CA3004349A1 - Methods and compositions for engineering immunity - Google Patents

Methods and compositions for engineering immunity Download PDF

Info

Publication number
CA3004349A1
CA3004349A1 CA3004349A CA3004349A CA3004349A1 CA 3004349 A1 CA3004349 A1 CA 3004349A1 CA 3004349 A CA3004349 A CA 3004349A CA 3004349 A CA3004349 A CA 3004349A CA 3004349 A1 CA3004349 A1 CA 3004349A1
Authority
CA
Canada
Prior art keywords
antibody
cell
transgene
dna
protein
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
CA3004349A
Other languages
English (en)
French (fr)
Inventor
Michael C. Holmes
Brigit E. RILEY
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sangamo Therapeutics Inc
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 CA3004349A1 publication Critical patent/CA3004349A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/30Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants from tumour cells
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/32Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against translation products of oncogenes
    • 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
    • C12N15/62DNA sequences coding for fusion proteins
    • 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
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/505Medicinal preparations containing antigens or antibodies comprising antibodies
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/60Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622Single chain antibody (scFv)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/60Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/626Diabody or triabody
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/31Fusion polypeptide fusions, other than Fc, for prolonged plasma life, e.g. albumin
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Psychology (AREA)
  • Oncology (AREA)
  • Hematology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3004349A 2015-11-23 2016-11-21 Methods and compositions for engineering immunity Pending CA3004349A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562258864P 2015-11-23 2015-11-23
US62/258,864 2015-11-23
US201662405521P 2016-10-07 2016-10-07
US62/405,521 2016-10-07
PCT/US2016/063146 WO2017091512A1 (en) 2015-11-23 2016-11-21 Methods and compositions for engineering immunity

Publications (1)

Publication Number Publication Date
CA3004349A1 true CA3004349A1 (en) 2017-06-01

Family

ID=58763566

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3004349A Pending CA3004349A1 (en) 2015-11-23 2016-11-21 Methods and compositions for engineering immunity

Country Status (6)

Country Link
US (2) US10639383B2 (enExample)
EP (1) EP3380622A4 (enExample)
JP (2) JP6976249B2 (enExample)
AU (1) AU2016361350B2 (enExample)
CA (1) CA3004349A1 (enExample)
WO (1) WO2017091512A1 (enExample)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11512287B2 (en) 2017-06-16 2022-11-29 Sangamo Therapeutics, Inc. Targeted disruption of T cell and/or HLA receptors
JP7061750B2 (ja) * 2017-12-01 2022-05-02 学校法人順天堂 抗ヒトil-26抗体
GB201720975D0 (en) 2017-12-15 2018-01-31 Ucb Biopharma Sprl Anti-alpha synuclein antibodies
GB201720970D0 (en) 2017-12-15 2018-01-31 Ucb Biopharma Sprl Antibodies
JP7520826B2 (ja) * 2018-10-17 2024-07-23 クリスパー・セラピューティクス・アクチェンゲゼルシャフト 導入遺伝子を送達するための組成物および方法
BR112021007301A2 (pt) * 2018-10-18 2021-07-27 Intellia Therapeutics, Inc. composições e métodos para expressar fator ix
EP3867381A2 (en) * 2018-10-18 2021-08-25 Intellia Therapeutics, Inc. Compositions and methods for transgene expression from an albumin locus
WO2020097561A1 (en) 2018-11-08 2020-05-14 Prothena Biosciences Limited Antibodies recognizing tau
AU2020231366A1 (en) 2019-03-03 2021-08-12 Prothena Biosciences Limited Antibodies recognizing tau
CN118064502A (zh) * 2019-04-03 2024-05-24 瑞泽恩制药公司 用于将抗体编码序列插入到安全港基因座中的方法和组合物
SG11202111256XA (en) 2019-06-07 2021-11-29 Regeneron Pharma Non-human animals comprising a humanized albumin locus
CA3183994A1 (en) 2020-06-24 2021-12-30 Philip James Dolan Iii Antibodies recognizing sortilin
WO2023081756A1 (en) 2021-11-03 2023-05-11 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Precise genome editing using retrons
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
WO2024044723A1 (en) 2022-08-25 2024-02-29 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
WO2025049959A2 (en) 2023-09-01 2025-03-06 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
TW202530252A (zh) 2023-09-15 2025-08-01 愛爾蘭商普羅希那生物科學有限公司 降低抗體免疫原性及改良抗體穩定性之組成物及方法
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025188676A1 (en) * 2024-03-04 2025-09-12 The Board Of Trustees Of The Leland Stanford Junior University Genetic engineering of cells for secretion of therapeutic antibodies

Family Cites Families (74)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US1013219A (en) 1910-04-26 1912-01-02 Rex Aerial Navigating And Construction Company Aerial vehicle.
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
AU704601B2 (en) 1994-01-18 1999-04-29 Scripps Research Institute, The Zinc finger protein derivatives and methods therefor
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
EP0781331B1 (en) 1994-08-20 2008-09-03 Gendaq Limited Improvements in or relating to binding proteins for recognition of dna
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
WO1999045132A1 (en) 1998-03-02 1999-09-10 Massachusetts Institute Of Technology Poly zinc finger proteins with improved linkers
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
AU776576B2 (en) 1999-12-06 2004-09-16 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
JP5047437B2 (ja) 2000-02-08 2012-10-10 サンガモ バイオサイエンシーズ, インコーポレイテッド 薬物の発見のための細胞
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
WO2001088197A2 (en) 2000-05-16 2001-11-22 Massachusetts Institute Of Technology Methods and compositions for interaction trap assays
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
JP2005500061A (ja) 2001-08-20 2005-01-06 ザ スクリップス リサーチ インスティテュート Cnnについての亜鉛フィンガー結合ドメイン
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
WO2003087341A2 (en) 2002-01-23 2003-10-23 The University Of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
US20030232410A1 (en) 2002-03-21 2003-12-18 Monika Liljedahl Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
US7074596B2 (en) 2002-03-25 2006-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse mRNA cap analogues
EP1519958B1 (en) 2002-06-14 2014-10-15 Immunomedics, Inc. Humanized monoclonal antibody hpam4
JP2006502748A (ja) 2002-09-05 2006-01-26 カリフォルニア インスティテュート オブ テクノロジー 遺伝子ターゲッティングを誘発するキメラヌクレアーゼの使用方法
WO2004055055A1 (en) 2002-12-13 2004-07-01 Licentia Ltd The transmembrane protein amigo and uses thereof
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
AU2005287278B2 (en) 2004-09-16 2011-08-04 Sangamo Biosciences, Inc. Compositions and methods for protein production
EP1877583A2 (en) 2005-05-05 2008-01-16 Arizona Board of Regents on behalf of the Unversity of Arizona Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences
EP2213731B1 (en) 2006-05-25 2013-12-04 Sangamo BioSciences, Inc. Variant foki cleavage half-domains
EP2447279B1 (en) 2006-05-25 2014-04-09 Sangamo BioSciences, Inc. Methods and compositions for gene inactivation
US8110379B2 (en) 2007-04-26 2012-02-07 Sangamo Biosciences, Inc. Targeted integration into the PPP1R12C locus
SI2167523T1 (sl) 2007-06-19 2014-09-30 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Sinteza in uporaba anti-reverznih fosforotioatnih analogov kape obveščevalne RNA
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
WO2009131632A1 (en) 2008-04-14 2009-10-29 Sangamo Biosciences, Inc. Linear donor constructs for targeted integration
KR20160015400A (ko) 2008-08-22 2016-02-12 상가모 바이오사이언스 인코포레이티드 표적화된 단일가닥 분할 및 표적화된 통합을 위한 방법 및 조성물
CN102625655B (zh) 2008-12-04 2016-07-06 桑格摩生物科学股份有限公司 使用锌指核酸酶在大鼠中进行基因组编辑
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
DK2459231T3 (en) 2009-07-31 2016-09-05 Ethris Gmbh RNA with a combination of unmodified and modified nucleotides for protein expression
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
ES2751916T3 (es) 2010-02-08 2020-04-02 Sangamo Therapeutics Inc Semidominios de escisión genomanipulados
WO2011100058A1 (en) 2010-02-09 2011-08-18 Sangamo Biosciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US9567573B2 (en) 2010-04-26 2017-02-14 Sangamo Biosciences, Inc. Genome editing of a Rosa locus using nucleases
CA2798988C (en) 2010-05-17 2020-03-10 Sangamo Biosciences, Inc. Tal-effector (tale) dna-binding polypeptides and uses thereof
CA2805442C (en) 2010-07-21 2020-05-12 Sangamo Biosciences, Inc. Methods and compositions for modification of an hla locus
WO2012051343A1 (en) 2010-10-12 2012-04-19 The Children's Hospital Of Philadelphia Methods and compositions for treating hemophilia b
CN107828820B (zh) * 2010-10-27 2022-06-07 学校法人自治医科大学 用于向神经系统细胞导入基因的腺相关病毒粒子
CA3186126A1 (en) 2011-09-21 2013-03-28 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
CA2852955C (en) 2011-10-27 2021-02-16 Sangamo Biosciences, Inc. Methods and compositions for modification of the hprt locus
HK1200871A1 (en) 2011-11-16 2015-08-14 Sangamo Therapeutics, Inc. Modified dna-binding proteins and uses thereof
ES2828663T3 (es) * 2012-04-18 2021-05-27 Univ Leland Stanford Junior Dirección genética no disruptiva
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
ES2824024T3 (es) 2012-10-10 2021-05-11 Sangamo Therapeutics Inc Compuestos modificadores de células T y usos de los mismos
WO2014059115A1 (en) * 2012-10-10 2014-04-17 University Of Washington Through Its Center For Commercialization Compositions and methods for diagnosis and treatment of neurological disease
AU2013355327A1 (en) 2012-12-05 2015-06-11 Sangamo Therapeutics, Inc. Methods and compositions for regulation of metabolic disorders
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP2981829A4 (en) * 2013-04-01 2016-10-26 Immunomedics Inc ANTI-MUCIN ANTIBODIES FOR EARLY DETECTION AND TREATMENT OF PANCREATIC CANCER
AU2014265331B2 (en) 2013-05-15 2019-12-05 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
WO2015057976A1 (en) 2013-10-17 2015-04-23 Sangamo Biosciences, Inc. Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells
EP3757116A1 (en) 2013-12-09 2020-12-30 Sangamo Therapeutics, Inc. Methods and compositions for genome engineering
RU2691102C2 (ru) 2014-05-08 2019-06-11 Сангамо Байосайенсиз, Инк. Способы и композиции для лечения болезни хантингтона
US9816074B2 (en) 2014-07-25 2017-11-14 Sangamo Therapeutics, Inc. Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells
US9616090B2 (en) 2014-07-30 2017-04-11 Sangamo Biosciences, Inc. Gene correction of SCID-related genes in hematopoietic stem and progenitor cells
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity

Also Published As

Publication number Publication date
AU2016361350B2 (en) 2023-04-06
JP6976249B2 (ja) 2021-12-08
EP3380622A1 (en) 2018-10-03
JP2018533966A (ja) 2018-11-22
US11793888B2 (en) 2023-10-24
JP2021166554A (ja) 2021-10-21
EP3380622A4 (en) 2019-08-07
US10639383B2 (en) 2020-05-05
WO2017091512A1 (en) 2017-06-01
US20200282079A1 (en) 2020-09-10
AU2016361350A1 (en) 2018-05-24
JP7493487B2 (ja) 2024-05-31
US20170196992A1 (en) 2017-07-13

Similar Documents

Publication Publication Date Title
US11793888B2 (en) Methods and compositions for engineering immunity
CA2852955C (en) Methods and compositions for modification of the hprt locus
JP6875362B2 (ja) ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法および組成物
US9631187B2 (en) Methods and compositions for gene correction
US9833479B2 (en) Gene correction of SCID-related genes in hematopoietic stem and progenitor cells
AU2012312260B2 (en) Methods and compositions for regulation of transgene expression
JP2022105621A (ja) 造血幹細胞および前駆細胞におけるscid関連遺伝子の遺伝子修正
HK1200696B (en) Methods and compositions for modification of the hprt locus

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20211118

EEER Examination request

Effective date: 20211118

EEER Examination request

Effective date: 20211118

EEER Examination request

Effective date: 20211118

EEER Examination request

Effective date: 20211118

EEER Examination request

Effective date: 20211118