230263828A1
公开(公告)日：2023-08-24
申请号：US18054521
申请日：2022-11-10
IPC分类号： A61K35/17 , C07K14/705 , C12N5/0783 , C12N9/22 , C12N15/11 , C07K14/725 , C07K16/28 , C12N5/16 , C12N15/113 , C12N15/86
CPC分类号： A61K35/17 , C07K14/7051 , C07K14/70521 , C07K14/70575 , C07K14/70578 , C07K14/70596 , C07K16/2803 , C07K16/2875 , C07K16/2878 , C12N5/16 , C12N5/0636 , C12N9/22 , C12N15/86 , C12N15/111 , C12N15/113 , A61K38/00 , C07K2317/622 , C12N2310/20 , C12N2310/315 , C12N2310/321 , C12N2510/00
摘要： A population of genetically engineered T cells, comprising a disrupted Reg1 gene and/or a disrupted TGFBRII gene. Such genetically engineered T cells may comprise further genetic modifications, for example, a disrupted CD70 gene. The population of genetically engineered T cells exhibit one or more of (a) improved cell growth activity; (b) enhanced persistence; and (c) reduced T cell exhaustion, (d) enhanced cytotoxicity activity, (e) resistant to inhibitory effects induced by TGF-b, and (f) resistant to inhibitory effects by fibroblasts and/or inhibitory factors secreted thereby, as compared to non-engineered T cell counterparts.
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公开(公告)号：US20230070540A1
公开(公告)日：2023-03-09
申请号：US17818669
申请日：2022-08-09
摘要： Genetically modified cells that are compatible with multiple subjects, e.g., universal donor cells, and methods of generating said genetic modified cells are provided herein. The universal donor cells comprise at least one genetic modification within or near at least one gene that encodes a survival factor, wherein the genetic modification comprises an insertion of a polynucleotide encoding a tolerogenic factor. The universal donor cells may further comprise at least one genetic modification within or near a gene that encodes one or more MHC-I or MHC-II human leukocyte antigens or a component or a transcriptional regulator of a MHC-I or MHC-II complex, wherein said genetic modification comprises an insertion of a polynucleotide encoding a second tolerogenic factor.
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公开(公告)号：US11591381B2
公开(公告)日：2023-02-28
申请号：US17538699
申请日：2021-11-30
发明人： Valentin Sluch , Alireza Rezania , Jason Sagert , Danielle Swain
IPC分类号： A61K35/17 , C07K14/74 , C07K14/54 , C07K14/725 , C07K14/715 , C07K14/81 , C07K16/28 , C12N5/0783 , C12N15/90 , C12N5/0735 , C12N5/074 , C12N5/10 , C12N15/66
摘要： The present invention relates to, inter alia, an engineered cell (e.g., iPSC, IPS-derived NK, or NK cell) comprising a disrupted B2M gene and an inserted polynucleotide encoding one or more of SERPINB9, a fusion of IL15 and IL15Rα, and/or HLA-E. The engineered cell can further comprise a disrupted CIITA gene and an inserted polynucleotide encoding a CAR, wherein the CAR can be an anti-BCMA CAR or an anti-CD30 CAR. The engineered cell may further comprise a disrupted ADAM17 gene, a disrupted FAS gene, a disrupted CISH gene, and/or a disrupted REGNASE-1 gene. Methods for producing the engineered cells are also provided, and therapeutic uses of the engineered cells are also described. Guide RNA sequences targeting described target sequences are also described.
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公开(公告)号：US20230059192A1
公开(公告)日：2023-02-23
申请号：US17821018
申请日：2022-08-19
发明人： Luke J. Turechek
IPC分类号： C12Q1/6855 , C12N15/10 , C12N15/113
摘要： The present disclosure provides methods for determining oligonucleotide purity and/or characterizing small RNAs. The methods comprising ligating adapters comprising unique molecule identifiers (UMIs), amplifying ligation products to generate a library, and sequencing the library. The methods of the disclosure exhibit reduced or no bias in terms of discrepancies that can arise during the ligation and/or amplification steps of the methods.
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公开(公告)号：US20230044761A1
公开(公告)日：2023-02-09
申请号：US17491757
申请日：2021-10-01
IPC分类号： A61K35/17 , C12N15/63 , A61K39/00 , C07K14/705 , C07K14/725 , C07K14/74 , C12N15/10 , C12N15/62
摘要： Materials and methods for producing genome-edited cells engineered to express a chimeric antigen receptor (CAR) construct on the cell surface, and materials and methods for genome editing to modulate the expression, function, or activity of one or more immuno-oncology related genes in a cell, and materials and methods for treating a patient using the genome-edited engineered cells.
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