公开(公告)号：US20240124897A1

公开(公告)日：2024-04-18

申请号：US18362867

申请日：2023-07-31

Applicant: Intellia Therapeutics, Inc.

Inventor： Yong Chang ,  Seth C. Alexander ,  Kristy M. Wood ,  Arti Mahendra Prakash Kanjolia ,  Shobu Odate ,  Jessica Lynn Seitzer ,  Reynald Michael Lescarbeau ,  Walter Strapps

IPC: C12N15/90 ,  C12N9/22 ,  C12N15/11

CPC classification number: C12N15/907 ,  C12N9/22 ,  C12N15/11 ,  A61K38/00 ,  C12N2310/20

Abstract: Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene are provided. Compositions and methods for treating subjects having amyloidosis associated with transthyretin (ATTR), are provided.

公开(公告)号：US20230203480A1

公开(公告)日：2023-06-29

申请号：US17897878

申请日：2022-08-29

Applicant: Intellia Therapeutics, Inc.

Inventor： David V. Morrissey ,  Mihir Chandrakant Patel ,  Jonathan D. Finn ,  Amy Madison Rhoden Smith ,  Lucinda J. Shaw ,  Christian Dombrowksi ,  Ruchi Rudraprasad Shah

IPC: C12N15/11 ,  C12N9/22 ,  C12N15/88 ,  C12N5/071

CPC classification number: C12N15/111 ,  C12N9/22 ,  C12N15/88 ,  C12N5/0602 ,  C12N2320/32 ,  C12N2310/20 ,  C12N2510/00

Abstract: The invention provides lipid nanoparticle-based compositions and methods useful for delivery of CRISPR/Cas gene editing components.

公开(公告)号：US20230012687A1

公开(公告)日：2023-01-19

申请号：US17486039

申请日：2021-09-27

Applicant: Intellia Therapeutics, Inc.

Inventor： Bradley Andrew Murray ,  Christian Dombrowski ,  Seth C. Alexander

IPC: C12N15/67 ,  C12N9/22 ,  C12N15/90 ,  C12N15/88 ,  C12N15/11

Abstract: Compositions and methods for gene editing. In some embodiments, a polynucleotide encoding Cas9 is provided that can provide one or more of improved editing efficiency, reduced immunogenicity, or other benefits.

公开(公告)号：US20210222173A1

公开(公告)日：2021-07-22

申请号：US17212901

申请日：2021-03-25

Applicant: Intellia Therapeutics, Inc.

Inventor： Zachary William Dymek ,  Shobu Odate ,  Anette Huebner ,  Srijani Sridhar ,  Bradley Andrew Murray ,  Walter Strapps

IPC: C12N15/113 ,  A61P13/02 ,  C12N9/22

Abstract: Compositions and methods for editing, e.g., introducing double-stranded breaks, within the LDHA gene are provided. Compositions and methods for treating subjects having hyperoxaluria are provided.

公开(公告)号：US20210123075A1

公开(公告)日：2021-04-29

申请号：US17058381

申请日：2019-06-07

Applicant: NOVARTIS AG ,  INTELLIA THERAPEUTICS, INC.

Inventor： Ming-Wei CHEN ,  Glenn DRANOFF

IPC: C12N15/86 ,  C12N9/22 ,  C12N15/113

Abstract: The present disclosure is directed to genome editing systems, reagents and methods for immunooncology.

公开(公告)号：US20210071182A1

公开(公告)日：2021-03-11

申请号：US16498361

申请日：2018-03-21

Applicant: NOVARTIS AG ,  INTELLIA THERAPEUTICS, INC.

Inventor： Jennifer BROGDON ,  Ming-Wei CHEN ,  Hyungwook LIM ,  Yi YANG ,  Morag STEWART ,  Sarah HESSE

IPC: C12N15/113 ,  C07K14/705 ,  A61K35/17 ,  C12N9/22 ,  C12N15/86

Abstract: The present disclosure is directed to genome editing systems, reagents and methods for immunooncology.

公开(公告)号：US20200308603A1

公开(公告)日：2020-10-01

申请号：US16651918

申请日：2018-09-28

Applicant: Intellia Therapeutics, Inc.

Inventor： Morag H. Stewart ,  Markus P. Vallaster ,  Anthony Monti ,  Pooja K. Narendra ,  Quan Ho

IPC: C12N15/88 ,  C12N15/113 ,  C12N5/0789

Abstract: The present disclosure relates to compositions and methods for introducing an mRNA into stem cells, such as HSPCs, and for delivering gene editing components to such cells in vitro. For example, the disclosure relates to modifying a gene sequence using a CRISPR-Cas9 complex in HSPCs, and methods and delivery systems for achieving such gene modification in HSPCs.

公开(公告)号：US20200172564A1

公开(公告)日：2020-06-04

申请号：US16791076

申请日：2020-02-14

Applicant: Intellia Therapeutics, Inc.

Inventor： Christian Dombrowski

IPC: C07H21/02 ,  C12N15/11 ,  C12N15/63

Abstract: This disclosure relates to the field of poly-adenylated (poly-A) tails. In some embodiments, a DNA encodes a poly-A tail located 3′ to nucleotides encoding a protein of interest, wherein the poly-A tail comprises one or more non-adenine nucleotide.

公开(公告)号：US20190142972A1

公开(公告)日：2019-05-16

申请号：US16095236

申请日：2017-04-21

Applicant: Intellia Therapeutics, Inc.

Inventor： Sean Michael Burns ,  Bradley Andrew Murray ,  Sarah Beth Hesse

IPC: A61K48/00 ,  A61K38/46 ,  A61K9/51 ,  A61P27/02 ,  A61P25/14 ,  A61K9/127

Abstract: This application relates to compositions and methods for excising trinucleotide repeats (TNRs) contained within intron 3 of TCF4, such as is seen in subjects having Fuchs endothelial corneal dystrophy (FECD), PSC, and Schizophrenia. Compositions comprising guide sequences targeting the alpha 2 subunit of collagen VIII are also disclosed for treatment of mutations therein that may contribute to FECD.

公开(公告)号：US20170260547A1

公开(公告)日：2017-09-14

申请号：US15457866

申请日：2017-03-13

Applicant: Intellia Therapeutics, Inc.

Inventor： Christian Dombrowski ,  Thomas Michael Barnes

IPC: C12N15/90 ,  C12N7/00 ,  C12N15/86 ,  C12N9/22 ,  C12N15/11

CPC classification number: C12N15/907 ,  C12N7/00 ,  C12N9/22 ,  C12N15/102 ,  C12N15/11 ,  C12N15/111 ,  C12N15/86 ,  C12N2310/20 ,  C12N2830/005

Abstract: Compositions and methods are provided for enhancing the efficiency of gene editing by timing the expression and activity of a nuclease to correspond with availability of a repair template. Compositions and methods for temporally regulating the duration of nuclease activity, and methods of selectively preventing nuclease expression during viral vector production, are also provided.