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MX2023001877A - Meganucleasas dise?adas especificas para secuencias de reconocimiento en el gen pcsk9. - Google Patents

Meganucleasas dise?adas especificas para secuencias de reconocimiento en el gen pcsk9.

Info

Publication number
MX2023001877A
MX2023001877A MX2023001877A MX2023001877A MX2023001877A MX 2023001877 A MX2023001877 A MX 2023001877A MX 2023001877 A MX2023001877 A MX 2023001877A MX 2023001877 A MX2023001877 A MX 2023001877A MX 2023001877 A MX2023001877 A MX 2023001877A
Authority
MX
Mexico
Prior art keywords
engineered meganucleases
pcsk9 gene
recognition sequences
engineered
hypercholesterolemia
Prior art date
Application number
MX2023001877A
Other languages
English (en)
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 MX2023001877A publication Critical patent/MX2023001877A/es

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    • 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
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    • 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
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    • 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
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
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    • 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
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    • 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)
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  • Wood Science & Technology (AREA)
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  • 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)

Abstract

La presente invención abarca meganucleasas diseñadas que reconocen y cortan una secuencia de reconocimiento dentro del gen humano PCSK9. La presente invención también abarca métodos para utilizar dichas meganucleasas diseñadas en una composición farmacéutica y en métodos para tratar o reducir los síntomas de trastornos relacionados con el colesterol, tales como hipercolesterolemia. Además, la invención abarca composiciones farmacéuticas que comprenden proteínas de meganucleasas diseñadas, ácidos nucleicos que codifican meganucleasas diseñadas y el uso de dichas composiciones para el tratamiento de trastornos relacionados con el colesterol, tales como hipercolesterolemia.
MX2023001877A 2017-04-21 2019-10-17 Meganucleasas dise?adas especificas para secuencias de reconocimiento en el gen pcsk9. MX2023001877A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762488403P 2017-04-21 2017-04-21
US201762516966P 2017-06-08 2017-06-08

Publications (1)

Publication Number Publication Date
MX2023001877A true MX2023001877A (es) 2023-03-10

Family

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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 Before (1)

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MX2019012452A MX2019012452A (es) 2017-04-21 2018-04-20 Meganucleasas diseñadas especificas para secuencias de reconocimiento en el gen pcsk9.

Country Status (11)

Country Link
US (4) US11680254B2 (es)
EP (2) EP4269596A3 (es)
JP (4) JP2020517255A (es)
KR (2) KR20230170125A (es)
CN (2) CN117264929A (es)
AU (2) AU2018254576B2 (es)
CA (1) CA3060112A1 (es)
ES (1) ES2953153T3 (es)
IL (3) IL301059B1 (es)
MX (2) MX2019012452A (es)
WO (1) WO2018195449A1 (es)

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IL301059A (en) 2023-05-01
US11680254B2 (en) 2023-06-20
KR20230170125A (ko) 2023-12-18
EP3612205A1 (en) 2020-02-26
IL270020B1 (en) 2023-04-01
EP3612205B9 (en) 2023-10-04
KR20190140982A (ko) 2019-12-20
WO2018195449A1 (en) 2018-10-25
CA3060112A1 (en) 2018-10-25
AU2018254576A1 (en) 2019-11-07
JP2020517255A (ja) 2020-06-18
CN110769845A (zh) 2020-02-07
ES2953153T3 (es) 2023-11-08
US20200131489A1 (en) 2020-04-30
US20240101985A1 (en) 2024-03-28
EP4269596A3 (en) 2024-02-14
KR102620399B1 (ko) 2024-01-04
IL324738A (en) 2026-01-01
AU2023201658A1 (en) 2023-06-22
IL270020A (es) 2019-12-31
US20240101986A1 (en) 2024-03-28
EP4269596A2 (en) 2023-11-01
CN117264929A (zh) 2023-12-22
JP2022188058A (ja) 2022-12-20
IL301059B1 (en) 2025-12-01
IL270020B2 (en) 2023-08-01
AU2018254576B2 (en) 2022-12-22
EP3612205C0 (en) 2023-06-07
JP7214915B2 (ja) 2023-01-30
JP2025066725A (ja) 2025-04-23
EP3612205B1 (en) 2023-06-07
US20250154481A1 (en) 2025-05-15
CN110769845B (zh) 2023-08-15
JP2023058509A (ja) 2023-04-25
MX2019012452A (es) 2020-08-03

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