JP2019532672A - 多孔質膜系巨大分子送達システム - Google Patents

多孔質膜系巨大分子送達システム Download PDF

Info

Publication number
JP2019532672A
JP2019532672A JP2019538110A JP2019538110A JP2019532672A JP 2019532672 A JP2019532672 A JP 2019532672A JP 2019538110 A JP2019538110 A JP 2019538110A JP 2019538110 A JP2019538110 A JP 2019538110A JP 2019532672 A JP2019532672 A JP 2019532672A
Authority
JP
Japan
Prior art keywords
cells
cell
microns
pores
membrane
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
JP2019538110A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019532672A5 (enExample
Inventor
ファチン,ファビオ
フィオリノ,マイケル
リー,キャメロン
ヤン,イ
イェン,ジョナサン
Original Assignee
ノバルティス アーゲー
ノバルティス アーゲー
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 ノバルティス アーゲー, ノバルティス アーゲー filed Critical ノバルティス アーゲー
Publication of JP2019532672A publication Critical patent/JP2019532672A/ja
Publication of JP2019532672A5 publication Critical patent/JP2019532672A5/ja
Pending legal-status Critical Current

Links

Images

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
    • 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
    • C12N15/87Introduction 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
    • 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
    • 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 [CRISPR]
    • 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)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Apparatus Associated With Microorganisms And Enzymes (AREA)
JP2019538110A 2016-09-28 2017-09-28 多孔質膜系巨大分子送達システム Pending JP2019532672A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662401053P 2016-09-28 2016-09-28
US62/401,053 2016-09-28
PCT/US2017/054110 WO2018064387A1 (en) 2016-09-28 2017-09-28 Porous membrane-based macromolecule delivery system

Publications (2)

Publication Number Publication Date
JP2019532672A true JP2019532672A (ja) 2019-11-14
JP2019532672A5 JP2019532672A5 (enExample) 2020-11-12

Family

ID=60183103

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2019538110A Pending JP2019532672A (ja) 2016-09-28 2017-09-28 多孔質膜系巨大分子送達システム

Country Status (5)

Country Link
US (1) US20190203230A1 (enExample)
EP (1) EP3519577A1 (enExample)
JP (1) JP2019532672A (enExample)
CN (1) CN109844126A (enExample)
WO (1) WO2018064387A1 (enExample)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9017991B2 (en) 2009-03-13 2015-04-28 Tufts University Methods tip assemblies and kits for introducing material into cells
EP3294896A1 (en) 2015-05-11 2018-03-21 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
WO2016201047A1 (en) 2015-06-09 2016-12-15 Editas Medicine, Inc. Crispr/cas-related methods and compositions for improving transplantation
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
CN110272810A (zh) * 2019-07-01 2019-09-24 广州世赛生物科技有限公司 外源物质递送至真核细胞内的装置和方法及其应用
CA3218511A1 (en) * 2021-05-10 2022-11-17 Sqz Biotechnologies Company Methods for delivering genome editing molecules to the nucleus or cytosol of a cell and uses thereof
WO2024073159A1 (en) * 2022-09-30 2024-04-04 Xellar, Inc. Techniques and systems for creating spatially controlled fluidic flows in surface functionalized microfluidic devices
WO2024238578A1 (en) * 2023-05-15 2024-11-21 Portal Biotechnologies, Inc. Cellular delivery of gene editing complexes and combinations thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016020616A1 (fr) * 2014-08-05 2016-02-11 Screencell Procédé pour le dépistage de la drépanocytose et kit pour sa mise en oeuvre
WO2017041050A1 (en) * 2015-09-04 2017-03-09 Sqz Biotechnologies Company Intracellular delivery of biomolecules mediated by a surface with pores

Family Cites Families (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7687267B2 (en) * 2006-09-30 2010-03-30 Rational Biotechnology Inc. High-throughput cell transfection device and methods of using thereof
US20110213288A1 (en) * 2007-04-23 2011-09-01 The Board Of Regents, The University Of Texas System Device And Method For Transfecting Cells For Therapeutic Uses
PE20100362A1 (es) 2008-10-30 2010-05-27 Irm Llc Derivados de purina que expanden las celulas madre hematopoyeticas
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
CN102656260A (zh) * 2009-10-19 2012-09-05 瑞生生物技术有限公司 用于诱导自调节细胞电穿孔的方法、装置和设备
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
GB2481983A (en) 2010-07-12 2012-01-18 Hart Fenton & Co Ltd A ship including a gas tank room
CA2805442C (en) 2010-07-21 2020-05-12 Sangamo Biosciences, Inc. Methods and compositions for modification of an hla locus
SI2807165T1 (sl) 2012-01-27 2019-08-30 Universite De Montreal Pirimido(4,5-B)indol derivati in njihova uporaba pri ekspanziji hematopoetskih matičnih celic
FI3597749T3 (fi) 2012-05-25 2023-10-09 Univ California Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten
ES2576126T3 (es) 2012-12-12 2016-07-05 The Broad Institute, Inc. Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP4234696A3 (en) 2012-12-12 2023-09-06 The Broad Institute Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
SG11201505896YA (en) 2013-02-20 2015-09-29 Novartis Ag Treatment of cancer using humanized anti-egfrviii chimeric antigen receptor
EP3744736A1 (en) 2013-02-20 2020-12-02 Novartis AG Effective targeting of primary human leukemia using anti-cd123 chimeric antigen receptor engineered t cells
UY35468A (es) 2013-03-16 2014-10-31 Novartis Ag Tratamiento de cáncer utilizando un receptor quimérico de antígeno anti-cd19
US11685935B2 (en) 2013-05-29 2023-06-27 Cellectis Compact scaffold of Cas9 in the type II CRISPR system
US20160237455A1 (en) 2013-09-27 2016-08-18 Editas Medicine, Inc. Crispr-related methods and compositions
CA3225453A1 (en) 2013-12-19 2015-06-25 Novartis Ag Human mesothelin chimeric antigen receptors and uses thereof
WO2015127428A1 (en) * 2014-02-24 2015-08-27 Massachusetts Institute Of Technology Methods for in vivo genome editing
WO2015142675A2 (en) 2014-03-15 2015-09-24 Novartis Ag Treatment of cancer using chimeric antigen receptor
CN106460011A (zh) * 2014-03-28 2017-02-22 加利福尼亚大学董事会 大运输物向多孔基质上的细胞中的高效递送
WO2015191693A2 (en) * 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
WO2016014565A2 (en) 2014-07-21 2016-01-28 Novartis Ag Treatment of cancer using humanized anti-bcma chimeric antigen receptor
CN107109420A (zh) 2014-07-21 2017-08-29 诺华股份有限公司 使用cll-1嵌合抗原受体的癌症治疗
BR112017001242A2 (pt) 2014-07-21 2017-12-05 Novartis Ag tratamento de câncer usando um receptor antigênico quimérico a cd33
MY189028A (en) 2014-08-19 2022-01-20 Novartis Ag Anti-cd123 chimeric antigen receptor (car) for use in cancer treatment
JP7523203B2 (ja) * 2014-10-31 2024-07-26 マサチューセッツ インスティテュート オブ テクノロジー 生体分子の免疫細胞への送達
WO2016094880A1 (en) * 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
EP3245294A4 (en) * 2015-01-12 2018-05-30 Massachusetts Institute of Technology Gene editing through microfluidic delivery
AU2016214301B2 (en) * 2015-02-06 2022-05-19 Cellectis Primary hematopoietic cells genetically engineered by slow release of nucleic acids using nanoparticles
IL254817B2 (en) 2015-04-08 2023-12-01 Novartis Ag Cd20 therapies, cd22 therapies, and combination therapies with a cd19 chimeric antigen receptor (car) - expressing cell
JP2018536436A (ja) 2015-12-04 2018-12-13 ノバルティス アーゲー 免疫腫瘍学のための組成物および方法
EA201891532A1 (ru) 2015-12-28 2019-01-31 Новартис Аг Композиции и способы лечения гемоглобинопатий

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016020616A1 (fr) * 2014-08-05 2016-02-11 Screencell Procédé pour le dépistage de la drépanocytose et kit pour sa mise en oeuvre
WO2017041050A1 (en) * 2015-09-04 2017-03-09 Sqz Biotechnologies Company Intracellular delivery of biomolecules mediated by a surface with pores

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
BIOTECHNOLOGY AND BIOENGINEERING, vol. 65, no. 3, JPN6022019560, 5 November 1999 (1999-11-05), pages 341 - 346, ISSN: 0004947385 *

Also Published As

Publication number Publication date
US20190203230A1 (en) 2019-07-04
EP3519577A1 (en) 2019-08-07
CN109844126A (zh) 2019-06-04
WO2018064387A1 (en) 2018-04-05

Similar Documents

Publication Publication Date Title
JP2019532672A (ja) 多孔質膜系巨大分子送達システム
KR102752985B1 (ko) Vcar 조성물 및 사용 방법
JP7292213B2 (ja) Crispr/cpf1を用いる、t細胞における遺伝子編集のための組成物および方法
CN107980059B (zh) 用于修饰基因组dna的方法和组合物
Yen et al. TRIAMF: A new method for delivery of Cas9 ribonucleoprotein complex to human hematopoietic stem cells
JP7428712B2 (ja) 低/最小操作による遺伝子改変細胞の製造
EP3700924A1 (en) Targeted replacement of endogenous t cell receptors
US20200208111A1 (en) Genome-edited nk cell and methods of making and using
WO2016115179A1 (en) Gene editing through microfluidic delivery
JP2016513974A (ja) 造血幹細胞系列を初期化するための組成物および方法
KR20220097985A (ko) Car-t 세포의 제조 방법
CN114729325A (zh) 在培养物中具有改善的持久性的基因工程化t细胞
EP4419672A2 (en) Compositions and methods for targeting, editing, or modifying genes
TW202016297A (zh) 抗藥性免疫細胞及使用其之方法
EP4452304A1 (en) Genetically engineered t cells with disrupted casitas b-lineage lymphoma proto-oncogene-b (cblb) and uses thereof
JP2024534720A (ja) 遺伝子改変細胞を作製するための方法
JP2021521850A (ja) X連鎖性高IgM症候群のゲノム編集治療
EP4460370A1 (en) Modified hematopoietic stem cells and uses thereof
EP4574979A1 (en) Engineered immune cell and preparation method therefor
CN118813625B (zh) 高度靶向人类HLA-A基因的sgRNA及应用
JP2024532305A (ja) 細胞を修飾する方法
WO2023086595A1 (en) Biomarkers of megakaryocyte-derived extracellular vesicles
WO2025221789A1 (en) Compositions and methods for use in car/tcr cell therapies
AU2022278634A1 (en) Gene therapy for the treatment of severe combined immunodeficiency (scid) related to rag1
JP2013116898A (ja) 幹細胞を操作する方法

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200918

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20200918

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20211005

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20211215

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220125

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220524

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20221220