公开(公告)号：US20250025580A1

公开(公告)日：2025-01-23

申请号：US18415032

申请日：2024-01-17

Applicant: Intellia Therapeutics, Inc.

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

IPC: A61K48/00 ,  A61K9/127 ,  A61K9/51 ,  A61K38/46 ,  A61P25/14 ,  A61P27/02 ,  C12N15/10

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：US20240417722A1

公开(公告)日：2024-12-19

申请号：US18534209

申请日：2023-12-08

Applicant: NOVARTIS AG ,  INTELLIA THERAPEUTICS, INC.

Inventor： Ming-Wei CHEN ,  Melissa DECK ,  Glenn DRANOFF ,  Craig Stephen MICKANIN ,  Reynald LESCARBEAU ,  Celeste RICHARDSON ,  Morag STEWART ,  Yi YANG

IPC: C12N15/113 ,  A61K9/00 ,  A61K31/7088 ,  A61K38/17 ,  A61K38/46 ,  A61K39/00 ,  A61K45/06 ,  C12N5/0783 ,  C12N15/09 ,  C12N15/10

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

公开(公告)号：US20240002843A1

公开(公告)日：2024-01-04

申请号：US17965366

申请日：2022-10-13

Applicant: Novartis AG ,  Intellia Therapeutics, Inc.

Inventor： Anthony Edward BOITANO ,  Michael COOKE ,  Lloyd B. KLICKSTEIN ,  Reynald LESCARBEAU ,  Craig Stephen MICKANIN ,  Kabungo MULUMBA ,  Seshidhar Reddy POLICE ,  Jennifer SNEAD ,  Susan C. STEVENSON ,  Morag STEWART ,  Yi YANG

IPC: C12N15/113 ,  A61K35/15 ,  A61P7/00

CPC classification number: C12N15/113 ,  A61K35/15 ,  A61P7/00 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/10 ,  C12N2310/321 ,  C12N2310/20

Abstract: The present invention is directed to genome editing systems, reagents and methods for the treatment of hemoglobinopathies.

公开(公告)号：US20230295587A1

公开(公告)日：2023-09-21

申请号：US17882099

申请日：2022-08-05

Applicant: Intellia Therapeutics, Inc.

Inventor： Shobu Odate ,  Jessica Lynn Seitzer

IPC: C12N9/22 ,  A61P43/00 ,  C12N15/10

CPC classification number: C12N9/22 ,  A61P43/00 ,  C12N15/102 ,  C12N2310/20 ,  C12N2310/32

Abstract: Compositions and methods for editing, e.g., introducing double-stranded breaks, within the KLKB1 gene are provided. Compositions and methods for treating subjects having hereditary angioedema (HAE), are provided.

公开(公告)号：US20230271997A1

公开(公告)日：2023-08-31

申请号：US18310298

申请日：2023-05-01

Applicant: Intellia Therapeutics, Inc.

Inventor： Christian Dombrowski

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

CPC classification number: C07H21/02 ,  C12N15/11 ,  C12N15/63 ,  C07K14/475

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：US20230257747A1

公开(公告)日：2023-08-17

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: C12N15/113 ,  C12N9/22 ,  C12N15/10 ,  A61P25/28

CPC classification number: C12N15/113 ,  A61P25/28 ,  C12N9/22 ,  C12N15/102 ,  A61K48/00 ,  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：US20230118337A1

公开(公告)日：2023-04-20

申请号：US17808802

申请日：2022-06-24

Applicant: Novartis AG ,  Intellia Therapeutics, Inc.

Inventor： Craig Stephen MICKANIN ,  Christian SCHMEDT ,  Jennifer SNEAD ,  Susan C. Stevenson ,  Yi YANG

IPC: C12N15/113 ,  C12N15/10 ,  A61K31/713 ,  C12N9/22

Abstract: The present invention is directed to genome editing systems, reagents and methods for the treatment of hemoglobinopathies.

公开(公告)号：US20220372483A1

公开(公告)日：2022-11-24

申请号：US17836265

申请日：2022-06-09

Applicant: Intellia Therapeutics, Inc.

Inventor： Seth C. Alexander ,  Sabin Mulepati ,  Matthew Roy

IPC: C12N15/113 ,  C12N9/22

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

公开(公告)号：US11466271B2

公开(公告)日：2022-10-11

申请号：US16484046

申请日：2018-02-05

Applicant: Novartis AG ,  Intellia Therapeutics, Inc.

Inventor： Craig Stephen Mickanin ,  Christian Schmedt ,  Jennifer Snead ,  Susan Stevenson ,  Yi Yang

IPC: C12N15/113 ,  A61K31/713 ,  C12N9/22 ,  C12N15/10

Abstract: The present invention is directed to genome editing systems, reagents and methods for the treatment of hemoglobinopathies.

公开(公告)号：US20210340530A1

公开(公告)日：2021-11-04

申请号：US17231556

申请日：2021-04-15

Applicant: Intellia Therapeutics, Inc.

Inventor： Amy Melissa Becker ,  Surbhi Goel ,  Sarah Beth Hesse ,  Troy Aaron Luster ,  Birgit Schultes ,  Stephanie A. Yazinski ,  Pooja Vinay

IPC: C12N15/11 ,  C12N9/22 ,  C07K14/725

Abstract: Compositions and methods for editing, e.g., altering a DNA sequence, within the TRBC1, TRBC2 and/or TRAC genes are provided. Compositions and methods for immunotherapy are provided, for example.