公开(公告)号：US20250043261A1

公开(公告)日：2025-02-06

申请号：US18770568

申请日：2024-07-11

Applicant: CRISPR Therapeutics AG

Inventor： Yi-Shan Chen ,  Sandeep Soni ,  Laura Serwer ,  Jonathan Terrett ,  John Kulman

IPC: C12N9/22 ,  A61P3/00 ,  C12N15/11

Abstract: The present disclosure relates to methods, compositions and kits for treating conditions that are related with angiopoietin-like 3 (ANGPTL3) by gene editing.

公开(公告)号：US20250011743A1

公开(公告)日：2025-01-09

申请号：US18770561

申请日：2024-07-11

Applicant: CRISPR Therapeutics AG

Inventor： Yi-Shan Chen ,  Sandeep Soni ,  Laura Serwer ,  Jonathan Terrett ,  John Kulman

IPC: C12N9/22 ,  A61P3/00 ,  C12N15/11

Abstract: The present disclosure relates to methods, compositions and kits for treating conditions that are related with angiopoietin-like 3 (ANGPTL3) by gene editing.

公开(公告)号：US20240115703A1

公开(公告)日：2024-04-11

申请号：US18482752

申请日：2023-10-06

Applicant: CRISPR Therapeutics AG

Inventor： Ziliang LI ,  Annie Yang WEAVER ,  Sarah COHEN ,  Ewelina MORAWA

IPC: A61K39/00 ,  A61P35/00 ,  A61P35/02

CPC classification number: A61K39/4611 ,  A61K39/4631 ,  A61K39/464412 ,  A61P35/00 ,  A61P35/02 ,  A61K2239/38 ,  A61K2239/39 ,  A61K2239/48

Abstract: 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.

公开(公告)号：US11926676B2

公开(公告)日：2024-03-12

申请号：US17313121

申请日：2021-05-06

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：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：US20230392134A1

公开(公告)日：2023-12-07

申请号：US18246416

申请日：2021-09-29

Applicant: CRISPR THERAPEUTICS AG

Inventor： Hari Kumar Padmanabhan ,  Adam James Donoghue

IPC: C12N9/22 ,  C12N15/86

CPC classification number: C12N9/22 ,  C12N15/86 ,  C12N2310/20 ,  C12N2750/14143

Abstract: 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.

公开(公告)号：US20230263828A1

公开(公告)日：2023-08-24

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 ,  C12N15/11 ,  C07K14/725 ,  C07K16/28 ,  C12N5/16 ,  C12N15/113 ,  C12N15/86

CPC classification number: 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

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：US20230070540A1

公开(公告)日：2023-03-09

申请号：US17818669

申请日：2022-08-09

Applicant: CRISPR THERAPEUTICS AG

Inventor： Alireza REZANIA ,  Rebeca RAMOS-ZAYAS

IPC: A61K35/39 ,  C12N5/074 ,  C12N5/071 ,  C12N5/10 ,  C12N5/16 ,  C07K14/74 ,  C12N5/0735

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 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.

公开(公告)号：US11591381B2

公开(公告)日：2023-02-28

申请号：US17538699

申请日：2021-11-30

Applicant: CRISPR THERAPEUTICS AG

Inventor： Valentin Sluch ,  Alireza Rezania ,  Jason Sagert ,  Danielle Swain

IPC: 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

Abstract: 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.