公开(公告)号：US20230183753A1

公开(公告)日：2023-06-15

申请号：US18050333

申请日：2022-10-27

Applicant: Intellia Therapeutics, Inc.

Inventor： Pooja Kyatsandra Narendra ,  Sean Michael Burns ,  Paula Gutierrez Martinez ,  Arti Mahendra Prakash Kanjolia ,  Anthony Monti ,  Aaron Prodeus ,  Mohamed Simo Arredouani ,  Özgün Kiliç ,  Reed Walker LaRiviere ,  Palak Sushil Sharma ,  Eleni Stampouloglou ,  Qingzhan Zhang

IPC: C12N15/90 ,  C12N5/0783 ,  C12N15/87 ,  C12N15/11 ,  C12N9/22 ,  A61K38/17 ,  A61K35/17 ,  A61P35/00

CPC classification number: C12N15/907 ,  C12N5/0636 ,  C12N15/87 ,  C12N15/11 ,  C12N9/22 ,  A61K38/1774 ,  A61K35/17 ,  A61P35/00 ,  C12N2510/00 ,  C12N2310/20 ,  C12N2800/80

Abstract: Compositions and methods for multiplex delivery and gene editing in vitro are provided.

公开(公告)号：US20250090471A1

公开(公告)日：2025-03-20

申请号：US18287239

申请日：2022-04-15

Applicant: Intellia Therapeutics, Inc.

Inventor： Archana Swami ,  Vishal Rakshe ,  Aaron Prodeus ,  Micah Maetani ,  Rubina Giare Parmar

IPC: A61K9/51 ,  A61K31/7105 ,  C12N9/22 ,  C12N15/11 ,  C12N15/88 ,  C12N15/90

Abstract: The disclosure provides lipid nanoparticle (LNP) compositions of ionizable lipids, helper lipids, neutral lipids, and PEG lipids useful for the delivery of biologically active agents, for example delivering biologically active agents to cells to prepare engineered cells. The LNP compositions disclosed herein are useful in methods of gene editing and methods of delivering a biologically active agent and methods of modifying or cleaving DNA.

公开(公告)号：US20240300977A1

公开(公告)日：2024-09-12

申请号：US18287227

申请日：2022-04-15

Applicant: Intellia Therapeutics, Inc.

Inventor： Micah Maetani ,  Xin Jenny Xie ,  Anthony Forget ,  Aaron Prodeus ,  Stephanie Yazinski ,  Rubina Giare Parmar

IPC: C07D519/00 ,  C12N5/071 ,  C12N5/0783 ,  C12N9/22 ,  C12N15/113 ,  C12N15/90

CPC classification number: C07D519/00 ,  C12N5/0636 ,  C12N5/067 ,  C12N9/22 ,  C12N15/113 ,  C12N15/907 ,  C12N2310/20 ,  C12N2510/00

Abstract: The present disclosure relates to inhibitors of DNA protein kinase, and compositions and methods of use thereof. In some embodiments, the inhibitors have the structure of Formula I:






or a salt thereof,
wherein:

x1 is C—R3 or N;
R1 is C1-C3 alkyl;
R2 is cycloalkyl or heterocyclyl, and cycloalkyl and heterocyclyl are optionally substituted with one or more R6;
R3 is H or C1-C3 alkyl;
R4 is H or C1-C3 alkyl;
R5 is C1-C3 alkyl;
each R6 is independently selected from hydroxy, halo, alkyl, alkoxy, cycloalkyl, amino, and cyano, or two R6, taken together with the atom or atoms to which they are bonded, form a spirocyclic or fused ring; and
R7 is H or C1-C3 alkyl.