BR112019015355A2 - modificação genética de células b - Google Patents

modificação genética de células b Download PDF

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Publication number
BR112019015355A2
BR112019015355A2 BR112019015355A BR112019015355A BR112019015355A2 BR 112019015355 A2 BR112019015355 A2 BR 112019015355A2 BR 112019015355 A BR112019015355 A BR 112019015355A BR 112019015355 A BR112019015355 A BR 112019015355A BR 112019015355 A2 BR112019015355 A2 BR 112019015355A2
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BR
Brazil
Prior art keywords
cells
cell
protein
genetically modified
fact
Prior art date
Application number
BR112019015355A
Other languages
English (en)
Portuguese (pt)
Inventor
E Riley Brigit
C Holmes Michael
AMORA Rainier
Original Assignee
Sangamo 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 Sangamo Therapeutics Inc filed Critical Sangamo Therapeutics Inc
Publication of BR112019015355A2 publication Critical patent/BR112019015355A2/pt

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    • 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
    • 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
    • A61K35/28Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • 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
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/48Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482Serine endopeptidases (3.4.21)
    • A61K38/4846Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/10Cellular immunotherapy characterised by the cell type used
    • A61K40/13B-cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/20Cellular immunotherapy characterised by the effect or the function of the cells
    • A61K40/22Immunosuppressive or immunotolerising
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/20Cellular immunotherapy characterised by the effect or the function of the cells
    • A61K40/24Antigen-presenting cells [APC]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/40Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41Vertebrate antigens
    • A61K40/416Antigens related to auto-immune diseases; Preparations to induce self-tolerance
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • 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
    • 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
    • C12N5/0635B 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
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10Cells modified by introduction of foreign genetic material
    • C12N5/12Fused cells, e.g. hybridomas
    • C12N5/16Animal cells
    • C12N5/163Animal cells one of the fusion partners being a B or a T lymphocyte
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Immunology (AREA)
  • Organic Chemistry (AREA)
  • Cell Biology (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Hematology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Developmental Biology & Embryology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Mycology (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
BR112019015355A 2017-01-26 2018-01-25 modificação genética de células b BR112019015355A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762450917P 2017-01-26 2017-01-26
PCT/US2018/015180 WO2018140573A1 (en) 2017-01-26 2018-01-25 B-cell engineering

Publications (1)

Publication Number Publication Date
BR112019015355A2 true BR112019015355A2 (pt) 2020-05-19

Family

ID=62979713

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112019015355A BR112019015355A2 (pt) 2017-01-26 2018-01-25 modificação genética de células b

Country Status (11)

Country Link
US (1) US20190352614A1 (enExample)
EP (1) EP3573464A4 (enExample)
JP (2) JP2020505044A (enExample)
KR (1) KR20190111063A (enExample)
CN (1) CN110536963A (enExample)
AU (1) AU2018212652B2 (enExample)
BR (1) BR112019015355A2 (enExample)
CA (1) CA3051113A1 (enExample)
IL (1) IL268110B2 (enExample)
MX (1) MX2019008844A (enExample)
WO (1) WO2018140573A1 (enExample)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
CA2935960C (en) 2014-01-08 2023-01-10 Bart Lipkens Acoustophoresis device with dual acoustophoretic chamber
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
EP3573464A4 (en) * 2017-01-26 2020-12-30 Sangamo Therapeutics, Inc. B CELL ENGINEERING
AU2018235957B2 (en) * 2017-03-16 2024-02-01 Seattle Children's Hospital (dba Seattle Children's Research Institute) Engraftable cell-based immunotherapy for long-term delivery of therapeutic proteins
KR20220066413A (ko) 2017-12-14 2022-05-24 프로디자인 소닉스, 인크. 음향 트랜스듀서 구동기 및 제어기
BR112021007301A2 (pt) 2018-10-18 2021-07-27 Intellia Therapeutics, Inc. composições e métodos para expressar fator ix
WO2021085504A1 (ja) * 2019-11-01 2021-05-06 京都府公立大学法人 B細胞抗体受容体、及びその利用
EP4127188A4 (en) 2020-03-31 2024-08-21 Walking Fish Therapeutics MODIFIED B CELLS AND METHODS OF USE THEREOF
WO2023192936A2 (en) * 2022-03-30 2023-10-05 Fred Hutchinson Cancer Center Systems and methods to produce b cells that express selected antibodies and gene products
WO2024151683A2 (en) * 2023-01-09 2024-07-18 Walking Fish Therapeutics, Inc. HUMAN B CELL ENGINEERED TO EXPRESS IgA AND IgM ANTIBODIES FOR THERAPEUTIC USE
WO2024196669A2 (en) * 2023-03-17 2024-09-26 Walking Fish Therapeutics, Inc. Methods for in vivo editing of b cells
WO2024220447A2 (en) * 2023-04-17 2024-10-24 Be Biopharma, Inc. Engineered cell preparations for treatment of niemann pick b disease

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003901511A0 (en) * 2003-03-28 2003-04-17 Bionomics Limited Nucleic acid molecules associated with angiogenesis II
DK1804839T3 (da) * 2004-09-22 2012-04-10 St Jude Childrens Res Hospital Forbedret ekspression af faktor ix i genterapivektorer
US9834787B2 (en) * 2009-04-09 2017-12-05 Sangamo Therapeutics, Inc. Targeted integration into stem cells
AU2014265331B2 (en) * 2013-05-15 2019-12-05 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
US9757420B2 (en) * 2014-07-25 2017-09-12 Sangamo Therapeutics, Inc. Gene editing for HIV gene therapy
CN106795488B (zh) * 2014-09-16 2021-03-30 桑格摩治疗股份有限公司 用于造血干细胞中核酸酶介导的基因组工程化和校正的方法和组合物
EP4177336A1 (en) * 2014-12-19 2023-05-10 Immusoft Corporation B cells for in vivo delivery of therapeutic agents
EP4335918A3 (en) * 2015-04-03 2024-04-17 Dana-Farber Cancer Institute, Inc. Composition and methods of genome editing of b-cells
HUE066300T2 (hu) * 2015-06-24 2024-07-28 Janssen Biotech Inc Immunmoduláció és szolid tumorok kezelése CD38-hoz specifikusan kötõdõ antitestekkel
WO2017011519A1 (en) * 2015-07-13 2017-01-19 Sangamo Biosciences, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
EP3402533B1 (en) * 2016-01-15 2021-08-04 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
EP3573464A4 (en) * 2017-01-26 2020-12-30 Sangamo Therapeutics, Inc. B CELL ENGINEERING
CN111533799A (zh) * 2017-04-10 2020-08-14 伊玛提克斯生物技术有限公司 用于白血病和其他癌症免疫治疗的肽和肽组合物
WO2018232296A1 (en) * 2017-06-16 2018-12-20 Sangamo Therapeutics, Inc. Targeted disruption of t cell and/or hla receptors
MX2020001594A (es) * 2017-08-08 2020-12-09 Sangamo Therapeutics Inc Direccionamiento celular mediado por receptor de antigeno quimerico.
CN111565730B (zh) * 2017-11-09 2024-09-17 桑格摩生物治疗股份有限公司 细胞因子诱导型含sh2蛋白(cish)基因的遗传修饰
US20230158070A1 (en) * 2018-01-30 2023-05-25 Cellectis Combination comprising allogeneic immune cells deficient for an antigen present on both t-cells and pathological cells and therapeutic antibody against said antigen
AU2019327913A1 (en) * 2018-08-29 2021-03-18 Nanjing Legend Biotech Co., Ltd. Anti-mesothelin chimeric antigen receptor (CAR) constructs and uses thereof
CN113748126A (zh) * 2018-11-30 2021-12-03 因提玛生物科学公司 鉴定免疫调节基因的方法

Also Published As

Publication number Publication date
US20190352614A1 (en) 2019-11-21
JP2020505044A (ja) 2020-02-20
IL268110B2 (en) 2024-01-01
IL268110B1 (en) 2023-09-01
KR20190111063A (ko) 2019-10-01
EP3573464A4 (en) 2020-12-30
JP2022191524A (ja) 2022-12-27
WO2018140573A1 (en) 2018-08-02
AU2018212652B2 (en) 2024-03-28
CA3051113A1 (en) 2018-08-02
EP3573464A1 (en) 2019-12-04
RU2019126606A3 (enExample) 2021-06-11
IL268110A (en) 2019-09-26
AU2018212652A1 (en) 2019-08-01
CN110536963A (zh) 2019-12-03
MX2019008844A (es) 2019-09-10
RU2019126606A (ru) 2021-02-26

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B15K Others concerning applications: alteration of classification

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Ipc: A61K 35/17 (2015.01), A61K 35/00 (2006.01), A61K 3

B15K Others concerning applications: alteration of classification

Free format text: AS CLASSIFICACOES ANTERIORES ERAM: A61K 35/17 , A61K 35/00 , A61K 35/28 , A61K 38/00 , A61K 38/37 , A61P 7/02

Ipc: C12N 15/85 (2006.01), A61K 35/17 (2015.01), C12N 1

B350 Update of information on the portal [chapter 15.35 patent gazette]
B08F Application dismissed because of non-payment of annual fees [chapter 8.6 patent gazette]

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B08K Patent lapsed as no evidence of payment of the annual fee has been furnished to inpi [chapter 8.11 patent gazette]

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