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

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

Info

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

Links

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)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Hematology (AREA)
  • Diabetes (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 (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9458450B2 (en) 2012-03-15 2016-10-04 Flodesign Sonics, Inc. Acoustophoretic separation technology using multi-dimensional standing waves
US10967298B2 (en) 2012-03-15 2021-04-06 Flodesign Sonics, Inc. Driver and control for variable impedence load
US9950282B2 (en) 2012-03-15 2018-04-24 Flodesign Sonics, Inc. Electronic configuration and control for acoustic standing wave generation
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
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11021699B2 (en) 2015-04-29 2021-06-01 FioDesign Sonics, Inc. Separation using angled acoustic waves
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
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
KR102622910B1 (ko) 2016-09-08 2024-01-10 2세븐티 바이오, 인코포레이티드 Pd-1 호밍 엔도뉴클레아제 변이체, 조성물 및 사용 방법
WO2018075541A1 (en) 2016-10-17 2018-04-26 Bluebird Bio, Inc. TGFβR2 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
CN110799644A (zh) 2017-05-25 2020-02-14 蓝鸟生物公司 Cblb核酸内切酶变体、组合物及使用方法
KR20220066413A (ko) 2017-12-14 2022-05-24 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
US12404500B2 (en) 2018-12-10 2025-09-02 Novo Nordisk A/S Homing endonuclease variants
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
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

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
WO2009059195A2 (en) * 2007-10-31 2009-05-07 Precision Biosciences Rationally-designed single-chain meganucleases with non-palindromic recognition sequences
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
EP3272356A1 (en) * 2012-02-24 2018-01-24 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
DK3004338T3 (en) * 2013-05-31 2019-04-08 Cellectis Sa LAGLIDADG HOMING ENDONUCLEASE DIVERSE T-CELL RECEPTOR ALPHA GENET AND APPLICATIONS THEREOF
JP6488283B2 (ja) * 2013-05-31 2019-03-20 セレクティスＣｅｌｌｅｃｔｉｓ Ｃ−ｃケモカイン受容体５型（ｃｃｒ５）遺伝子を開裂するｌａｇｌｉｄａｄｇホーミングエンドヌクレアーゼおよびその用途

Also Published As

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

Similar Documents

Publication Publication Date Title
US20190184035A1 (en) Bcl11a homing endonuclease variants, compositions, and methods of use
US20230174967A1 (en) Donor repair templates multiplex genome editing
US20190309274A1 (en) Il-10 receptor alpha homing endonuclease variants, compositions, and methods of use
US12264343B2 (en) TCRa homing endonuclease variants
US20250262323A1 (en) Pcsk9 endonuclease variants, compositions, and methods of use
US12385070B2 (en) Homology directed repair compositions for the treatment of hemoglobinopathies
JP2024109943A (ja) ブルトン型チロシンキナーゼに対するｔａｌｅｎベースのおよびｃｒｉｓｐｒ／ｃａｓベースのゲノム編集
EP3551750A1 (en) Gene therapy for mucopolysaccharidosis, type i
WO2019126558A1 (en) Ahr homing endonuclease variants, compositions, and methods of use
US20210230565A1 (en) Bruton's tyrosine kinase homing endonuclease variants, compositions, and methods of use
US20220364123A1 (en) Wiskott-aldrich syndrome gene homing endonuclease variants, compositions, and methods of use

Legal Events

Date Code Title Description
FZDE Discontinued

Effective date: 20230126

FZDE Discontinued

Effective date: 20230126