公开(公告)号：US20210163943A1

公开(公告)日：2021-06-03

申请号：US17162377

申请日：2021-01-29

Applicant: Intellia Therapeutics, Inc.

Inventor： Zachary William Dymek ,  Shobu Odate ,  Bradley Andrew Murray ,  Reynald Michael Lescarbeau ,  Anette Huebner ,  Walter Strapps ,  Sarah Beth Hesse

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

Abstract: Compositions and methods for editing, e.g., introducing double-stranded breaks, within the HAO1 gene are provided. Compositions and methods for treating subjects having primary hyperoxaluria type 1 (PH1), are provided.

公开(公告)号：US20200268906A1

公开(公告)日：2020-08-27

申请号：US16657939

申请日：2019-10-18

Applicant: Intellia Therapeutics, Inc. ,  Regeneron Pharmaceuticals, Inc.

Inventor： John Finn ,  Hon-Ren Huang

IPC: A61K48/00 ,  C12N15/11 ,  C12N7/00 ,  A61K9/51 ,  C12N9/22 ,  C12N9/64 ,  C07K14/765

Abstract: Nucleic acid constructs that allow insertion and/or expression of a sequence of interest, such as a transgene, are provided. Compositions and methods of using such constructs for expression of a polypeptide or therapeutic agent, for example, are also provided.

公开(公告)号：US12173284B2

公开(公告)日：2024-12-24

申请号：US17900960

申请日：2022-09-01

Applicant: Intellia Therapeutics, Inc.

Inventor： Amy Madison Rhoden Smith ,  David V. Morrissey ,  Walter Strapps

IPC: C07H21/02 ,  C12N9/22 ,  C12N15/11

Abstract: This disclosure relates to modified single and dual guide RNAs having improved in vitro and in vivo activity in gene editing methods.

公开(公告)号：US20240417752A1

公开(公告)日：2024-12-19

申请号：US18742805

申请日：2024-06-13

Applicant: Regeneron Pharmaceuticals, Inc. ,  Intellia Therapeutics, Inc.

Inventor： Ayush Saxena ,  Rong-sen Calvin Chen

IPC: C12N15/86 ,  C12Q1/6869

Abstract: Disclosed are data pipelines for string extraction, clustering, and comparison. A method may include extracting, from each plasmid genome sequence sequenced from genomes of plasmids, based on the presence of fixed flanking sequence markers (FFSMs) in the plasmid genome sequence, sequence regions. Each sequence region is within the FFSMs and includes a candidate inverted terminal repeat (ITR) sequence. The example method further includes clustering, based on perfect sequence identity, two or more sequence regions of the sequence regions to generate a clusters, merging, based on an alignment between their corresponding sequence regions, two or more clusters of the clusters; when a single cluster remains, identifying, based on a local alignment, a genotype of a candidate ITR sequence of the single cluster, and manufacturing, based on the genotype of the candidate ITR sequence, a plurality of AAV vectors using plasmids having ITR sequences with the genotype of the candidate ITR sequence.

公开(公告)号：US12077483B2

公开(公告)日：2024-09-03

申请号：US17299925

申请日：2019-12-05

Applicant: Intellia Therapeutics, Inc.

Inventor： Stephen S. Scully ,  Micah Maetani ,  Ramsey Majzoub

IPC: C07C219/04 ,  A61P1/16 ,  C07C271/20 ,  C07D205/04 ,  C07D207/09 ,  C07D211/46 ,  C07D211/62 ,  C07D271/10 ,  C07D295/08 ,  C12N15/113 ,  B82Y5/00 ,  B82Y30/00 ,  B82Y40/00

CPC classification number: C07C219/04 ,  A61P1/16 ,  C07C271/20 ,  C07D205/04 ,  C07D207/09 ,  C07D211/46 ,  C07D211/62 ,  C07D271/10 ,  C07D295/08 ,  C12N15/113 ,  B82Y5/00 ,  B82Y30/00 ,  B82Y40/00 ,  C12N2310/315 ,  C12N2310/321

Abstract: The disclosure provides ionizable amine lipids and salts thereof (e.g., pharmaceutically acceptable salts thereof) useful for the delivery of biologically active agents, for example delivering biologically active agents to cells to prepare engineered cells. The ionizable amine lipids disclosed herein are useful as ionizable lipids in the formulation of lipid nanoparticle-based compositions.

公开(公告)号：US11965165B2

公开(公告)日：2024-04-23

申请号：US18063972

申请日：2022-12-09

Applicant: Intellia Therapeutics, Inc.

Inventor： Arti Mahendra Prakash Kanjolia ,  Shobu Odate ,  Jessica Lynn Seitzer ,  Reynald Michael Lescarbeau ,  Walter Strapps

IPC: A61P25/28 ,  C12N9/22 ,  C12N15/10 ,  C12N15/113 ,  A61K48/00 ,  C12N15/11

CPC classification number: C12N15/113 ,  A61P25/28 ,  C12N9/22 ,  C12N15/102 ,  A61K48/00 ,  A61K48/0091 ,  C12N15/111 ,  C12N2310/20 ,  C12N2310/321 ,  C12N2800/80

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：US20240076636A1

公开(公告)日：2024-03-07

申请号：US18132278

申请日：2023-04-07

Applicant: Intellia Therapeutics, Inc.

Inventor： Christian Dombrowski ,  Jonathan Douglas Finn ,  Amy Madison Rhoden Smith ,  Seth C. Alexander

IPC: C12N9/22 ,  A61K9/51 ,  C12N15/11 ,  C12N15/85 ,  C12N15/88

CPC classification number: C12N9/22 ,  A61K9/5123 ,  C12N15/11 ,  C12N15/111 ,  C12N15/85 ,  C12N15/88 ,  C12N2310/20 ,  C12N2320/32 ,  C12N2800/80

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：US20240016934A1

公开(公告)日：2024-01-18

申请号：US18332390

申请日：2023-06-09

Applicant: Intellia Therapeutics, Inc.

Inventor： Srijani Sridhar ,  Yong Zhang ,  William Frederick Harrington ,  Surbhi Goel

IPC: A61K39/00 ,  A61P37/02 ,  C12N5/0783 ,  A61K35/17 ,  C12N15/86

CPC classification number: A61K39/4632 ,  A61P37/02 ,  C12N5/0636 ,  A61K35/17 ,  C12N15/86 ,  C12N2510/00 ,  C12N2750/14143

Abstract: Compositions and methods for reducing MHC class II protein expression in a cell comprising genetically modifying CIITA for use e.g., in adoptive cell transfer therapies.

公开(公告)号：US20230140670A1

公开(公告)日：2023-05-04

申请号：US16651911

申请日：2018-09-28

Applicant: Intellia Therapeutics, Inc.

Inventor： Kristy M. Wood ,  Noah P. Gardner ,  Ruchi R. Shah ,  Stephen S. Scully ,  Ramsey Majzoub

IPC: C12N15/88 ,  A61K9/127 ,  C12N15/11 ,  C12N9/22

Abstract: The invention provides lipid nanoparticle-based compositions with improved properties for delivery of biologically active agents, engineered cells, and methods for delivery of the agents.

公开(公告)号：US20230076357A1

公开(公告)日：2023-03-09

申请号：US17735929

申请日：2022-05-03

Applicant: Intellia Therapeutics, Inc.

Inventor： Schaked Omer HALPERIN

IPC: C12N15/11 ,  C12N9/22 ,  C12N15/90 ,  C12N9/12 ,  C12N15/113 ,  C12N15/52

Abstract: Provided herein are compositions and methods for increasing editing efficiency of a target nucleic acid. A composition may comprise a guide nucleic acid, a Cas9 nickase, or a reverse transcriptase. The reverse transcriptase may be fused to the Cas9 nickase. The reverse transcriptase may heterodimerize with the Cas9 nickase. The reverse transcriptase may bind to a guide nucleic acid. The reverse transcriptase may be engineered to increase processivity. The guide nucleic acid may be engineered to facilitate synthesis or editing of a sequence. The guide nucleic acid, Cas9 nickase, and reverse transcriptase may be engineered to fit within AAV vectors. The guide nucleic acid may comprise a region that binds to another region on the guide nucleic acid to improve gene editing.