JP2020517255A - Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ - Google Patents

Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ Download PDF

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JP2020517255A
JP2020517255A JP2019556884A JP2019556884A JP2020517255A JP 2020517255 A JP2020517255 A JP 2020517255A JP 2019556884 A JP2019556884 A JP 2019556884A JP 2019556884 A JP2019556884 A JP 2019556884A JP 2020517255 A JP2020517255 A JP 2020517255A
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meganuclease
seq
engineered meganuclease
engineered
acid sequence
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JP2020517255A5 (enExample
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バートサビチ，ビクター
ジャンツ，デレック
スミス，ジェームス，ジェファーソン
ラペ，ジャネル
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プレシジョン バイオサイエンシズ，インク．
プレシジョン バイオサイエンシズ，インク．
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Publication of JP2020517255A publication Critical patent/JP2020517255A/ja
Publication of JP2020517255A5 publication Critical patent/JP2020517255A5/ja
Priority to JP2022145178A priority Critical patent/JP7214915B2/ja
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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
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2019556884A 2017-04-21 2018-04-20 Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ Pending JP2020517255A (ja)

Priority Applications (1)

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JP2022145178A JP7214915B2 (ja) 2017-04-21 2022-09-13 Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ

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US201762488403P 2017-04-21 2017-04-21
US62/488,403 2017-04-21
US201762516966P 2017-06-08 2017-06-08
US62/516,966 2017-06-08
PCT/US2018/028607 WO2018195449A1 (en) 2017-04-21 2018-04-20 Engineered meganucleases specific for recognition sequences in the pcsk9 gene

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JP2020517255A true JP2020517255A (ja) 2020-06-18
JP2020517255A5 JP2020517255A5 (enExample) 2021-05-27

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JP2019556884A Pending JP2020517255A (ja) 2017-04-21 2018-04-20 Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ
JP2022145178A Active JP7214915B2 (ja) 2017-04-21 2022-09-13 Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ
JP2023005733A Pending JP2023058509A (ja) 2017-04-21 2023-01-18 Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ
JP2024231599A Pending JP2025066725A (ja) 2017-04-21 2024-12-27 Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ

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JP2023005733A Pending JP2023058509A (ja) 2017-04-21 2023-01-18 Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ
JP2024231599A Pending JP2025066725A (ja) 2017-04-21 2024-12-27 Ｐｃｓｋ９遺伝子の認識配列に特異的な操作されたメガヌクレアーゼ

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US (4) US11680254B2 (enExample)
EP (2) EP4269596A3 (enExample)
JP (4) JP2020517255A (enExample)
KR (2) KR102620399B1 (enExample)
CN (2) CN117264929A (enExample)
AU (2) AU2018254576B2 (enExample)
CA (1) CA3060112A1 (enExample)
ES (1) ES2953153T3 (enExample)
IL (2) IL270020B2 (enExample)
MX (2) MX2019012452A (enExample)
WO (1) WO2018195449A1 (enExample)

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US20230167464A1 (en) * 2020-04-27 2023-06-01 The Trustees Of The University Of Pennsylvania Compositions and methods for reducing nuclease expression and off-target activity using a promoter with low transcriptional activity
EP4143308A4 (en) * 2020-04-27 2024-06-26 The Trustees of The University of Pennsylvania COMPOSITIONS AND METHODS FOR REDUCING NUCLEASE EXPRESSION AND OFF-TARGET ACTIVITY USING A PROMOTER WITH LOW TRANSCRIPTION ACTIVITY
WO2021231259A1 (en) 2020-05-11 2021-11-18 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
AU2021270447A1 (en) 2020-05-12 2023-01-05 The Trustees Of The University Of Pennsylvania Compositions for DRG-specific reduction of transgene expression
EP4236975A1 (en) 2020-10-29 2023-09-06 The Trustees of The University of Pennsylvania Aav capsids and compositions containing same
EP4244342A1 (en) * 2020-11-12 2023-09-20 Precision BioSciences, Inc. Engineered meganucleases having specificity for recognition sequences in the dystrophin gene
TW202309066A (zh) 2021-04-27 2023-03-01 賓州大學委員會 衍生自豬的腺相關病毒衣殼及其用途
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2024015966A2 (en) 2022-07-15 2024-01-18 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
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JP2009511085A (ja) * 2005-10-18 2009-03-19 プレシジョン バイオサイエンシズ 配列特異性およびｄｎａ−結合親和度が変更された、合理設計メガヌクレアーゼ
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MX2019012452A (es) 2020-08-03
JP2023058509A (ja) 2023-04-25
CN117264929A (zh) 2023-12-22
MX2023001877A (es) 2023-03-10
AU2018254576A1 (en) 2019-11-07
US20250154481A1 (en) 2025-05-15
IL301059B1 (en) 2025-12-01
EP3612205B9 (en) 2023-10-04
IL270020B1 (en) 2023-04-01
EP4269596A3 (en) 2024-02-14
WO2018195449A1 (en) 2018-10-25
AU2023201658A1 (en) 2023-06-22
US20240101985A1 (en) 2024-03-28
EP3612205A1 (en) 2020-02-26
CN110769845B (zh) 2023-08-15
KR102620399B1 (ko) 2024-01-04
IL270020A (enExample) 2019-12-31
JP7214915B2 (ja) 2023-01-30
EP4269596A2 (en) 2023-11-01
AU2018254576B2 (en) 2022-12-22
EP3612205C0 (en) 2023-06-07
ES2953153T3 (es) 2023-11-08
EP3612205B1 (en) 2023-06-07
US20200131489A1 (en) 2020-04-30
IL270020B2 (en) 2023-08-01
IL301059A (en) 2023-05-01
CN110769845A (zh) 2020-02-07
JP2025066725A (ja) 2025-04-23
US11680254B2 (en) 2023-06-20
CA3060112A1 (en) 2018-10-25
US20240101986A1 (en) 2024-03-28
KR20190140982A (ko) 2019-12-20
JP2022188058A (ja) 2022-12-20
KR20230170125A (ko) 2023-12-18

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