AU2018254576B2 - Engineered meganucleases specific for recognition sequences in the PCSK9 gene - Google Patents

Engineered meganucleases specific for recognition sequences in the PCSK9 gene Download PDF

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AU2018254576B2
AU2018254576B2 AU2018254576A AU2018254576A AU2018254576B2 AU 2018254576 B2 AU2018254576 B2 AU 2018254576B2 AU 2018254576 A AU2018254576 A AU 2018254576A AU 2018254576 A AU2018254576 A AU 2018254576A AU 2018254576 B2 AU2018254576 B2 AU 2018254576B2
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AU2018254576A1 (en
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Victor Bartsevich
Derek Jantz
Janel LAPE
James Jefferson Smith
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Precision Biosciences Inc
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    • 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
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50Microcapsules 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
    • A61K9/51Nanocapsules; Nanoparticles
    • A61K9/5107Excipients; Inactive ingredients
    • A61K9/5123Organic compounds, e.g. fats, sugars
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N9/14Hydrolases (3)
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    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424Serine endopeptidases (3.4.21)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
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    • C07KPEPTIDES
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • 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)
AU2018254576A 2017-04-21 2018-04-20 Engineered meganucleases specific for recognition sequences in the PCSK9 gene Active AU2018254576B2 (en)

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AU2023201658A AU2023201658A1 (en) 2017-04-21 2023-03-16 Engineered meganucleases specific for recognition sequences in the PCSK9 gene

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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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AU2018254576A1 AU2018254576A1 (en) 2019-11-07
AU2018254576B2 true AU2018254576B2 (en) 2022-12-22

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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)
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US20220195407A1 (en) 2019-05-07 2022-06-23 Precision Biosciences, Inc. Optimization of engineered meganucleases for recognition sequences
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
WO2025122552A1 (en) * 2023-12-04 2025-06-12 The Trustees Of The University Of Pennsylvania In silico prediction tool for meganuclease off-target sites

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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
JP2020517255A (ja) 2020-06-18
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
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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