KR102451510B1 - Pd-1 호밍 엔도뉴클레아제 변이체, 조성물 및 사용 방법 - Google Patents

Pd-1 호밍 엔도뉴클레아제 변이체, 조성물 및 사용 방법 Download PDF

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KR102451510B1
KR102451510B1 KR1020197009497A KR20197009497A KR102451510B1 KR 102451510 B1 KR102451510 B1 KR 102451510B1 KR 1020197009497 A KR1020197009497 A KR 1020197009497A KR 20197009497 A KR20197009497 A KR 20197009497A KR 102451510 B1 KR102451510 B1 KR 102451510B1
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delete delete
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cell
certain embodiments
polypeptide
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KR20190045928A (ko
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자스딥 만
조엘 가이
조던 자르주르
조이 장
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2세븐티 바이오, 인코포레이티드
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KR1020197009497A 2016-09-08 2017-09-08 Pd-1 호밍 엔도뉴클레아제 변이체, 조성물 및 사용 방법 Active KR102451510B1 (ko)

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KR1020227034173A KR102622910B1 (ko) 2016-09-08 2017-09-08 Pd-1 호밍 엔도뉴클레아제 변이체, 조성물 및 사용 방법

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US201662385079P 2016-09-08 2016-09-08
US62/385,079 2016-09-08
US201662414279P 2016-10-28 2016-10-28
US62/414,279 2016-10-28
PCT/US2017/050774 WO2018049226A1 (en) 2016-09-08 2017-09-08 Pd-1 homing endonuclease variants, compositions, and methods of use

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KR102451510B1 true KR102451510B1 (ko) 2022-10-07

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Families Citing this family (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10967298B2 (en) 2012-03-15 2021-04-06 Flodesign Sonics, Inc. Driver and control for variable impedence load
US9458450B2 (en) 2012-03-15 2016-10-04 Flodesign Sonics, Inc. Acoustophoretic separation technology using multi-dimensional standing waves
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
US9950282B2 (en) 2012-03-15 2018-04-24 Flodesign Sonics, Inc. Electronic configuration and control for acoustic standing wave generation
CN105939767B (zh) 2014-01-08 2018-04-06 弗洛设计声能学公司 具有双声电泳腔的声电泳装置
US11021699B2 (en) 2015-04-29 2021-06-01 FioDesign Sonics, Inc. Separation using angled acoustic waves
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
US11474085B2 (en) 2015-07-28 2022-10-18 Flodesign Sonics, Inc. Expanded bed affinity selection
US11459540B2 (en) 2015-07-28 2022-10-04 Flodesign Sonics, Inc. Expanded bed affinity selection
US11085035B2 (en) 2016-05-03 2021-08-10 Flodesign Sonics, Inc. Therapeutic cell washing, concentration, and separation utilizing acoustophoresis
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
SG11201901757YA (en) 2016-09-08 2019-03-28 Bluebird Bio Inc Pd-1 homing endonuclease variants, compositions, and methods of use
US11530395B2 (en) 2016-10-17 2022-12-20 2Seventy Bio, Inc. TGFBetaR2 endonuclease variants, compositions, and methods of use
EP3529347A1 (en) 2016-10-19 2019-08-28 Flodesign Sonics, Inc. Affinity cell extraction by acoustics
AU2018221730B2 (en) 2017-02-15 2024-06-20 Novo Nordisk A/S Donor repair templates multiplex genome editing
IL270776B2 (en) 2017-05-25 2024-11-01 Bluebird Bio Inc Cblb endonuclease variants, compositions, and methods of use
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
CN114900773A (zh) 2017-12-14 2022-08-12 弗洛设计声能学公司 声泳系统及其操作方法、控制声换能器及声学系统的方法
EP3765094A4 (en) 2018-03-15 2021-12-22 KSQ Therapeutics, Inc. GENE REGULATORY COMPOSITIONS AND PROCEDURES FOR IMPROVED IMMUNOTHERAPY
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
CA3095179A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
JP7577648B2 (ja) * 2018-05-14 2024-11-05 センバ インコーポレイテッド 自己免疫疾患のための遺伝子編集
WO2020072059A1 (en) * 2018-10-04 2020-04-09 Bluebird Bio, Inc. Cblb endonuclease variants, compositions, and methods of use
JP2022513750A (ja) * 2018-12-10 2022-02-09 2セブンティ バイオ インコーポレイテッド ホーミングエンドヌクレアーゼバリアント
CA3122094A1 (en) * 2018-12-10 2020-06-18 Bluebird Bio, Inc. Pdcd-1 homing endonuclease variants
WO2020186219A1 (en) * 2019-03-14 2020-09-17 The Regents Of The University Of California Pooled knock-in screening and heterologous polypeptides co-expressed under the control of endogenous loci
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
CA3150095A1 (en) * 2019-09-06 2021-03-11 Shirley O'dea Engineering of immune cells for ex vivo cell therapy applications
WO2021072163A1 (en) * 2019-10-09 2021-04-15 The Regents Of The University Of California Compositions and methods for reprogramming age-restricted non-neuronal cells
MX2022004353A (es) 2019-10-17 2022-07-19 Casete de transferencia de aav.
CN114787180A (zh) 2019-10-17 2022-07-22 斯特里迪比奥公司 用于治疗c型尼曼-匹克病的腺相关病毒载体
JP2023542614A (ja) 2020-08-19 2023-10-11 サレプタ セラピューティクス, インコーポレイテッド レット症候群の治療のためのアデノ随伴ウイルスベクター
TW202242124A (zh) * 2021-01-14 2022-11-01 美商史崔德生物公司 靶向t細胞之aav載體
EP4355875A1 (en) 2021-06-14 2024-04-24 2seventy bio, Inc. Single stranded rna purification methods
WO2023064973A1 (en) * 2021-10-19 2023-04-27 Peter Maccallum Cancer Institute Compositions and methods for immunotherapy
EP4698670A1 (en) * 2023-04-20 2026-02-25 Peter MacCallum Cancer Institute Compositions and methods for immunotherapy - ii

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2010539929A (ja) 2007-09-27 2010-12-24 サンガモ バイオサイエンシーズ, インコーポレイテッド 生物学的活性のあるヌクレアーゼの迅速なインビボ同定法
WO2014184744A1 (en) 2013-05-13 2014-11-20 Cellectis Methods for engineering highly active t cell for immunotherapy
WO2015017214A1 (en) 2013-07-29 2015-02-05 Bluebird Bio, Inc. Multipartite signaling proteins and uses thereof

Family Cites Families (57)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4751180A (en) 1985-03-28 1988-06-14 Chiron Corporation Expression using fused genes providing for protein product
US4935233A (en) 1985-12-02 1990-06-19 G. D. Searle And Company Covalently linked polypeptide cell modulators
US4873192A (en) 1987-02-17 1989-10-10 The United States Of America As Represented By The Department Of Health And Human Services Process for site specific mutagenesis without phenotypic selection
GB8918616D0 (en) 1989-08-15 1989-09-27 Univ Glasgow Herpes simplex virus type 1 mutant
AU649074B2 (en) 1990-10-12 1994-05-12 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Modified ribozymes
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
US5804413A (en) 1992-07-31 1998-09-08 University Of Pittsburgh Of The Commonwealth System Of Higher Education Herpes simplex virus strains for gene transfer
GB9415319D0 (en) 1994-07-29 1994-09-21 Medical Res Council HSV viral vector
US5846782A (en) 1995-11-28 1998-12-08 Genvec, Inc. Targeting adenovirus with use of constrained peptide motifs
US6187757B1 (en) 1995-06-07 2001-02-13 Ariad Pharmaceuticals, Inc. Regulation of biological events using novel compounds
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
EP1002119A1 (en) 1997-07-31 2000-05-24 University Of Pittsburgh Of The Commonwealth System Of Higher Education Targeted hsv vectors
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
FR2777909B1 (fr) 1998-04-24 2002-08-02 Pasteur Institut Utilisation de sequences d'adn de structure triplex pour le tranfert de sequences de nucleotides dans des cellules, vecteurs recombinants contenant ces sequences triplex
US6692736B2 (en) 2000-03-24 2004-02-17 Cell Genesys, Inc. Cell-specific adenovirus vectors comprising an internal ribosome entry site
US7465583B2 (en) 2000-06-01 2008-12-16 The University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
WO2002088346A2 (en) 2001-05-01 2002-11-07 National Research Council Of Canada A system for inducible expression in eukaryotic cells
FR2872170B1 (fr) 2004-06-25 2006-11-10 Centre Nat Rech Scient Cnrse Lentivirus non interactif et non replicatif, preparation et utilisations
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
WO2007049095A1 (en) 2005-10-25 2007-05-03 Cellectis Laglidadg homing endonuclease variants having mutations in two functional subdomains and use thereof
AU2007347328B2 (en) 2007-02-19 2013-03-07 Cellectis LAGLIDADG homing endonuclease variants having novel substrate specificity and use thereof
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
ES2422291T3 (es) 2007-10-31 2013-09-10 Prec Biosciences Inc Meganucleasas monocatenarias diseñadas racionalmente con secuencias de reconocimiento no palindrómicas
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
US9169494B2 (en) 2010-01-12 2015-10-27 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
WO2011097456A2 (en) 2010-02-05 2011-08-11 The University Of North Carolina At Chapel Hill Compositions and methods for enhanced parvovirus transduction
US20110256607A1 (en) 2010-04-16 2011-10-20 Georg Hausner Homing endonucleases
WO2011156430A2 (en) 2010-06-07 2011-12-15 Fred Hutchinson Cancer Research Center Generation and expression of engineered i-onui endonuclease and its homologues and uses thereof
SG189482A1 (en) 2010-10-27 2013-05-31 Cellectis Method for increasing the efficiency of double-strand break-induced mutagenesis
US8673557B2 (en) 2011-02-28 2014-03-18 Seattle Children's Research Institute Coupling endonucleases with end-processing enzymes drives high efficiency gene disruption
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
EP2836226B1 (en) 2012-02-24 2017-06-28 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
SG10201806573TA (en) 2012-05-25 2018-09-27 Cellectis Methods for engineering allogeneic and immunosuppressive resistant t cell for immunotherapy
US20150017136A1 (en) 2013-07-15 2015-01-15 Cellectis Methods for engineering allogeneic and highly active t cell for immunotherapy
US20140015679A1 (en) 2012-07-15 2014-01-16 Green Badge LLC Specialty Plant Moisture Sensing
JP5372297B1 (ja) 2012-12-20 2013-12-18 三菱電機株式会社 車載装置及びプログラム
CA2913872C (en) 2013-05-31 2022-01-18 Cellectis A laglidadg homing endonuclease cleaving the t-cell receptor alpha gene and uses thereof
CA2913871C (en) 2013-05-31 2021-07-13 Cellectis A laglidadg homing endonuclease cleaving the c-c chemokine receptor type-5 (ccr5) gene and uses thereof
US9108442B2 (en) 2013-08-20 2015-08-18 Ricoh Company, Ltd. Image forming apparatus
SMT202300039T1 (it) 2013-09-13 2023-03-17 Beigene Switzerland Gmbh Anticorpi anti-pd1 e loro uso come terapia e per diagnostica
US20190241910A1 (en) 2016-03-11 2019-08-08 Bluebird Bio, Inc. Genome edited immune effector cells
AU2017248259A1 (en) 2016-04-07 2018-10-25 Bluebird Bio, Inc. Chimeric antigen receptor T cell compositions
CA3031785A1 (en) 2016-07-25 2018-02-01 Bluebird Bio, Inc. Bcl11a homing endonuclease variants, compositions, and methods of use
EP3500696A4 (en) 2016-08-16 2020-04-08 Bluebird Bio, Inc. VARIANTS OF IL-10 ALPHA RECEPTOR HOMING ENDONUCLEASE, COMPOSITIONS AND METHODS OF USE
MA46018A (fr) 2016-08-19 2019-06-26 Bluebird Bio Inc Activateurs d'édition du génome
US20190262398A1 (en) 2016-08-23 2019-08-29 Bluebird Bio, Inc. Tim3 homing endonuclease variants, compositions, and methods of use
SG11201901757YA (en) 2016-09-08 2019-03-28 Bluebird Bio Inc Pd-1 homing endonuclease variants, compositions, and methods of use
SG11201903230QA (en) 2016-10-11 2019-05-30 Bluebird Bio Inc TCRa HOMING ENDONUCLEASE VARIANTS
US11530395B2 (en) 2016-10-17 2022-12-20 2Seventy Bio, Inc. TGFBetaR2 endonuclease variants, compositions, and methods of use
CN106749666B (zh) 2016-12-22 2018-12-25 福州大学 一种人源程序性死亡受体hPD-1单克隆抗体
CN106939049B (zh) 2017-04-20 2019-10-01 苏州思坦维生物技术股份有限公司 拮抗抑制人pd-1抗原与其配体结合的单克隆抗体及其制备方法与应用
IL270776B2 (en) 2017-05-25 2024-11-01 Bluebird Bio Inc Cblb endonuclease variants, compositions, and methods of use
US11779654B2 (en) 2017-10-04 2023-10-10 2Seventy Bio, Inc. PCSK9 endonuclease variants, compositions, and methods of use
WO2020072059A1 (en) 2018-10-04 2020-04-09 Bluebird Bio, Inc. Cblb endonuclease variants, compositions, and methods of use
CA3122094A1 (en) 2018-12-10 2020-06-18 Bluebird Bio, Inc. Pdcd-1 homing endonuclease variants

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2010539929A (ja) 2007-09-27 2010-12-24 サンガモ バイオサイエンシーズ, インコーポレイテッド 生物学的活性のあるヌクレアーゼの迅速なインビボ同定法
WO2014184744A1 (en) 2013-05-13 2014-11-20 Cellectis Methods for engineering highly active t cell for immunotherapy
WO2015017214A1 (en) 2013-07-29 2015-02-05 Bluebird Bio, Inc. Multipartite signaling proteins and uses thereof

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