公开(公告)号：US11866727B2

公开(公告)日：2024-01-09

申请号：US15759202

申请日：2016-11-07

Applicant: CRISPR THERAPEUTICS AG

Inventor： Chad Albert Cowan ,  Roman Lvovitch Bogorad ,  Ante Sven Lundberg ,  Kirsten Leah Beaudry

IPC: C12N9/22 ,  C12N15/90 ,  C12N9/16 ,  C12N15/11 ,  A61K38/00

CPC classification number: C12N15/907 ,  C12N9/16 ,  C12N9/22 ,  C12N15/11 ,  C12Y301/03009 ,  A61K38/00 ,  C12N2310/20 ,  C12N2800/80

Abstract: 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.

公开(公告)号：US11851653B2

公开(公告)日：2023-12-26

申请号：US15779836

申请日：2016-12-01

Applicant: CRISPR THERAPEUTICS AG

Inventor： Chad Albert Cowan ,  Roman Lvovitch Bogorad ,  Jeffrey Li ,  Ante Sven Lundberg ,  Matthias Johannes John ,  Jeffrey William Stebbins ,  Thao Thi Nguyen

IPC: C12N9/22 ,  C12N15/11 ,  C12N15/10 ,  A61K9/00 ,  A61K31/7088 ,  A61K48/00

CPC classification number: C12N15/111 ,  A61K9/0019 ,  A61K31/7088 ,  C12N9/22 ,  C12N15/102 ,  A61K48/00 ,  C12N2310/20 ,  C12N2320/30 ,  C12N2320/32 ,  C12N2800/80 ,  C12N15/102 ,  C12Q2521/301

Abstract: 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.

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：US20200330609A1

公开(公告)日：2020-10-22

申请号：US16094408

申请日：2017-04-18

Applicant: CRISPR Therapeutics AG

Inventor： Chad Albert Cowan ,  Ante Sven Lundberg ,  Tirtha Chakraborty ,  Michelle l-ching Lin ,  Bibhu Prasad Mishra ,  Elizabeth Jae-eun Paik ,  Andrew Kernytsky ,  Todd Douglass Borland

IPC: A61K48/00 ,  C12N9/22 ,  C12N15/10

Abstract: Materials and methods for treating a patient with a hemoglobinopathy, both ex vivo and in vivo, and materials and methods for deleting, modulating, or inactivating a transcriptional control sequence of a BCL11A gene in a cell by genome editing.

公开(公告)号：US20210260219A1

公开(公告)日：2021-08-26

申请号：US16065535

申请日：2016-12-22

Applicant: Crispr Therapeutics AG

Inventor： Chad Albert Cowan ,  Ami Meda Kabadi ,  Ante Sven Lundberg

IPC: A61K48/00 ,  A61K35/28 ,  A61K35/30 ,  A61K38/19 ,  A61K31/395 ,  A61K38/46 ,  A61K31/7105 ,  A61K35/761 ,  A61K41/00 ,  A61P25/28 ,  C12N15/90 ,  C12N5/0789 ,  C12N5/0797 ,  C12N5/0793 ,  C12N5/074 ,  C12N5/0775

Abstract: The present application provides materials and methods for treating a patient with Amyotrophic Lateral Sclerosis (ALS) and/or Frontaltemporal Lobular Degeneration (FTLD), both ex vivo and in vivo. In addition, the present application provides materials and methods for editing to modulate the expression, function or activity of the C9ORF72 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：US20190076551A1

公开(公告)日：2019-03-14

申请号：US16084531

申请日：2017-03-16

Applicant: CRISPR Therapeutics AG

Inventor： Roman Lvovitch Bogorad ,  Chad Albert Cowan ,  Ante Sven Lundberg

IPC: A61K48/00 ,  A61K9/00 ,  C12N5/0775 ,  C12N5/071

Abstract: Materials and methods for treating a patient with hereditary hemochromatosis (HHC), both ex vivo and in vivo, and materials and methods for modulating the expression, function, or activity of a haemochromatosis (HFE) 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.