公开(公告)号：US20220364082A1

公开(公告)日：2022-11-17

申请号：US17726068

申请日：2022-04-21

Applicant: Crispr Therapeutics AG

Inventor： Ante Sven Lundberg ,  Samarth Kulkarni ,  Lawrence Klein ,  Hari Kumar Padmanabhan

IPC: C12N15/11 ,  C12N15/113 ,  A61K9/00 ,  A61K31/7088 ,  A61K35/407 ,  A61K38/46 ,  A61K48/00 ,  C12N7/00 ,  C12N9/22

Abstract: The present application provides materials and methods for treating a patient with one or more conditions associated with ANGPTL4 whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of ANGPTL4 gene in a cell by genome editing.

公开(公告)号：US20200157565A1

公开(公告)日：2020-05-21

申请号：US16487300

申请日：2018-02-21

Applicant: CRISPR THERAPEUTICS AG

Inventor： Ante Sven Lundberg ,  Samarth Kulkarni ,  Lawrence Klein ,  Hari Kumar Padmanabhan ,  Yvonne Sarah Aratyn

IPC: C12N15/86 ,  C12N7/00 ,  C12N15/11 ,  C12N9/22

Abstract: The present application provides materials and methods for treating a patient with one or more conditions associated with ANGPTL3 whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of ANGPTL3 gene in a cell by genome editing.

公开(公告)号：US20190185849A1

公开(公告)日：2019-06-20

申请号：US16312676

申请日：2017-06-29

Applicant: CRISPR Therapeutics AG

Inventor： Ante Sven Lundberg ,  Samarth Kulkarni ,  Lawrence Klein ,  Hari Kumar Padmanabhan

IPC: C12N15/11 ,  A61K48/00 ,  A61K35/407 ,  A61K31/7088 ,  A61K38/46 ,  A61K9/00 ,  C12N7/00 ,  C12N9/22

CPC classification number: C12N15/11 ,  A61K9/0019 ,  A61K31/7088 ,  A61K35/407 ,  A61K38/465 ,  A61K48/0066 ,  C12N7/00 ,  C12N9/22 ,  C12N15/1136 ,  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 ANGPTL4 whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of ANGPTL4 gene in a cell by genome editing.

公开(公告)号：US20190160186A1

公开(公告)日：2019-05-30

申请号：US16312630

申请日：2017-06-22

Applicant: CRISPR Therapeutics AG

Inventor： Ante Sven Lundberg ,  Samarth Kulkarni ,  Lawrence Klein ,  Hari Kumar Padmanabhan

IPC: A61K48/00 ,  C12N15/90 ,  A61K35/30 ,  C12N5/079 ,  C12N5/0793 ,  C12N15/11 ,  C12N9/22 ,  C07K14/47 ,  A61P25/28

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

公开(公告)号：US11564997B2

公开(公告)日：2023-01-31

申请号：US16312630

申请日：2017-06-22

Applicant: CRISPR Therapeutics AG

Inventor： Ante Sven Lundberg ,  Samarth Kulkarni ,  Lawrence Klein ,  Hari Kumar Padmanabhan

IPC: A61K48/00 ,  C12N15/90 ,  C12N15/11 ,  C12N9/22 ,  C07K14/47 ,  C12N15/113 ,  A61P25/28 ,  A61K35/30 ,  C12N5/0793 ,  C12N5/079

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

公开(公告)号：US11559588B2

公开(公告)日：2023-01-24

申请号：US16487689

申请日：2018-02-20

Applicant: CRISPR THERAPEUTICS AG

Inventor： Ante Sven Lundberg ,  Samarth Kulkarni ,  Lawrence Klein ,  Hari Kumar Padmanabhan

IPC: A61K48/00 ,  A61K38/46 ,  C12N15/10

Abstract: The present disclosure provides materials and methods for treating a patient with one or more conditions or disorders associated with ATXN1 whether ex vivo or in vivo. For example, the present disclosure provides materials and methods for treating a patient with Spinocerebellar ataxia type 1 (SCA1). Also provided are materials and methods for editing a ATXN1 gene in a cell by genome editing. The present disclosure also provides materials and methods for altering the contiguous genomic sequence of a ATXN1 gene in a cell. In addition, the present disclosure provides one or more gRNAs for editing a ATXN1 gene. Also provided are therapeutics comprising at least one or more gRNAs for editing a ATXN1 gene. In addition, the present disclosure provides therapeutics for treating patients with a ATXN1 related condition or disorder.

公开(公告)号：US20200080082A1

公开(公告)日：2020-03-12

申请号：US16487282

申请日：2018-02-21

Applicant: CRISPR THERAPEUTICS AG

Inventor： Ante Sven Lundberg ,  Samarth Kulkarni ,  Lawrence Klein ,  Hari Kumar Padmanabhan

IPC: C12N15/113 ,  C12N9/22 ,  C12N15/90

Abstract: The present application provides materials and methods for treating a patient with one or more conditions or disorders associated with AGXT, both ex vivo or in vivo. For example, the present disclosure provides materials and methods for treating a patient with Primary Hyperoxaluria Type 1 (PH1). The present application also provides materials and methods for editing an AGXT gene in a cell by genome editing. The present application also provides materials and methods for altering a contiguous genomic sequence of an AGXT gene in a cell. In addition, the present application provides one or more gRNAs for editing an AGXT gene. The present application also provides a therapeutic comprising at least one or more gRNAs for editing an AGXT gene. In addition, the present application provides a therapeutic for treating a patient with an AGXT related condition or disorder.

公开(公告)号：US20190224340A1

公开(公告)日：2019-07-25

申请号：US16315540

申请日：2017-07-06

Applicant: CRISPR Therapeutics AG

Inventor： Ante Sven Lundberg ,  Samarth Kulkarni ,  Lawrence Klein ,  Hari Kumar Padmanabhan

IPC: A61K48/00 ,  C12N9/22 ,  A61K9/00 ,  C12N15/113 ,  C07K14/705 ,  C12N15/90 ,  A61K35/30 ,  C12N5/0775 ,  C12N5/0793 ,  A61K9/127 ,  A61K9/51 ,  A61P43/00

Abstract: The present application provides materials and methods for treating a patient with one or more conditions associated with SCN10A whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of SCN10A gene in a cell by genome editing.

公开(公告)号：US20240318200A1

公开(公告)日：2024-09-26

申请号：US18444368

申请日：2024-02-16

Applicant: CRISPR THERAPEUTICS AG

Inventor： Ante Sven Lundberg ,  Samarth Kulkarni ,  Lawrence Klein ,  Hari Kumar Padmanabhan ,  Yvonne Sarah Aratyn

IPC: C12N15/86 ,  C12N7/00 ,  C12N9/22 ,  C12N15/11

CPC classification number: C12N15/86 ,  C12N7/00 ,  C12N9/22 ,  C12N15/111 ,  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 ANGPTL3 whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of ANGPTL3 gene in a cell by genome editing.

公开(公告)号：US11920148B2

公开(公告)日：2024-03-05

申请号：US16487300

申请日：2018-02-21

Applicant: CRISPR THERAPEUTICS AG

Inventor： Ante Sven Lundberg ,  Samarth Kulkarni ,  Lawrence Klein ,  Hari Kumar Padmanabhan ,  Yvonne Sarah Aratyn

IPC: C12N15/86 ,  C12N7/00 ,  C12N9/22 ,  C12N15/11

CPC classification number: C12N15/86 ,  C12N7/00 ,  C12N9/22 ,  C12N15/111 ,  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 ANGPTL3 whether ex vivo or in vivo. In addition, the present application provides materials and methods for editing and/or modulating the expression of ANGPTL3 gene in a cell by genome editing.