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

Métodos de distribuição de célula in vitro

Info

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
Authority
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

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14Blood; Artificial blood
    • A61K35/17Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/1774Immunoglobulin superfamily (e.g. CD2, CD4, CD8, ICAM molecules, B7 molecules, Fc-receptors, MHC-molecules)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/46Cellular immunotherapy
    • A61K39/461Cellular immunotherapy characterised by the cell type used
    • A61K39/4611T-cells, e.g. tumor infiltrating lymphocytes [TIL], lymphokine-activated killer cells [LAK] or regulatory T cells [Treg]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/46Cellular immunotherapy
    • A61K39/463Cellular immunotherapy characterised by recombinant expression
    • A61K39/4632T-cell receptors [TCR]; antibody T-cell receptor constructs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/46Cellular immunotherapy
    • A61K39/464Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643Vertebrate antigens
    • A61K39/4644Cancer antigens
    • A61K39/464452Transcription factors, e.g. SOX or c-MYC
    • A61K39/464453Wilms tumor 1 [WT1]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/46Cellular immunotherapy
    • A61K39/464Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643Vertebrate antigens
    • A61K39/4644Cancer antigens
    • A61K39/46449Melanoma antigens
    • A61K39/464491Melan-A/MART
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00Indexing codes associated with cellular immunotherapy of group A61K39/46
    • A61K2239/31Indexing codes associated with cellular immunotherapy of group A61K39/46 characterized by the route of administration
    • 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
    • A61K2239/38Indexing codes associated with cellular immunotherapy of group A61K39/46 characterised by the dose, timing or administration schedule
    • 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
    • A61K2239/46Indexing codes associated with cellular immunotherapy of group A61K39/46 characterised by the cancer treated
    • A61K2239/48Blood cells, e.g. leukemia or lymphoma
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00Genetically modified cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Mycology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Oncology (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

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)

Publication Number Publication Date
BR112022021676A2 true BR112022021676A2 (pt) 2023-01-31

Family

ID=76270024

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022021676A BR112022021676A2 (pt) 2020-04-28 2021-04-27 Métodos de distribuição de célula in vitro

Country Status (13)

Country Link
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 (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117098541A (zh) 2020-11-25 2023-11-21 阿卡格拉医药公司 用于递送核酸的脂质纳米粒及相关使用方法
CR20230320A (es) * 2020-12-23 2023-10-23 Intellia Therapeutics Inc Composiciones y métodos para reducir los hla-a en una célula
WO2022170193A2 (en) 2021-02-08 2022-08-11 Intellia Therapeutics, Inc. T-cell immunoglobulin and mucin domain 3 (tim3) 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
JP2024505678A (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 免疫療法のためのリンパ球活性化遺伝子3(lag3)組成物及び方法
TW202317764A (zh) 2021-06-10 2023-05-01 美商英特利亞醫療公司 用於基因編輯之包含內部連接子之經修飾引導rna
CN117940138A (zh) * 2021-07-01 2024-04-26 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
TW202408595A (zh) * 2022-06-16 2024-03-01 美商英特利亞醫療公司 用於對細胞進行遺傳修飾之方法及組合物
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
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

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
ATE515510T1 (de) 1991-12-24 2011-07-15 Isis Pharmaceuticals Inc Durch dna-abschnitte unterbrochene modifizierte oligonukleotide
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
EP2526199A4 (en) 2010-01-22 2013-08-07 Scripps Research Inst METHODS FOR PRODUCING ZINC FINGER NUCLEASES HAVING MODIFIED 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
WO2014093694A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Crispr-cas nickase systems, methods and compositions for sequence manipulation in eukaryotes
US8993233B2 (en) 2012-12-12 2015-03-31 The Broad Institute Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
EP3553174A1 (en) 2012-12-17 2019-10-16 President and Fellows of Harvard College Rna-guided human genome engineering
EA030650B1 (ru) 2013-03-08 2018-09-28 Новартис Аг Липиды и липидные композиции для доставки активных агентов
KR102216284B1 (ko) 2013-03-12 2021-02-18 버텍스 파마슈티칼스 인코포레이티드 Dna-pk 억제제
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
US10059655B2 (en) 2013-12-19 2018-08-28 Novartis Ag Lipids and lipid compositions for the delivery of active agents
JP6778175B2 (ja) 2014-07-16 2020-10-28 ノバルティス アーゲー 脂質ナノ粒子ホスト中に核酸を封入する方法
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
JP6883653B2 (ja) 2016-12-20 2021-06-09 アストラゼネカ・アクチエボラーグAstrazeneca Aktiebolag アミノ−トリアゾロピリジン化合物および癌の治療におけるその使用
PT3688162T (pt) 2017-09-29 2024-04-23 Intellia Therapeutics Inc Formulações
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
SG11202102921WA (en) 2018-10-02 2021-04-29 Intellia Therapeutics Inc Ionizable amine lipids
EA202191559A1 (ru) 2018-12-05 2021-08-24 Интеллиа Терапьютикс, Инк. Модифицированные аминовые липиды
CA3137956A1 (en) 2019-04-25 2020-10-29 Intellia Therapeutics, Inc. Ionizable amine lipids and lipid nanoparticles

Also Published As

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

Similar Documents

Publication Publication Date Title
BR112022021676A2 (pt) Métodos de distribuição de célula in vitro
MX2018013445A (es) Celulas diseñadas geneticamente y metodos para obtener las mismas.
MX2020004608A (es) Metodos, composiciones y componentes para edicion crispr-cas9 de tgfbr2 en celulas t para inmunoterapia.
CO2019013001A2 (es) Proteínas de anticuerpo injertadas con citocina y métodos de uso en el tratamiento del cáncer
MX2020013293A (es) Guías modificadas de arn para edición génica.
BR112015030946A8 (pt) composição compreendendo uma célula ß pancreática não-nativa, método de diferenciação de células progenitoras em células ß pancreáticas in vitro e uso de uma célula ß pancreática não-nativa
MX2021004187A (es) Métodos y composiciones para editar ácidos ribonucleicos.
CR20210572A (es) Métodos y composiciones para editar ácido ribonucleicos (arn)
EP4249074A3 (en) Genomic engineering of pluripotent cells
WO2017115128A3 (en) Vector-free delivery of gene editing proteins and compositions to cells and tissues
BR112018005937A2 (pt) conjugados de oligonucleotídeo de afinidade e usos destes
CO6501191A2 (es) Medio de cultivo celular para expresión de la proteina adamts
PH12016502162A1 (en) Multiplex gene editing in swine
PE20110358A1 (es) Metodos y composiciones para el tratamiento de defectos oseos con poblaciones de celulas placentarias
BR112012026379A2 (pt) métodos para gerar proteína endogenamente marcada
EP4324962A3 (en) Methods and compositions for deconvoluting partition barcodes
MX2022006950A (es) Arn guía modificados para edición de genes.
BR112021019349A2 (pt) Células-tronco preparadas altamente funcionais
BR112021023554A2 (pt) Composições e métodos para cultura de células vegetais
EP4142754A4 (en) METHOD FOR THE IN VITRO PRODUCTION OF THYMIC CELLS
BR112023018948A2 (pt) Edição multiplex com enzimas cas
EP3984596A4 (en) POPULATION OF CELLS COMPRISING MESENCHYMATOUS CELLS, PHARMACEUTICAL COMPOSITION COMPRISING THEM AND METHOD FOR PRODUCTION THEREOF
MX2021002702A (es) Metodos para expandir celulas t del receptor de antigeno quimerico especificas de antigenos, composiciones y usos relacionados con las mismas.
MX2020011470A (es) Metodos de terapia genica.
UY32732A (es) Medios para cultivo de células de mamíferos que comprenden sobrenadante del fraccionamiento de cohn y uso de los mismos