BR112020003403A2 - enzimas de dnase engenheiradas e uso na terapia - Google Patents

enzimas de dnase engenheiradas e uso na terapia Download PDF

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Publication number
BR112020003403A2
BR112020003403A2 BR112020003403-7A BR112020003403A BR112020003403A2 BR 112020003403 A2 BR112020003403 A2 BR 112020003403A2 BR 112020003403 A BR112020003403 A BR 112020003403A BR 112020003403 A2 BR112020003403 A2 BR 112020003403A2
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BR
Brazil
Prior art keywords
seq
enzyme
fact
variant
dnase
Prior art date
Application number
BR112020003403-7A
Other languages
English (en)
Portuguese (pt)
Inventor
Tobias A. Fuchs
Miguel JIMÉNEZ-ALCÁZAR
Josephine GÖBEL
Hanna ENGLERT
Original Assignee
Neutrolis 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 Neutrolis Inc. filed Critical Neutrolis Inc.
Publication of BR112020003403A2 publication Critical patent/BR112020003403A2/pt

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • A61K48/005—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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P41/00—Drugs used in surgical methods, e.g. surgery adjuvants for preventing adhesion or for vitreum substitution
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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
    • 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
    • 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
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • 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)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/21—Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)
    • C12Y301/21001—Deoxyribonuclease I (3.1.21.1)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/31—Fusion polypeptide fusions, other than Fc, for prolonged plasma life, e.g. albumin
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/90—Fusion polypeptide containing a motif for post-translational modification
    • C07K2319/91—Fusion polypeptide containing a motif for post-translational modification containing a motif for glycosylation

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Cardiology (AREA)
  • Urology & Nephrology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Surgery (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
BR112020003403-7A 2017-08-18 2018-08-20 enzimas de dnase engenheiradas e uso na terapia BR112020003403A2 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762547220P 2017-08-18 2017-08-18
US62/547,220 2017-08-18
US201762611166P 2017-12-28 2017-12-28
US62/611,166 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
BR112020003403A2 true BR112020003403A2 (pt) 2020-08-25

Family

ID=65362608

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112020003403-7A BR112020003403A2 (pt) 2017-08-18 2018-08-20 enzimas de dnase engenheiradas e uso na terapia

Country Status (15)

Country Link
US (5) US10696956B2 (https=)
EP (2) EP3668972B8 (https=)
JP (1) JP7577314B2 (https=)
KR (2) KR20250073498A (https=)
CN (1) CN111566207A (https=)
AU (1) AU2018318313B2 (https=)
BR (1) BR112020003403A2 (https=)
CA (1) CA3073317A1 (https=)
DK (1) DK3668972T3 (https=)
ES (1) ES3008982T3 (https=)
IL (2) IL272715B2 (https=)
MX (1) MX2020001913A (https=)
SG (1) SG11202001445XA (https=)
UA (1) UA129696C2 (https=)
WO (1) WO2019036719A2 (https=)

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DK3668972T3 (da) 2017-08-18 2025-01-13 Neutrolis Inc Konstruerede dnase-enzymer og anvendelse i terapi
AU2019209770B2 (en) 2018-01-16 2025-07-31 Cls Therapeutics Limited Treatment of diseases by liver expression of an enzyme which has a deoxyribonuclease (DNase) activity
US11058724B2 (en) 2018-10-08 2021-07-13 Neutrolis, Inc. Methods of using DNASE1-like 3 in therapy
IL325846A (en) * 2018-10-08 2026-03-01 Neutrolis Inc Engineering deoxyribonuclease enzymes for manufacturing and medicine
US10988746B2 (en) 2018-10-08 2021-04-27 Neutrolis, Inc. Manufacturing and engineering of DNASE enzymes for therapy
WO2020163264A1 (en) * 2019-02-04 2020-08-13 Neutrolis, Inc. Engineered human extracellular dnase enzymes for drug candidate selection
US11352613B2 (en) 2019-02-04 2022-06-07 Neutrolis, Inc. Engineered human extracellular DNASE enzymes
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
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WO2021168413A1 (en) * 2020-02-20 2021-08-26 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
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EP4161645A4 (en) * 2020-06-08 2023-11-01 Yale University COMPOSITIONS AND METHODS FOR TREATING AND/OR PREVENTING COAGULOPATHY AND/OR SEPSIS IN PATIENTS WITH BACTERIAL AND/OR VIRAL INFECTIONS
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WO2025076525A1 (en) * 2023-10-06 2025-04-10 Neutrolis, Inc. Biomarker and precision therapy for inflammatory disease

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Publication number Publication date
AU2018318313B2 (en) 2025-01-23
MX2020001913A (es) 2020-09-07
KR20250073498A (ko) 2025-05-27
IL317968A (en) 2025-02-01
US20210277372A1 (en) 2021-09-09
WO2019036719A3 (en) 2019-03-28
IL272715A (en) 2020-04-30
CN111566207A (zh) 2020-08-21
EP3668972B1 (en) 2024-10-09
DK3668972T3 (da) 2025-01-13
KR20200047588A (ko) 2020-05-07
EP4509135A2 (en) 2025-02-19
KR102806169B1 (ko) 2025-05-13
IL272715B1 (en) 2025-01-01
US20200347370A1 (en) 2020-11-05
WO2019036719A2 (en) 2019-02-21
EP3668972B8 (en) 2024-11-13
SG11202001445XA (en) 2020-03-30
ES3008982T3 (en) 2025-03-25
US20200399622A1 (en) 2020-12-24
CA3073317A1 (en) 2019-02-21
JP2020534865A (ja) 2020-12-03
EP4509135A3 (en) 2025-05-07
EP3668972A2 (en) 2020-06-24
EP3668972A4 (en) 2021-04-21
US11046943B2 (en) 2021-06-29
IL272715B2 (en) 2025-05-01
US10696956B2 (en) 2020-06-30
AU2018318313A1 (en) 2020-03-19
US20200024585A1 (en) 2020-01-23
JP7577314B2 (ja) 2024-11-05
US11046942B2 (en) 2021-06-29
UA129696C2 (uk) 2025-07-09
US20210040461A1 (en) 2021-02-11
US10801019B1 (en) 2020-10-13

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