公开(公告)号：US20200095579A1

公开(公告)日：2020-03-26

申请号：US16487796

申请日：2018-02-14

Applicant: CRISPR THERAPEUTICS AG

Inventor： Ante Sven LUNDBERG ,  Samarth KULKARNI ,  Lawrence KLEIN ,  Hari Kumar PADMANABHAN

IPC: C12N15/113 ,  C12N15/86 ,  A61K35/34 ,  C12N15/90 ,  C12N9/22 ,  C12N5/077 ,  C12N5/074

Abstract: The present application provides materials and methods for treating a patient with one or more conditions or disorders associated with LAMA2, both ex vivo or in vivo. For example, the present disclosure provides materials and methods for treating a patient with Merosin-deficient Cogenital Muscular Dystrophy (MDCMD). The present application also provides materials and methods for editing a LAMA2 gene in a cell by genome editing. The present application also provides materials and methods for altering a contiguous genomic sequence of a LAMA2 gene in a cell. In addition, the present application provides one or more gRNAs for editing a LAMA2 gene. The present application also provides a therapeutic comprising at least one or more gRNAs for editing a LAMA2 gene. In addition, the present application provides a therapeutic for treating a patient with a LAMA2 related condition or disorder.

公开(公告)号：US20190211362A1

公开(公告)日：2019-07-11

申请号：US16312651

申请日：2017-06-27

Applicant: CRISPR THERAPEUTICS AG

Inventor： Ante Sven LUNDBERG ,  Samarth KULKARNI ,  Lawrence KLEIN ,  Hari Kumar PADMANABHAN

IPC: C12N15/90 ,  C12N15/10 ,  C12N9/22 ,  A61K35/34 ,  A61K38/46 ,  C12N15/11

CPC classification number: C12N15/907 ,  A61K35/34 ,  A61K38/465 ,  C12N9/22 ,  C12N15/102 ,  C12N15/11 ,  C12N15/113 ,  C12N2310/20 ,  C12N2750/14143 ,  C12N2800/80

Abstract: The present application provides materials and methods for treating a patient with one or more conditions associated with DMPK whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of DMPK gene in a cell by genome editing.

公开(公告)号：US20160298096A1

公开(公告)日：2016-10-13

申请号：US15037371

申请日：2014-11-17

Applicant: Crispr Therapeutics AG

Inventor： Emmanuelle Charpentier ,  Krzysztof Chylinski ,  Ines Fontara

IPC: C12N9/22 ,  C12N15/86 ,  C12N15/90

CPC classification number: C12N9/22 ,  C12N15/113 ,  C12N15/86 ,  C12N15/902 ,  C12N15/907 ,  C12N2310/20 ,  C12N2310/3519 ,  C12N2310/531 ,  C12Y301/00

Abstract: The invention relates to Type II CRIS-PR-Cas systems of Cas9 enzymes, guide RNAs and associated specific PAMs.

Abstract translation: 本发明涉及Cas9酶的II型CRIS-PR-Cas系统，引导RNA和相关的特异性PAM。

公开(公告)号：US12173290B2

公开(公告)日：2024-12-24

申请号：US16912999

申请日：2020-06-26

Applicant: CRISPR THERAPEUTICS AG ,  BAYER HEALTHCARE LLC

Inventor： Ryo Takeuchi ,  Abraham Scaria

IPC: C12N15/11 ,  C12N9/22 ,  C12N15/64 ,  C12N15/86

Abstract: The present application provides a CRISPR/Cas system comprising a nuclease segment that encodes a Cas9 nuclease or variant thereof, a guide RNA segment comprising a nucleotide sequence that encodes a gRNA or sgRNA, and a promoter segment comprising a nucleotide sequence that encodes a first promoter comprising one or more tetracycline operator sequence, wherein the gRNA segment is operably linked to the promoter segment. The present application also provides materials and methods for controlling transcriptional expression of guide RNAs and/or post-transcriptional expression of Cas nuclease.

公开(公告)号：US12146157B2

公开(公告)日：2024-11-19

申请号：US16677207

申请日：2019-11-07

Applicant: CRISPR THERAPEUTICS AG

Inventor： Jonathan Alexander Terrett ,  Jason Sagert

IPC: C12N5/0783 ,  A61K35/17 ,  A61K39/00 ,  A61P35/00 ,  C07K14/705 ,  C07K14/725 ,  C07K16/40 ,  C12N15/11 ,  C12N15/86

Abstract: Provided herein, in some embodiments, are methods and compositions (e.g., cell compositions) for the treatment of cancer, such as PTK7+ malignancies.

公开(公告)号：US20240352151A1

公开(公告)日：2024-10-24

申请号：US18594693

申请日：2024-03-04

Applicant: CRISPR Therapeutics AG

Inventor： Jason Sagert ,  Jui Dutta-Simmons ,  Jonathan Alexander Terrett

IPC: C07K16/40 ,  A61K35/17 ,  A61K38/00 ,  A61K39/00 ,  A61P35/00 ,  C07K7/08 ,  C07K14/47 ,  C07K14/705 ,  C07K14/725 ,  C12N5/0783

CPC classification number: 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

Abstract: 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.

公开(公告)号：US11857574B2

公开(公告)日：2024-01-02

申请号：US18054521

申请日：2022-11-10

Applicant: CRISPR Therapeutics AG

Inventor： Mary-Lee Dequeant ,  Demetrios Kalaitzidis ,  Mohammed Ghonime

IPC: A61K35/17 ,  C07K14/705 ,  C12N5/0783 ,  C12N9/22 ,  C12N15/01 ,  C07K14/725 ,  C07K16/28 ,  C12N5/16 ,  C12N15/113 ,  C12N15/86 ,  A61K38/00 ,  C12N15/11

CPC classification number: A61K35/17 ,  C07K14/7051 ,  C07K14/70521 ,  C07K14/70575 ,  C07K14/70578 ,  C07K14/70596 ,  C07K16/2803 ,  C07K16/2875 ,  C07K16/2878 ,  C12N5/0636 ,  C12N5/16 ,  C12N9/22 ,  C12N15/111 ,  C12N15/113 ,  C12N15/86 ,  A61K38/00 ,  C07K2317/622 ,  C12N2310/20 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2510/00

Abstract: 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.

公开(公告)号：US11795238B2

公开(公告)日：2023-10-24

申请号：US17392740

申请日：2021-08-03

Applicant: CRISPR Therapeutics AG

Inventor： Lalit Kumar ,  Mary-Lee Dequeant

IPC: C07K16/42 ,  C07K16/46 ,  G01N33/53 ,  G01N33/68

CPC classification number: C07K16/4266 ,  G01N33/6854 ,  C07K2317/76 ,  G01N2333/70575

Abstract: High affinity antibodies capable of binding to a single-chain variable fragment (scFv) of anti-CD70 antibody, for example, the scFv expressed on cell surface as a portion of a chimeric antigen receptor (CAR). Also provided herein are methods for producing such anti-scFv antibodies and methods of using the antibodies disclosed herein for detecting, for example, T cells expressing an anti-CD70 CAR that comprise the scFv as an extracellular domain.

公开(公告)号：US20230302053A1

公开(公告)日：2023-09-28

申请号：US18181135

申请日：2023-03-09

Applicant: CRISPR THERAPEUTICS AG

Inventor： Jonathan Alexander TERRETT ,  Demetrios KALAITZIDIS ,  Lawrence KLEIN

IPC: A61K35/17 ,  C12N15/62 ,  C12N15/63 ,  A61K39/00 ,  C07K14/705 ,  C07K14/725 ,  C07K14/74 ,  C12N15/10 ,  A61K48/00 ,  C12N5/078 ,  C12N9/22 ,  C12N15/86

CPC classification number: A61K35/17 ,  C12N15/62 ,  C12N15/63 ,  A61K39/001138 ,  C07K14/70578 ,  C07K14/70517 ,  C07K14/7051 ,  A61K39/001112 ,  C07K14/70539 ,  C12N15/102 ,  C07K14/70521 ,  A61K48/0066 ,  C12N5/0634 ,  C12N9/22 ,  C12N15/86 ,  A61K2039/5156 ,  C07K2319/33 ,  A61K2039/5158 ,  C07K2319/02 ,  C07K2319/03 ,  C12N15/907

Abstract: 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.

公开(公告)号：US20230193210A1

公开(公告)日：2023-06-22

申请号：US18177703

申请日：2023-03-02

Applicant: CRISPR Therapeutics AG

Inventor： Alireza Rezania ,  Valentin Sluch

IPC: C12N5/071 ,  C07K14/705 ,  C07K14/525 ,  C12N5/074 ,  A61K35/545 ,  C12N5/0735 ,  C12N15/90 ,  C07K14/74 ,  C12N9/22 ,  A61K35/39 ,  A61P1/18 ,  C07K14/475 ,  C12N15/85 ,  A61K38/00

CPC classification number: C12N5/0676 ,  C07K14/70532 ,  C07K14/525 ,  C12N5/0696 ,  A61K35/545 ,  C12N5/0606 ,  C12N15/907 ,  C07K14/70539 ,  C12N9/22 ,  A61K35/39 ,  A61P1/18 ,  C07K14/475 ,  C12N15/85 ,  C12N2510/00 ,  A61K38/00 ,  C12N2506/45 ,  C07K2319/02 ,  C07K2319/09 ,  C07K2319/035 ,  C12N2506/03 ,  C12N2506/02

Abstract: 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 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 genetic modification comprises an insertion of a polynucleotide encoding a tolerogenic factor and/or survival factor. The universal donor cells may further comprise at least one genetic modification within or near a gene that encodes a survival factor, wherein said genetic modification comprises an insertion of a polynucleotide encoding a second tolerogenic factor and/or a different survival factor.