CA3176697A1 - Vectors and methods for in vivo transduction - Google Patents

Vectors and methods for in vivo transduction Download PDF

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Publication number
CA3176697A1
CA3176697A1 CA3176697A CA3176697A CA3176697A1 CA 3176697 A1 CA3176697 A1 CA 3176697A1 CA 3176697 A CA3176697 A CA 3176697A CA 3176697 A CA3176697 A CA 3176697A CA 3176697 A1 CA3176697 A1 CA 3176697A1
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mir
cells
cell
domain
cancer
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CA3176697A
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English (en)
French (fr)
Inventor
Doughlas J. JOLLY
Derek G. Ostertag
Noriyuki Kasahara
Carlos Ibanez
Cornelia A. Bentley
Mohd Mushtaq HUSAIN
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Abintus Bio Inc
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Individual
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2803—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily
    • C07K16/2815—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily against CD8
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
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    • C07K14/7051—T-cell receptor (TcR)-CD3 complex
    • C—CHEMISTRY; METALLURGY
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    • C07K14/70578—NGF-receptor/TNF-receptor superfamily, e.g. CD27, CD30, CD40, CD95
    • C—CHEMISTRY; METALLURGY
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    • C—CHEMISTRY; METALLURGY
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    • C07K—PEPTIDES
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
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    • C07K16/2803—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily
    • C07K16/2812—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily against CD4
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
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    • C07K16/2878—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the NGF-receptor/TNF-receptor superfamily, e.g. CD27, CD30, CD40, CD95
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • C12N5/06—Animal cells or tissues; Human cells or tissues
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    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0636—T lymphocytes
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CA3176697A 2020-05-11 2021-05-11 Vectors and methods for in vivo transduction Pending CA3176697A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063023191P 2020-05-11 2020-05-11
US63/023,191 2020-05-11
US202063133224P 2020-12-31 2020-12-31
US63/133,224 2020-12-31
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Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020191241A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN116178567B (zh) * 2022-10-28 2025-01-28 四川大学华西医院 一种靶向TGFβRII的嵌合抗原受体及其用途
WO2024254346A1 (en) * 2023-06-07 2024-12-12 The Broad Institute, Inc. Engineered viral like particles (evlps) for the selective transduction of target cells
WO2025033948A1 (ko) * 2023-08-07 2025-02-13 마루테라퓨틱스 주식회사 유전자 고효율 발현이 가능한 비바이러스성 듀얼 플라스미드 벡터와 이를 발현하는 줄기세포 및 분화된 조혈계 세포
CN116814664B (zh) * 2023-08-25 2023-12-12 中国医学科学院肿瘤医院 一种扩展肿瘤识别表位的cea嵌合抗原受体t细胞的制备与应用
CN116814595B (zh) * 2023-08-30 2023-11-28 江苏申基生物科技有限公司 一种腺苷脱氨酶突变体及其固定化
WO2026149447A1 (zh) * 2025-01-07 2026-07-16 深圳市济因生物科技有限公司 一种颗粒及其应用

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5693508A (en) * 1994-11-08 1997-12-02 Chang; Lung-Ji Retroviral expression vectors containing MoMLV/CMV-IE/HIV-TAR chimeric long terminal repeats
US5681746A (en) * 1994-12-30 1997-10-28 Chiron Viagene, Inc. Retroviral delivery of full length factor VIII
US7001733B1 (en) * 1998-05-12 2006-02-21 Rigel Pharmaceuticals, Inc. Methods and compositions for screening for modulations of IgE synthesis, secretion and switch rearrangement
WO2001091802A1 (en) * 2000-05-30 2001-12-06 Baylor College Of Medicine Chimeric viral vectors for gene therapy
CN1860232A (zh) * 2003-09-30 2006-11-08 莱克康遗传公司 鉴别基因功能的方法和组合物
WO2009114725A2 (en) * 2008-03-12 2009-09-17 Children's Hospital Medical Center Mobilization of hematopoietic stem cells
US20110098346A1 (en) * 2008-05-19 2011-04-28 Agency For Science, Technology And Research Nucleic acid molecule and method of targeting gene expression to gliomas
EP2294218A2 (en) * 2008-06-02 2011-03-16 New York University Compositions and methods for diagnosis, prognosis and treatment of mesothelioma
US20110020814A1 (en) * 2009-06-05 2011-01-27 Ipierian, Inc. Methods and compositions for selection of stem cells
EP3546583A1 (en) * 2009-06-17 2019-10-02 Tocagen Inc. Producer cells for replication competent retroviral vectors
US9839696B2 (en) * 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US9642921B2 (en) * 2012-12-20 2017-05-09 Tocagen Inc. Cancer combination therapy and recombinant vectors
CA2912389C (en) * 2013-06-10 2020-01-28 The General Hospital Corporation D/B/A Massachusetts General Hospital Methods and compositions for reducing immunosupression by tumor cells
JP2016526920A (ja) * 2013-08-05 2016-09-08 トカジェン インコーポレーテッド 最適化ａ−バルジを有する組換えベクター
US10383929B2 (en) * 2014-12-12 2019-08-20 Bluebird Bio, Inc. BCMA chimeric antigen receptors
AU2016317936A1 (en) * 2015-09-04 2018-03-08 Tocagen Inc. Recombinant vectors comprising 2A peptide
CN108884460B (zh) * 2016-03-19 2023-04-28 埃克苏马生物技术公司 淋巴细胞转导及其扩增调节的方法与组合物
US11111505B2 (en) * 2016-03-19 2021-09-07 Exuma Biotech, Corp. Methods and compositions for transducing lymphocytes and regulating the activity thereof
CA3030003A1 (en) * 2016-07-08 2018-01-11 F1 Oncology, Inc. Methods and compositions for transducing lymphocytes and regulating the activity thereof
DK3612624T3 (da) * 2017-04-21 2024-12-09 Ospedale San Raffaele Srl Genterapi
US20180327857A1 (en) * 2017-05-09 2018-11-15 Shanghai Realgen Biotech Co., Ltd. Diagnostic biomarker and diagnostic method
WO2019025800A1 (en) * 2017-08-02 2019-02-07 Autolus Limited CELLS EXPRESSING A CHIMERIC ANTIGENIC RECEPTOR OR A MANIPULATED TCR AND COMPRISING A SELECTIVELY EXPRESSED SELECTIVE NUCLEOTIDE SEQUENCE
KR20210063348A (ko) * 2018-08-28 2021-06-01 이뮤노테크 바이오팜 씨오., 엘티디. 개선된 치료용 t 세포

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