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DK3668972T3 - Konstruerede dnase-enzymer og anvendelse i terapi - Google Patents

Konstruerede dnase-enzymer og anvendelse i terapi Download PDF

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Publication number
DK3668972T3
DK3668972T3 DK18846060.4T DK18846060T DK3668972T3 DK 3668972 T3 DK3668972 T3 DK 3668972T3 DK 18846060 T DK18846060 T DK 18846060T DK 3668972 T3 DK3668972 T3 DK 3668972T3
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DK
Denmark
Prior art keywords
therapy
dnase enzymes
engineered
engineered dnase
enzymes
Prior art date
Application number
DK18846060.4T
Other languages
English (en)
Inventor
Tobias A Fuchs
Josephine Göbel (Formerly Bitterling Josephine)
Hanna Englert
Miguel Jiménez-AlcãZar
Original Assignee
Neutrolis Inc
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Filing date
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Publication of DK3668972T3 publication Critical patent/DK3668972T3/da

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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 RNAses, DNAses
    • 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
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/0075Medicinal 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 delivery route, e.g. oral, subcutaneous
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P41/00Drugs used in surgical methods, e.g. surgery adjuvants for preventing adhesion or for vitreum substitution
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • 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
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/31Fusion polypeptide fusions, other than Fc, for prolonged plasma life, e.g. albumin
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/90Fusion polypeptide containing a motif for post-translational modification
    • C07K2319/91Fusion polypeptide containing a motif for post-translational modification containing a motif for glycosylation
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/21Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)
    • C12Y301/21001Deoxyribonuclease I (3.1.21.1)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Surgery (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Urology & Nephrology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
DK18846060.4T 2017-08-18 2018-08-20 Konstruerede dnase-enzymer og anvendelse i terapi DK3668972T3 (da)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762547220P 2017-08-18 2017-08-18
US201762611166P 2017-12-28 2017-12-28
PCT/US2018/047084 WO2019036719A2 (en) 2017-08-18 2018-08-20 GENETICALLY MODIFIED DNASE ENZYMES AND THEIR USE IN THERAPY

Publications (1)

Publication Number Publication Date
DK3668972T3 true DK3668972T3 (da) 2025-01-13

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
DK18846060.4T DK3668972T3 (da) 2017-08-18 2018-08-20 Konstruerede dnase-enzymer og anvendelse i terapi

Country Status (14)

Country Link
US (5) US10696956B2 (da)
EP (2) EP4509135A2 (da)
JP (1) JP7577314B2 (da)
KR (1) KR20200047588A (da)
CN (1) CN111566207A (da)
AU (1) AU2018318313B2 (da)
BR (1) BR112020003403A2 (da)
CA (1) CA3073317A1 (da)
DK (1) DK3668972T3 (da)
ES (1) ES3008982T3 (da)
IL (2) IL272715B1 (da)
MX (1) MX2020001913A (da)
SG (1) SG11202001445XA (da)
WO (1) WO2019036719A2 (da)

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JP7577314B2 (ja) 2017-08-18 2024-11-05 ニュートロリス インコーポレイテッド 改変型dnase酵素及び治療における使用
WO2019143272A1 (en) 2018-01-16 2019-07-25 Cls Therapeutics Limited Treatment of diseases by liver expression of an enzyme which has a deoxyribonuclease (dnase) activity
US10988746B2 (en) 2018-10-08 2021-04-27 Neutrolis, Inc. Manufacturing and engineering of DNASE enzymes for therapy
AU2019358915A1 (en) * 2018-10-08 2021-05-20 Neutrolis, Inc. Engineering of DNASE enzymes for manufacturing and therapy
US11058724B2 (en) 2018-10-08 2021-07-13 Neutrolis, Inc. Methods of using DNASE1-like 3 in therapy
US11352613B2 (en) * 2019-02-04 2022-06-07 Neutrolis, Inc. Engineered human extracellular DNASE enzymes
WO2020163264A1 (en) * 2019-02-04 2020-08-13 Neutrolis, Inc. Engineered human extracellular dnase enzymes for drug candidate selection
WO2020227415A1 (en) * 2019-05-06 2020-11-12 President And Fellows Of Harvard College Dna-degrading proteins and uses thereof
WO2021071733A1 (en) * 2019-10-07 2021-04-15 Texas Tech University System Mutant dnase1l3 with improved serum half-life
IL294114A (en) * 2020-01-11 2022-08-01 Univ Yale Compositions and methods for treating, ameliorating, and/or preventing diseases or disorders caused by or associated with dnase1 and/or dnase1l3 deficiency
US20230072226A1 (en) * 2020-02-20 2023-03-09 Neutrolis, Inc. Basic domain-deleted dnase1-like 3 and uses thereof
US20230174944A1 (en) * 2020-04-15 2023-06-08 Cartesian Therapeutics, Inc. Engineered cells secreting therapeutic enzymes
CN116322743A (zh) * 2020-06-08 2023-06-23 耶鲁大学 用于治疗和/或预防患有细菌和/或病毒感染的患者的凝血病和/或脓毒症的组合物和方法
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JP7577314B2 (ja) 2017-08-18 2024-11-05 ニュートロリス インコーポレイテッド 改変型dnase酵素及び治療における使用

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Publication number Publication date
IL272715A (en) 2020-04-30
EP4509135A2 (en) 2025-02-19
BR112020003403A2 (pt) 2020-08-25
AU2018318313A1 (en) 2020-03-19
US20200399622A1 (en) 2020-12-24
MX2020001913A (es) 2020-09-07
US10801019B1 (en) 2020-10-13
WO2019036719A3 (en) 2019-03-28
CN111566207A (zh) 2020-08-21
EP3668972B8 (en) 2024-11-13
SG11202001445XA (en) 2020-03-30
CA3073317A1 (en) 2019-02-21
US11046943B2 (en) 2021-06-29
EP3668972A4 (en) 2021-04-21
US10696956B2 (en) 2020-06-30
US20200024585A1 (en) 2020-01-23
JP7577314B2 (ja) 2024-11-05
US20210277372A1 (en) 2021-09-09
JP2020534865A (ja) 2020-12-03
EP3668972A2 (en) 2020-06-24
IL317968A (en) 2025-02-01
KR20200047588A (ko) 2020-05-07
ES3008982T3 (en) 2025-03-25
IL272715B1 (en) 2025-01-01
EP3668972B1 (en) 2024-10-09
US20200347370A1 (en) 2020-11-05
US20210040461A1 (en) 2021-02-11
US11046942B2 (en) 2021-06-29
WO2019036719A2 (en) 2019-02-21
AU2018318313B2 (en) 2025-01-23

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