CA2913871C - A laglidadg homing endonuclease cleaving the c-c chemokine receptor type-5 (ccr5) gene and uses thereof - Google Patents

A laglidadg homing endonuclease cleaving the c-c chemokine receptor type-5 (ccr5) gene and uses thereof Download PDF

Info

Publication number
CA2913871C
CA2913871C CA2913871A CA2913871A CA2913871C CA 2913871 C CA2913871 C CA 2913871C CA 2913871 A CA2913871 A CA 2913871A CA 2913871 A CA2913871 A CA 2913871A CA 2913871 C CA2913871 C CA 2913871C
Authority
CA
Canada
Prior art keywords
seq
ccr5
variant
sequence
cells
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.)
Active
Application number
CA2913871A
Other languages
English (en)
French (fr)
Other versions
CA2913871A1 (en
Inventor
Jordan JARJOUR
Alexander ASTRAKHAN
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.)
Cellectis SA
Precision Genome Engineering Inc
Original Assignee
Cellectis SA
Precision Genome Engineering 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 Cellectis SA, Precision Genome Engineering Inc filed Critical Cellectis SA
Publication of CA2913871A1 publication Critical patent/CA2913871A1/en
Application granted granted Critical
Publication of CA2913871C publication Critical patent/CA2913871C/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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
    • C12N15/87Introduction 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • A61K40/11T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
    • 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
    • A61K40/46Viral antigens
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • A61P31/18Antivirals for RNA viruses for HIV
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Cell Biology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • AIDS & HIV (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
CA2913871A 2013-05-31 2014-05-28 A laglidadg homing endonuclease cleaving the c-c chemokine receptor type-5 (ccr5) gene and uses thereof Active CA2913871C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
DKPA201370302 2013-05-31
DKPA201370302 2013-05-31
PCT/EP2014/061186 WO2014191525A1 (en) 2013-05-31 2014-05-28 A laglidadg homing endonuclease cleaving the c-c chemokine receptor type-5 (ccr5) gene and uses thereof

Publications (2)

Publication Number Publication Date
CA2913871A1 CA2913871A1 (en) 2014-12-04
CA2913871C true CA2913871C (en) 2021-07-13

Family

ID=48628225

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2913871A Active CA2913871C (en) 2013-05-31 2014-05-28 A laglidadg homing endonuclease cleaving the c-c chemokine receptor type-5 (ccr5) gene and uses thereof

Country Status (6)

Country Link
US (1) US10006052B2 (https=)
EP (1) EP3004149B1 (https=)
JP (1) JP6488283B2 (https=)
AU (1) AU2014273089B2 (https=)
CA (1) CA2913871C (https=)
WO (1) WO2014191525A1 (https=)

Families Citing this family (69)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
US9458450B2 (en) 2012-03-15 2016-10-04 Flodesign Sonics, Inc. Acoustophoretic separation technology using multi-dimensional standing waves
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
CN106459995B (zh) 2013-11-07 2020-02-21 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
WO2015105955A1 (en) 2014-01-08 2015-07-16 Flodesign Sonics, Inc. Acoustophoresis device with dual acoustophoretic chamber
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
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
WO2017053729A1 (en) 2015-09-25 2017-03-30 The Board Of Trustees Of The Leland Stanford Junior University Nuclease-mediated genome editing of primary cells and enrichment thereof
JP6816133B2 (ja) 2015-10-05 2021-01-20 プレシジョン バイオサイエンシズ,インク. 改変ヒトt細胞受容体アルファ定常領域遺伝子を含む遺伝子改変細胞
EP3359660B1 (en) 2015-10-05 2019-12-04 Precision Biosciences, Inc. Engineered meganucleases with recognition sequences found in the human t cell receptor alpha constant region gene
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
US11085035B2 (en) 2016-05-03 2021-08-10 Flodesign Sonics, Inc. Therapeutic cell washing, concentration, and separation utilizing acoustophoresis
WO2018022619A1 (en) * 2016-07-25 2018-02-01 Bluebird Bio, Inc. Bcl11a homing endonuclease variants, compositions, and methods of use
CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
MA45996A (fr) 2016-08-16 2021-06-02 Bluebird Bio Inc Variants d'endonucléase de homing du récepteur alpha de l'il-10, compositions et méthodes d'utilisation associées
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR102451510B1 (ko) 2016-09-08 2022-10-07 2세븐티 바이오, 인코포레이티드 Pd-1 호밍 엔도뉴클레아제 변이체, 조성물 및 사용 방법
KR102622411B1 (ko) 2016-10-14 2024-01-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
MA46543A (fr) * 2016-10-17 2019-08-21 Bluebird Bio Inc Variants de l'endonucléase tgfbéta r2, compositions et procédés d'utilisation
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
WO2018189360A1 (en) 2017-04-13 2018-10-18 Cellectis New sequence specific reagents targeting ccr5 in primary hematopoietic cells
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
MA48797A (fr) * 2017-05-25 2020-04-08 Bluebird Bio Inc Variants de l'endonucléase cblb , compositions et procédés d'utilisation
EP3645038B1 (en) 2017-06-30 2026-02-18 Precision Biosciences, Inc. Genetically-modified t cells comprising a modified intron in the t cell receptor alpha gene
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
KR102439221B1 (ko) 2017-12-14 2022-09-01 프로디자인 소닉스, 인크. 음향 트랜스듀서 구동기 및 제어기
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
WO2019126555A1 (en) * 2017-12-20 2019-06-27 Bluebird Bio, Inc. Ctla4 homing endonuclease variants, compositions, and methods of use
US11786554B2 (en) 2018-04-12 2023-10-17 Precision Biosciences, Inc. Optimized engineered nucleases having specificity for the human T cell receptor alpha constant region gene
EP3781677A4 (en) 2018-04-16 2022-01-19 University of Massachusetts COMPOSITIONS AND METHODS FOR IMPROVED GENE EDITTING
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
DK3806903T5 (da) 2018-06-14 2024-08-19 2Seventy Bio Inc Cd79a kimæriske antigenreceptorer
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
WO2020072059A1 (en) 2018-10-04 2020-04-09 Bluebird Bio, Inc. Cblb endonuclease variants, compositions, and methods of use
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
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
IL283724B1 (en) 2018-12-10 2026-02-01 Genetix Biotherapeutics Inc pdcd-1 homing endonuclease variants
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
US20220348912A1 (en) 2019-06-20 2022-11-03 University Of Massachusetts Compositions and methods for improved gene editing
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CN115916963A (zh) 2020-03-27 2023-04-04 门德斯有限公司 白血病来源的经修饰细胞用于增强过继性细胞治疗的效力的离体用途
WO2021222451A2 (en) * 2020-04-28 2021-11-04 City Of Hope Tagged gene editing technology for clinical cell sorting and enrichment
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
EP4171617A1 (en) 2020-06-30 2023-05-03 Mendus B.V. Use of leukemia-derived cells in ovarian cancer vaccines
CA3212351A1 (en) 2021-03-12 2022-09-15 Mendus B.V. Methods of vaccination and use of cd47 blockade
CA3237482A1 (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
GB202117314D0 (en) 2021-11-30 2022-01-12 Clarke David John Cyclic nucleic acid fragmentation
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

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20140148361A1 (en) * 2010-06-07 2014-05-29 Barry L. Stoddard Generation and Expression of Engineered I-ONUI Endonuclease and Its Homologues and Uses Thereof
US8673557B2 (en) * 2011-02-28 2014-03-18 Seattle Children's Research Institute Coupling endonucleases with end-processing enzymes drives high efficiency gene disruption
CA3111953C (en) * 2011-04-05 2023-10-24 Cellectis Method for the generation of compact tale-nucleases and uses thereof
EP2729567B1 (en) * 2011-07-08 2016-10-05 Cellectis Method for increasing the efficiency of double-strand break-induced mutagenssis
WO2013068845A2 (en) * 2011-11-07 2013-05-16 The University Of Western Ontario Endonuclease for genome editing

Also Published As

Publication number Publication date
CA2913871A1 (en) 2014-12-04
WO2014191525A1 (en) 2014-12-04
AU2014273089A1 (en) 2015-12-24
US20160102323A1 (en) 2016-04-14
AU2014273089B2 (en) 2018-02-22
HK1223373A1 (en) 2017-07-28
EP3004149A1 (en) 2016-04-13
US10006052B2 (en) 2018-06-26
JP6488283B2 (ja) 2019-03-20
JP2016520319A (ja) 2016-07-14
EP3004149B1 (en) 2018-12-19

Similar Documents

Publication Publication Date Title
CA2913871C (en) A laglidadg homing endonuclease cleaving the c-c chemokine receptor type-5 (ccr5) gene and uses thereof
AU2014273091B2 (en) A LAGLIDADG homing endonuclease cleaving the T cell receptor alpha gene and uses thereof
EP2729567B1 (en) Method for increasing the efficiency of double-strand break-induced mutagenssis
US11220683B2 (en) Method to overcome DNA chemical modifications sensitivity of engineered TALE DNA binding domains
US20130337454A1 (en) Method for increasing the efficiency of double-strand break-induced mutagenesis
CN103608027A (zh) 用于生成致密tale-核酸酶的方法及其用途
US20230046668A1 (en) Targeted integration in mammalian sequences enhancing gene expression
HK1223373B (en) A laglidadg homing endonuclease cleaving the c-c chemokine receptor type-5 (ccr5) gene and uses thereof
US20160138047A1 (en) Improved polynucleotide sequences encoding tale repeats
WO2012007848A2 (en) Meganuclease variants cleaving a dna target sequence in the was gene and uses thereof
HK1223394B (en) A laglidadg homing endonuclease cleaving the t cell receptor alpha gene and uses thereof
WO2026046724A1 (en) Tale protein scaffolds involving fusions of monopartite and bipartite nls
HK1201553B (en) Method to overcome dna chemical modifications sensitivity of engineered tale dna binding domains

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20190528

MPN Maintenance fee for patent paid

Free format text: FEE DESCRIPTION TEXT: MF (PATENT, 11TH ANNIV.) - STANDARD

Year of fee payment: 11

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250401

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250401