CA3031785A1 - Bcl11a homing endonuclease variants, compositions, and methods of use - Google Patents

Bcl11a homing endonuclease variants, compositions, and methods of use Download PDF

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Publication number
CA3031785A1
CA3031785A1 CA3031785A CA3031785A CA3031785A1 CA 3031785 A1 CA3031785 A1 CA 3031785A1 CA 3031785 A CA3031785 A CA 3031785A CA 3031785 A CA3031785 A CA 3031785A CA 3031785 A1 CA3031785 A1 CA 3031785A1
Authority
CA
Canada
Prior art keywords
beta
polypeptide
amino acid
seq
cell
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.)
Abandoned
Application number
CA3031785A
Other languages
English (en)
French (fr)
Inventor
Jordan JARJOUR
Jasdeep MANN
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.)
Bluebird Bio Inc
Original Assignee
Bluebird Bio 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 Bluebird Bio Inc filed Critical Bluebird Bio Inc
Publication of CA3031785A1 publication Critical patent/CA3031785A1/en
Abandoned legal-status Critical Current

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/21—Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Manufacturing & Machinery (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3031785A 2016-07-25 2017-07-25 Bcl11a homing endonuclease variants, compositions, and methods of use Abandoned CA3031785A1 (en)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201662366530P 2016-07-25 2016-07-25
US62/366,530 2016-07-25
US201662367465P 2016-07-27 2016-07-27
US62/367,465 2016-07-27
US201662375829P 2016-08-16 2016-08-16
US62/375,829 2016-08-16
US201662414273P 2016-10-28 2016-10-28
US62/414,273 2016-10-28
PCT/US2017/043726 WO2018022619A1 (en) 2016-07-25 2017-07-25 Bcl11a homing endonuclease variants, compositions, and methods of use

Publications (1)

Publication Number Publication Date
CA3031785A1 true CA3031785A1 (en) 2018-02-01

Family

ID=61017227

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3031785A Abandoned CA3031785A1 (en) 2016-07-25 2017-07-25 Bcl11a homing endonuclease variants, compositions, and methods of use

Country Status (7)

Country Link
US (1) US20190184035A1 (enExample)
EP (1) EP3487994A4 (enExample)
JP (1) JP2019525759A (enExample)
CN (1) CN109689865A (enExample)
AU (1) AU2017301609A1 (enExample)
CA (1) CA3031785A1 (enExample)
WO (1) WO2018022619A1 (enExample)

Families Citing this family (26)

* 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
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
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
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
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
IL270776B2 (en) 2017-05-25 2024-11-01 Bluebird Bio Inc Cblb endonuclease variants, compositions, and methods of use
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
WO2019210213A1 (en) * 2018-04-27 2019-10-31 Seattle Children's Hospital D/B/A Seattle Children's Research Institute Bruton's tyrosine kinase homing 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
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
US20220364123A1 (en) * 2019-04-24 2022-11-17 Seattle Children's Hospital D/B/A Seattle Children's Research Institute Wiskott-aldrich syndrome gene homing endonuclease variants, compositions, and methods of use
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
AU2025209576A1 (en) * 2024-01-16 2026-06-11 Novo Nordisk A/S Albumin-targeted endonucleases, compositions, and methods of use
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

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009095742A1 (en) * 2008-01-31 2009-08-06 Cellectis New i-crei derived single-chain meganuclease and uses thereof
ES2422291T3 (es) * 2007-10-31 2013-09-10 Prec Biosciences Inc Meganucleasas monocatenarias diseñadas racionalmente con secuencias de reconocimiento no palindrómicas
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
EP2836226B1 (en) * 2012-02-24 2017-06-28 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
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

Also Published As

Publication number Publication date
JP2019525759A (ja) 2019-09-12
WO2018022619A1 (en) 2018-02-01
CN109689865A (zh) 2019-04-26
EP3487994A4 (en) 2020-01-29
US20190184035A1 (en) 2019-06-20
AU2017301609A1 (en) 2019-02-21
EP3487994A1 (en) 2019-05-29

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Legal Events

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FZDE Discontinued

Effective date: 20230126