MX2019012452A - Meganucleasas diseñadas especificas para secuencias de reconocimiento en el gen pcsk9. - Google Patents

Meganucleasas diseñadas especificas para secuencias de reconocimiento en el gen pcsk9.

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Publication number
MX2019012452A
MX2019012452A MX2019012452A MX2019012452A MX2019012452A MX 2019012452 A MX2019012452 A MX 2019012452A MX 2019012452 A MX2019012452 A MX 2019012452A MX 2019012452 A MX2019012452 A MX 2019012452A MX 2019012452 A MX2019012452 A MX 2019012452A
Authority
MX
Mexico
Prior art keywords
engineered meganucleases
pcsk9 gene
recognition sequences
engineered
hypercholesterolemia
Prior art date
Application number
MX2019012452A
Other languages
English (en)
Spanish (es)
Inventor
Derek Jantz
Victor Bartsevich
James Jefferson Smith
Janel Lape
Original Assignee
Prec Biosciences 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 Prec Biosciences Inc filed Critical Prec Biosciences Inc
Publication of MX2019012452A publication Critical patent/MX2019012452A/es

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • 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
    • 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
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • 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
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors 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)
MX2019012452A 2017-04-21 2018-04-20 Meganucleasas diseñadas especificas para secuencias de reconocimiento en el gen pcsk9. MX2019012452A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762488403P 2017-04-21 2017-04-21
US201762516966P 2017-06-08 2017-06-08
PCT/US2018/028607 WO2018195449A1 (en) 2017-04-21 2018-04-20 Engineered meganucleases specific for recognition sequences in the pcsk9 gene

Publications (1)

Publication Number Publication Date
MX2019012452A true MX2019012452A (es) 2020-08-03

Family

ID=62117064

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2019012452A MX2019012452A (es) 2017-04-21 2018-04-20 Meganucleasas diseñadas especificas para secuencias de reconocimiento en el gen pcsk9.
MX2023001877A MX2023001877A (es) 2017-04-21 2019-10-17 Meganucleasas dise?adas especificas para secuencias de reconocimiento en el gen pcsk9.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2023001877A MX2023001877A (es) 2017-04-21 2019-10-17 Meganucleasas dise?adas especificas para secuencias de reconocimiento en el gen pcsk9.

Country Status (11)

Country Link
US (4) US11680254B2 (https=)
EP (2) EP3612205B9 (https=)
JP (4) JP2020517255A (https=)
KR (2) KR102620399B1 (https=)
CN (2) CN117264929A (https=)
AU (2) AU2018254576B2 (https=)
CA (1) CA3060112A1 (https=)
ES (1) ES2953153T3 (https=)
IL (3) IL324738A (https=)
MX (2) MX2019012452A (https=)
WO (1) WO2018195449A1 (https=)

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WO2020214724A1 (en) * 2019-04-15 2020-10-22 The Trustees Of The University Of Pennsylvania Compositions for regulating and self-inactivating enzyme expression and methods for modulating off-target activity of enzymes
CA3137975A1 (en) 2019-05-07 2020-11-12 Precision Biosciences, Inc. Optimization of engineered meganucleases for recognition sequences
WO2021222249A1 (en) * 2020-04-27 2021-11-04 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
WO2021222238A1 (en) * 2020-04-27 2021-11-04 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
US20230183664A1 (en) 2020-05-11 2023-06-15 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
US20230304034A1 (en) 2020-05-12 2023-09-28 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
AU2021369793A1 (en) 2020-10-29 2023-06-08 The Trustees Of The University Of Pennsylvania Aav capsids and compositions containing same
CA3172481A1 (en) * 2020-11-12 2022-05-19 Precision Biosciences, Inc. Engineered meganucleases having specificity for recognition sequences in the dystrophin gene
US20240197916A1 (en) * 2021-04-27 2024-06-20 The Trustees Of The University Of Pennsylvania Compositions and methods for in vivo nuclease-mediated gene targeting for the treatment of genetic disorders
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
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2023240076A1 (en) 2022-06-07 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
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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US5652224A (en) * 1995-02-24 1997-07-29 The Trustees Of The University Of Pennsylvania Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism
US6015832A (en) 1997-12-31 2000-01-18 The Regents Of The University Of Michigan Methods of inactivating bacteria including bacterial spores
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US7767216B2 (en) 1999-04-28 2010-08-03 The Regents Of The University Of Michigan Antimicrobial compositions and methods of use
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Also Published As

Publication number Publication date
CN117264929A (zh) 2023-12-22
IL270020A (https=) 2019-12-31
CA3060112A1 (en) 2018-10-25
CN110769845A (zh) 2020-02-07
EP3612205C0 (en) 2023-06-07
AU2023201658A1 (en) 2023-06-22
KR20230170125A (ko) 2023-12-18
EP4269596A3 (en) 2024-02-14
EP3612205B9 (en) 2023-10-04
IL270020B1 (en) 2023-04-01
JP7214915B2 (ja) 2023-01-30
IL270020B2 (en) 2023-08-01
KR102620399B1 (ko) 2024-01-04
WO2018195449A1 (en) 2018-10-25
US20250154481A1 (en) 2025-05-15
EP3612205B1 (en) 2023-06-07
JP2020517255A (ja) 2020-06-18
US20240101985A1 (en) 2024-03-28
AU2018254576A1 (en) 2019-11-07
EP3612205A1 (en) 2020-02-26
IL301059A (en) 2023-05-01
IL301059B2 (en) 2026-04-01
US20240101986A1 (en) 2024-03-28
IL324738A (en) 2026-01-01
CN110769845B (zh) 2023-08-15
US11680254B2 (en) 2023-06-20
ES2953153T3 (es) 2023-11-08
IL301059B1 (en) 2025-12-01
KR20190140982A (ko) 2019-12-20
JP2022188058A (ja) 2022-12-20
US20200131489A1 (en) 2020-04-30
JP2025066725A (ja) 2025-04-23
MX2023001877A (es) 2023-03-10
AU2018254576B2 (en) 2022-12-22
EP4269596A2 (en) 2023-11-01
JP2023058509A (ja) 2023-04-25

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