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WO2018237369A3 - Administration médiée par des nanoparticules lipidiques (lnp) d'un adn plasmidique exprimant crispr pour le traitement d'une infection chronique par le virus de l'hépatite b - Google Patents

Administration médiée par des nanoparticules lipidiques (lnp) d'un adn plasmidique exprimant crispr pour le traitement d'une infection chronique par le virus de l'hépatite b Download PDF

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Publication number
WO2018237369A3
WO2018237369A3 PCT/US2018/039179 US2018039179W WO2018237369A3 WO 2018237369 A3 WO2018237369 A3 WO 2018237369A3 US 2018039179 W US2018039179 W US 2018039179W WO 2018237369 A3 WO2018237369 A3 WO 2018237369A3
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Prior art keywords
plasmid dna
crispr
expressing plasmid
lnp
virus infection
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PCT/US2018/039179
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English (en)
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WO2018237369A2 (fr
Inventor
Sean M. Sullivan
Larry Smith
Brenda CLEMENTE
Qun Wei
Ming Ye
Yasuhiro Hayashi
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Vical Incorporated
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Application filed by Vical Incorporated filed Critical Vical Incorporated
Publication of WO2018237369A2 publication Critical patent/WO2018237369A2/fr
Publication of WO2018237369A3 publication Critical patent/WO2018237369A3/fr

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/70—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving virus or bacteriophage
    • C12Q1/701—Specific hybridization probes
    • C12Q1/706—Specific hybridization probes for hepatitis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/16—Amides, e.g. hydroxamic acids
    • A61K31/165—Amides, e.g. hydroxamic acids having aromatic rings, e.g. colchicine, atenolol, progabide
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • C12Q1/6886—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material for cancer
    • 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
    • C12N2730/00—Reverse transcribing DNA viruses
    • C12N2730/00011—Details
    • C12N2730/10011—Hepadnaviridae
    • C12N2730/10111—Orthohepadnavirus, e.g. hepatitis B virus
    • C12N2730/10122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Analytical Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Pathology (AREA)
  • Communicable Diseases (AREA)
  • Veterinary Medicine (AREA)
  • Oncology (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Hospice & Palliative Care (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biomedical Technology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

L'invention concerne un ADN plasmidique exprimant CRISPR et un système d'administration de gène ciblé sur des hépatocytes infectés par le virus de l'hépatite B. L'invention concerne également des procédés d'orientation de la formation d'un système CRISPR dans ces cellules pour s'assurer d'une spécificité améliorée de la reconnaissance de cible afin de modifier, améliorer ou traiter l'infection chronique par le VHB. En outre, la teneur en CpG de l'ADN plasmidique d'expression a été réduite au minimum pour réduire l'inflammation et maximiser l'expression génique.
PCT/US2018/039179 2017-06-23 2018-06-23 Administration médiée par des nanoparticules lipidiques (lnp) d'un adn plasmidique exprimant crispr pour le traitement d'une infection chronique par le virus de l'hépatite b WO2018237369A2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762524389P 2017-06-23 2017-06-23
US62/524,389 2017-06-23

Publications (2)

Publication Number Publication Date
WO2018237369A2 WO2018237369A2 (fr) 2018-12-27
WO2018237369A3 true WO2018237369A3 (fr) 2019-02-14

Family

ID=64735812

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2018/039179 WO2018237369A2 (fr) 2017-06-23 2018-06-23 Administration médiée par des nanoparticules lipidiques (lnp) d'un adn plasmidique exprimant crispr pour le traitement d'une infection chronique par le virus de l'hépatite b

Country Status (1)

Country Link
WO (1) WO2018237369A2 (fr)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB2595106B (en) * 2019-01-22 2022-10-12 Emulate Inc High-content imaging of microfluidic devices
CA3132164A1 (fr) * 2019-03-06 2020-09-10 Generation Bio Co. Adn a extremite fermee (cedna) et composes de modulation immunitaire
JP7650242B2 (ja) 2019-04-18 2025-03-24 ツールゲン インコーポレイテッド Ｂ型肝炎ウイルスの増殖を抑制する組成物及びその方法
BR112021021979A2 (pt) 2019-05-03 2022-12-06 Specific Biologics Inc Polipeptídeo, nucleases quiméricas, formulações farmaceuticamente aceitáveis, métodos para editar dna genômico, para deletar comprimentos definidos de uma molécula de dna e para substituir sequências selecionadas de uma molécula de dna, processo, métodos de administração da formulação, de tratamento de uma doença relacionada ao pulmão e de tratamento de doenças monogenéticas e infecciosas, ligante e moléculas de dna doadoras modificadas
CA3150452A1 (fr) * 2019-09-06 2021-03-11 Generation Bio Co. Compositions de nanoparticules lipidiques comprenant de l'adn a extremites fermees et des lipides clivables et leurs procedes d'utilisation
US20230090515A1 (en) * 2019-12-20 2023-03-23 Curevac Ag Lipid nanoparticles for delivery of nucleic acids
EP4308087A1 (fr) * 2021-03-17 2024-01-24 Board of Regents of the University of Nebraska Formulations de nanoparticules lipidiques et leurs méthodes d'utilisation
CN113368261A (zh) * 2021-06-17 2021-09-10 苏州大学 一种非病毒载体及其制备方法与应用
CN114903987A (zh) * 2022-05-31 2022-08-16 复旦大学 一种用于治疗慢性乙型肝炎病毒感染的mRNA药物及其制备方法和应用
WO2024186890A1 (fr) * 2023-03-06 2024-09-12 Intellia Therapeutics, Inc. Compositions et méthodes d'édition du génome du virus de l'hépatite b (vhb)

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