CN116018403A - 体外细胞递送方法 - Google Patents

体外细胞递送方法 Download PDF

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Publication number
CN116018403A
CN116018403A CN202180044410.4A CN202180044410A CN116018403A CN 116018403 A CN116018403 A CN 116018403A CN 202180044410 A CN202180044410 A CN 202180044410A CN 116018403 A CN116018403 A CN 116018403A
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cells
population
cell
lipid
nucleic acid
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P·K·纳伦德拉
S·M·伯恩斯
P·古铁雷斯马丁内斯
A·M·P·坎乔利亚
A·蒙蒂
A·普罗迪厄斯
M·S·阿雷杜阿尼
O·基利奇
R·W·拉里维耶尔
P·S·夏尔马
E·斯坦普洛格鲁
张青占
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Intellia Therapeutics Inc
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Intellia Therapeutics Inc
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CN202180044410.4A 2020-04-28 2021-04-27 体外细胞递送方法 Pending CN116018403A (zh)

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US202063016913P 2020-04-28 2020-04-28
US63/016,913 2020-04-28
US202063121781P 2020-12-04 2020-12-04
US63/121,781 2020-12-04
US202063124058P 2020-12-11 2020-12-11
US63/124,058 2020-12-11
US202063130100P 2020-12-23 2020-12-23
US63/130,100 2020-12-23
US202163165619P 2021-03-24 2021-03-24
US63/165,619 2021-03-24
US202163176221P 2021-04-17 2021-04-17
US63/176,221 2021-04-17
PCT/US2021/029446 WO2021222287A2 (en) 2020-04-28 2021-04-27 Methods of in vitro cell delivery

Publications (1)

Publication Number Publication Date
CN116018403A true CN116018403A (zh) 2023-04-25

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CN202180044410.4A Pending CN116018403A (zh) 2020-04-28 2021-04-27 体外细胞递送方法

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US (1) US20230183753A1 (https=)
EP (1) EP4143304A2 (https=)
JP (1) JP2023524666A (https=)
KR (1) KR20230017783A (https=)
CN (1) CN116018403A (https=)
AU (1) AU2021263745A1 (https=)
BR (1) BR112022021676A2 (https=)
CA (1) CA3181340A1 (https=)
CL (1) CL2022002974A1 (https=)
IL (1) IL297550A (https=)
MX (1) MX2022013403A (https=)
TW (1) TW202204612A (https=)
WO (1) WO2021222287A2 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
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IL303195A (en) 2020-11-25 2023-07-01 Akagera Medicines Inc Lipid nanoparticles for delivery of nucleic acids and related methods of use
AU2021409732A1 (en) * 2020-12-23 2023-07-20 Intellia Therapeutics, Inc. Compositions and methods for reducing hla-a in a cell
JP2024505672A (ja) 2021-02-08 2024-02-07 インテリア セラピューティクス,インコーポレイテッド 免疫療法のためのナチュラルキラー細胞受容体2b4組成物及び方法
WO2022170194A2 (en) 2021-02-08 2022-08-11 Intellia Therapeutics, Inc. Lymphocyte activation gene 3 (lag3) compositions and methods for immunotherapy
JP2024506016A (ja) 2021-02-08 2024-02-08 インテリア セラピューティクス,インコーポレイテッド 免疫療法のためのt細胞免疫グロブリン及びムチンドメイン3(tim3)組成物及び方法
KR20240032013A (ko) 2021-06-10 2024-03-08 인텔리아 테라퓨틱스, 인크. 유전자 편집을 위한 내부 링커를 포함하는 변형된 가이드 rna
WO2023274386A1 (zh) * 2021-07-01 2023-01-05 宁波茂行生物医药科技有限公司 靶向egfr的通用型car-t细胞及其制备方法
EP4392060A1 (en) 2021-08-24 2024-07-03 Intellia Therapeutics, Inc. Programmed cell death protein 1 (pd1) compositions and methods for cell-based therapy
GB202117583D0 (en) * 2021-12-06 2022-01-19 Cambridge Entpr Ltd Protein expression
US20250049850A1 (en) * 2021-12-15 2025-02-13 Regents Of The University Of Minnesota Non-viral b cell engineering methods
CN117004604A (zh) * 2022-04-28 2023-11-07 南京北恒生物科技有限公司 一种ciita基因被敲除的工程化免疫细胞及其用途
EP4531819A2 (en) 2022-05-25 2025-04-09 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids and methods of use thereof
CN119384498A (zh) * 2022-06-16 2025-01-28 因特利亚治疗公司 用于对细胞进行遗传修饰的方法和组合物
CN119585419A (zh) 2022-06-29 2025-03-07 因特利亚治疗公司 工程化t细胞
CN119730863A (zh) * 2022-08-10 2025-03-28 普洛迈博生物技术公司 用car rna-lnp转染的nk细胞
WO2024118634A2 (en) * 2022-11-28 2024-06-06 The Trustees Of The University Of Pennsylvania In vivo expansion of car t cells and delivery of car target antigen to tumors using mrna lipid nanoparticles
WO2024137766A2 (en) 2022-12-21 2024-06-27 Intellia Therapeutics, Inc. Compositions and methods for proprotein convertase subtilisin kexin 9 (pcsk9) editing
WO2024138189A2 (en) 2022-12-22 2024-06-27 Intellia Therapeutics, Inc. Methods for analyzing nucleic acid cargos of lipid nucleic acid assemblies
WO2024186890A1 (en) 2023-03-06 2024-09-12 Intellia Therapeutics, Inc. Compositions and methods for hepatitis b virus (hbv) genome editing
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025137301A1 (en) * 2023-12-20 2025-06-26 Intellia Therapeutics, Inc. Methods for rapid engineering of cells
WO2025137439A2 (en) 2023-12-20 2025-06-26 Intellia Therapeutics, Inc. Engineered t cells
WO2025184520A1 (en) 2024-02-29 2025-09-04 Intellia Therapeutics, Inc. Compositions and methods for angiopoietin like 3 (angptl3) editing
WO2025244134A1 (en) * 2024-05-24 2025-11-27 Fujifilm Corporation Method to deliver a payload to cells, and lipid composition

Family Cites Families (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
KR940703846A (ko) 1991-12-24 1994-12-12 비. 린네 파샬 갭(gap)이 형성된 2′ 변성된 올리고뉴클레오티드(gapped 2′ modifed oligonucleotides)
US6169169B1 (en) 1994-05-19 2001-01-02 Dako A/S PNA probes for detection of Neisseria gonorrhoeae and Chlamydia trachomatis
GB0119865D0 (en) 2001-08-14 2001-10-10 Cancer Res Campaign Tech DNA-PK inhibitors
WO2006007712A1 (en) 2004-07-19 2006-01-26 Protiva Biotherapeutics, Inc. Methods comprising polyethylene glycol-lipid conjugates for delivery of therapeutic agents
WO2011091324A2 (en) 2010-01-22 2011-07-28 The Scripps Research Institute Methods of generating zinc finger nucleases having altered activity
CN103668470B (zh) 2012-09-12 2015-07-29 上海斯丹赛生物技术有限公司 一种dna文库及构建转录激活子样效应因子核酸酶质粒的方法
DK2931898T3 (en) 2012-12-12 2016-06-20 Massachusetts Inst Technology CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION WITH FUNCTIONAL DOMAINS
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
CA2895155C (en) 2012-12-17 2021-07-06 President And Fellows Of Harvard College Rna-guided human genome engineering
EP3608308B1 (en) 2013-03-08 2021-07-21 Novartis AG Lipids and lipid compositions for the delivery of active agents
SI3527563T1 (sl) 2013-03-12 2022-01-31 Vertex Pharmaceuticals Incorporated Inhibitorji DNA-PK
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
ES2895651T3 (es) 2013-12-19 2022-02-22 Novartis Ag Lípidos y composiciones lipídicas para la administración de agentes activos
EP3169309B1 (en) 2014-07-16 2023-05-10 Novartis AG Method of encapsulating a nucleic acid in a lipid nanoparticle host
TWI773666B (zh) 2016-03-30 2022-08-11 美商英特利亞醫療公司 Crispr/cas 組分之脂質奈米粒子調配物
WO2018073393A2 (en) 2016-10-19 2018-04-26 Cellectis Tal-effector nuclease (talen) -modified allogenic cells suitable for therapy
EP3551169A4 (en) * 2016-12-09 2020-09-09 Sangamo Therapeutics, Inc. TARGET-SPECIFIC NUCLEASES ADMINISTRATION
SMT202100225T1 (it) 2016-12-20 2021-05-07 Astrazeneca Ab Composti di ammino-triazolopiridina e loro uso nel trattamento del cancro
MX2019013514A (es) * 2017-05-12 2020-01-20 Crispr Therapeutics Ag Materiales y metodos para modificar celulas por ingenieria genetica y usos de los mismos en inmunooncologia.
PT3688162T (pt) 2017-09-29 2024-04-23 Intellia Therapeutics Inc Formulações
BR112020006256A2 (pt) * 2017-09-29 2020-10-20 Intellia Therapeutics, Inc. método in vitro de administração de mrna usando nanopartículas de lipídio
WO2019147805A2 (en) 2018-01-26 2019-08-01 The Board Of Trustees Of The Leland Stanford Junior University Regulatory t cells targeted with chimeric antigen receptors
CN112292139B (zh) * 2018-03-13 2024-03-08 明尼苏达大学董事会 使用Cas9碱基编辑器的淋巴造血改造
WO2020072605A1 (en) 2018-10-02 2020-04-09 Intellia Therapeutics, Inc. Ionizable amine lipids
IL314386A (en) 2018-12-05 2024-09-01 Intellia Therapeutics Inc amine modified lipids
WO2020219876A1 (en) 2019-04-25 2020-10-29 Intellia Therapeutics, Inc. Ionizable amine lipids and lipid nanoparticles

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025217892A1 (zh) * 2024-04-19 2025-10-23 深圳市先康达生命科学有限公司 多位点同时敲入dna片段的基因编辑方法及其应用

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