BR112022021676A2 - Métodos de distribuição de célula in vitro - Google Patents

Métodos de distribuição de célula in vitro

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Publication number
BR112022021676A2
BR112022021676A2 BR112022021676A BR112022021676A BR112022021676A2 BR 112022021676 A2 BR112022021676 A2 BR 112022021676A2 BR 112022021676 A BR112022021676 A BR 112022021676A BR 112022021676 A BR112022021676 A BR 112022021676A BR 112022021676 A2 BR112022021676 A2 BR 112022021676A2
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BR
Brazil
Prior art keywords
vitro cell
cell delivery
delivery methods
methods
vitro
Prior art date
Application number
BR112022021676A
Other languages
English (en)
Inventor
Kyatsandra Narendra Pooja
Michael Burns Sean
Gutierrez Martinez Paula
Mahendra Prakash Kanjolia Arti
Monti Anthony
Prodeus Aaron
Simo Arredouani Mohamed
Kiliç Özgün
Walker Lariviere Reed
Sushil Sharma Palak
Stampouloglou Eleni
Zhang Qingzhan
Original Assignee
Intellia Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Intellia Therapeutics Inc filed Critical Intellia Therapeutics Inc
Publication of BR112022021676A2 publication Critical patent/BR112022021676A2/pt

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    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
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    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • A61K38/177Receptors; Cell surface antigens; Cell surface determinants
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    • A61K39/4611T-cells, e.g. tumor infiltrating lymphocytes [TIL], lymphokine-activated killer cells [LAK] or regulatory T cells [Treg]
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    • A61K39/464Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643Vertebrate antigens
    • A61K39/4644Cancer antigens
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    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • C12N5/0636T lymphocytes
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases RNAses, DNAses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00Indexing codes associated with cellular immunotherapy of group A61K39/46
    • A61K2239/27Indexing codes associated with cellular immunotherapy of group A61K39/46 characterized by targeting or presenting multiple antigens
    • A61K2239/28Expressing multiple CARs, TCRs or antigens
    • AHUMAN NECESSITIES
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    • A61K2239/31Indexing codes associated with cellular immunotherapy of group A61K39/46 characterized by the route of administration
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    • A61K2239/46Indexing codes associated with cellular immunotherapy of group A61K39/46 characterised by the cancer treated
    • A61K2239/48Blood cells, e.g. leukemia or lymphoma
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Abstract

MÉTODOS DE DISTRIBUIÇÃO DE CÉLULA IN VITRO. Composições e métodos para distribuição multiplex e edição de gene in vitro são fornecidos.
BR112022021676A 2020-04-28 2021-04-27 Métodos de distribuição de célula in vitro BR112022021676A2 (pt)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202063016913P 2020-04-28 2020-04-28
US202063121781P 2020-12-04 2020-12-04
US202063124058P 2020-12-11 2020-12-11
US202063130100P 2020-12-23 2020-12-23
US202163165619P 2021-03-24 2021-03-24
US202163176221P 2021-04-17 2021-04-17
PCT/US2021/029446 WO2021222287A2 (en) 2020-04-28 2021-04-27 Methods of in vitro cell delivery

Publications (1)

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BR112022021676A2 true BR112022021676A2 (pt) 2023-01-31

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BR112022021676A BR112022021676A2 (pt) 2020-04-28 2021-04-27 Métodos de distribuição de célula in vitro

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

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2021385572A1 (en) 2020-11-25 2023-06-22 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids, and related methods of use
CR20230320A (es) * 2020-12-23 2023-10-23 Intellia Therapeutics Inc Composiciones y métodos para reducir los hla-a en una célula
EP4288089A2 (en) 2021-02-08 2023-12-13 Intellia Therapeutics, Inc. T-cell immunoglobulin and mucin domain 3 (tim3) compositions and methods for immunotherapy
EP4288088A2 (en) 2021-02-08 2023-12-13 Intellia Therapeutics, Inc. Lymphocyte activation gene 3 (lag3) compositions and methods for immunotherapy
WO2022170172A1 (en) 2021-02-08 2022-08-11 Intellia Therapeutics, Inc. Natural killer cell receptor 2b4 compositions and methods for immunotherapy
BR112023025724A2 (pt) 2021-06-10 2024-02-27 Intellia Therapeutics Inc Rnas-guia modificados que compreendem um ligante interno para edição de gene
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
WO2023028471A1 (en) 2021-08-24 2023-03-02 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
WO2023114899A1 (en) * 2021-12-15 2023-06-22 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
WO2023245113A1 (en) * 2022-06-16 2023-12-21 Intellia Therapeutics, Inc. Methods and compositions for genetically modifying a cell
WO2024006955A1 (en) 2022-06-29 2024-01-04 Intellia Therapeutics, Inc. Engineered t cells
WO2024036188A2 (en) * 2022-08-10 2024-02-15 Promab Biotechnologies, Inc. Nk cells transfected with car rna-lnp

Family Cites Families (24)

* 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
ATE204879T1 (de) 1991-12-24 2001-09-15 Isis Pharmaceuticals Inc Antisense oligonukleotide
JPH10500310A (ja) 1994-05-19 1998-01-13 ダコ アクティーゼルスカブ 淋菌及びトラコーマ クラミジアの検出のためのpna プローブ
GB0119865D0 (en) 2001-08-14 2001-10-10 Cancer Res Campaign Tech DNA-PK inhibitors
US20060051405A1 (en) 2004-07-19 2006-03-09 Protiva Biotherapeutics, Inc. Compositions for the delivery of therapeutic agents and uses thereof
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
ES2576128T3 (es) 2012-12-12 2016-07-05 The Broad Institute, Inc. Modificación por tecnología genética y optimización de sistemas, métodos y composiciones para la manipulación de secuencias con dominios funcionales
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
CA3081054A1 (en) 2012-12-17 2014-06-26 President And Fellows Of Harvard College Rna-guided human genome engineering
BR112015021791B1 (pt) 2013-03-08 2022-08-30 Novartis Ag Compostos de lipídio catiônico e composições de lipídios e farmacêuticas
HRP20211855T1 (hr) 2013-03-12 2022-03-04 Vertex Pharmaceuticals Incorporated Inhibitori dnk-pk
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
PL3083556T3 (pl) 2013-12-19 2020-06-29 Novartis Ag Lipidy i kompozycje lipidowe dla dostarczania środków czynnych
EP4223285A3 (en) 2014-07-16 2023-11-22 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
EP3558997B1 (en) 2016-12-20 2021-01-27 AstraZeneca AB Amino-triazolopyridine compounds and their use in treating cancer
WO2019067992A1 (en) 2017-09-29 2019-04-04 Intellia Therapeutics, Inc. Formulations
US20190307795A1 (en) 2018-01-26 2019-10-10 The Board Of Trustees Of The Leland Stanford Junior University Regulatory t cells targeted with chimeric antigen receptors
CN113039174B (zh) 2018-10-02 2023-11-17 英特利亚治疗股份有限公司 可电离的胺脂质
CN113260607A (zh) 2018-12-05 2021-08-13 英特利亚治疗股份有限公司 改性的胺脂质
WO2020219876A1 (en) 2019-04-25 2020-10-29 Intellia Therapeutics, Inc. Ionizable amine lipids and lipid nanoparticles

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IL297550A (en) 2022-12-01
KR20230017783A (ko) 2023-02-06
TW202204612A (zh) 2022-02-01
WO2021222287A2 (en) 2021-11-04
JP2023524666A (ja) 2023-06-13
AU2021263745A1 (en) 2022-12-08
EP4143304A2 (en) 2023-03-08
CL2022002974A1 (es) 2023-09-15
CN116018403A (zh) 2023-04-25
US20230183753A1 (en) 2023-06-15
MX2022013403A (es) 2023-01-11
CA3181340A1 (en) 2021-11-04

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