CN109689865A - Bcl11a归巢核酸内切酶变体、组合物和使用方法 - Google Patents

Bcl11a归巢核酸内切酶变体、组合物和使用方法 Download PDF

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Publication number
CN109689865A
CN109689865A CN201780054710.4A CN201780054710A CN109689865A CN 109689865 A CN109689865 A CN 109689865A CN 201780054710 A CN201780054710 A CN 201780054710A CN 109689865 A CN109689865 A CN 109689865A
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polypeptide
amino acid
cell
seq
variant
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Pending
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CN201780054710.4A
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English (en)
Chinese (zh)
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乔丹·贾儒尔
贾斯迪普·曼恩
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Bluebird Bio Inc
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Bluebird Bio Inc
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    • 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)

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  • 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)
CN201780054710.4A 2016-07-25 2017-07-25 Bcl11a归巢核酸内切酶变体、组合物和使用方法 Pending CN109689865A (zh)

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
CN109689865A true CN109689865A (zh) 2019-04-26

Family

ID=61017227

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201780054710.4A Pending CN109689865A (zh) 2016-07-25 2017-07-25 Bcl11a归巢核酸内切酶变体、组合物和使用方法

Country Status (7)

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

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US9458450B2 (en) 2012-03-15 2016-10-04 Flodesign Sonics, Inc. Acoustophoretic separation technology using multi-dimensional standing waves
US9950282B2 (en) 2012-03-15 2018-04-24 Flodesign Sonics, Inc. Electronic configuration and control for acoustic standing wave generation
US10967298B2 (en) 2012-03-15 2021-04-06 Flodesign Sonics, Inc. Driver and control for variable impedence load
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
WO2015105955A1 (en) 2014-01-08 2015-07-16 Flodesign Sonics, Inc. Acoustophoresis device with dual acoustophoretic chamber
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
KR102451510B1 (ko) 2016-09-08 2022-10-07 2세븐티 바이오, 인코포레이티드 Pd-1 호밍 엔도뉴클레아제 변이체, 조성물 및 사용 방법
MA46543A (fr) 2016-10-17 2019-08-21 Bluebird Bio Inc Variants de l'endonucléase tgfbéta r2, compositions et procédés d'utilisation
WO2018075830A1 (en) 2016-10-19 2018-04-26 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
MA48797A (fr) 2017-05-25 2020-04-08 Bluebird Bio Inc Variants de l'endonucléase cblb , compositions et procédés d'utilisation
KR102439221B1 (ko) 2017-12-14 2022-09-01 프로디자인 소닉스, 인크. 음향 트랜스듀서 구동기 및 제어기
EP3784790A4 (en) * 2018-04-27 2022-02-09 Seattle Children's Hospital d/b/a Seattle Children's Research Institute BRUTON TYROSINE KINASE HOMING ENDONUCLEAR VARIANTS, COMPOSITIONS AND METHOD OF USE
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
IL283724B1 (en) 2018-12-10 2026-02-01 Genetix Biotherapeutics Inc pdcd-1 homing endonuclease variants
AU2020262409A1 (en) * 2019-04-24 2021-12-23 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
WO2025153530A1 (en) * 2024-01-16 2025-07-24 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

Citations (3)

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US20110179506A1 (en) * 2008-01-31 2011-07-21 Sylvestre Grizot New i-crei derived single-chain meganuclease and uses thereof
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
WO2014191527A1 (en) * 2013-05-31 2014-12-04 Cellectis A laglidadg homing endonuclease cleaving the t cell receptor alpha gene and uses thereof

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EP2215223B1 (en) * 2007-10-31 2013-05-01 Precision Biosciences, Inc. Rationally-designed single-chain meganucleases with non-palindromic recognition sequences
US20150166969A1 (en) * 2012-02-24 2015-06-18 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
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

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110179506A1 (en) * 2008-01-31 2011-07-21 Sylvestre Grizot New i-crei derived single-chain meganuclease and uses thereof
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
WO2014191527A1 (en) * 2013-05-31 2014-12-04 Cellectis A laglidadg homing endonuclease cleaving the t cell receptor alpha gene and uses thereof

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
TAKEUCHI ET AL.,: ""Redesign of extensive protein-DNA interfaces of meganucleases using iterative cycles of in vitro compartmentallization"", 《PNAS》 *

Also Published As

Publication number Publication date
EP3487994A4 (en) 2020-01-29
AU2017301609A1 (en) 2019-02-21
JP2019525759A (ja) 2019-09-12
EP3487994A1 (en) 2019-05-29
CA3031785A1 (en) 2018-02-01
US20190184035A1 (en) 2019-06-20
WO2018022619A1 (en) 2018-02-01

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