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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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WIPO (PCT)
Prior art keywords
plasmid dna
crispr
expressing plasmid
lnp
virus infection
Prior art date
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Ceased
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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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Fresh Tracks Therapeutics Inc
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Vical Inc
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Publication date
Application filed by Vical Inc filed Critical Vical Inc
Publication of WO2018237369A2 publication Critical patent/WO2018237369A2/fr
Publication of WO2018237369A3 publication Critical patent/WO2018237369A3/fr
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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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)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Analytical Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Oncology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Pathology (AREA)
  • Communicable Diseases (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Hospice & Palliative Care (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (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 Ceased 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

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Family Applications (1)

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PCT/US2018/039179 Ceased 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 (12)

* 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
JP2022523806A (ja) * 2019-03-06 2022-04-26 ジェネレーション バイオ カンパニー 閉端ｄｎａ（ｃｅｄｎａ）および免疫調節化合物
US20230293645A1 (en) * 2019-04-18 2023-09-21 Toolgen Incorporated Composition and method for inhibiting proliferation hepatitis b virus
AU2020268959A1 (en) * 2019-05-03 2021-12-09 Specific Biologics Inc. Lipid-encapsulated dual-cleaving endonuclease for DNA and gene editing
AU2020342668B2 (en) * 2019-09-06 2025-10-09 Generation Bio Co. Lipid nanoparticle compositions comprising closed-ended DNA and cleavable lipids and methods of use thereof
JP2023502576A (ja) * 2019-11-22 2023-01-25 ジェネレーション バイオ カンパニー イオン化可能な脂質およびそれらのナノ粒子組成物
AU2020407285A1 (en) * 2019-12-20 2022-08-11 CureVac SE Lipid nanoparticles for delivery of nucleic acids
EP4308087A4 (fr) * 2021-03-17 2025-06-18 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
TW202436622A (zh) * 2023-03-06 2024-09-16 美商英特利亞醫療公司 用於ｂ型肝炎病毒（ｈｂｖ）基因體編輯之組合物及方法
WO2025049606A1 (fr) * 2023-08-29 2025-03-06 Earli Inc. Compositions d'administration d'acides nucléiques

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US20110105593A1 (en) * 2006-10-11 2011-05-05 Drexel University MicroRNA-Formatted Multitarget Interfering RNA Vector Constructs and Methods of Using The Same
US20140271550A1 (en) * 2013-03-14 2014-09-18 The Trustees Of The University Of Pennsylvania Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses
US20160317677A1 (en) * 2013-12-12 2016-11-03 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hbv and viral diseases and disorders
US20170049909A1 (en) * 2014-02-18 2017-02-23 Duke University Compositions for the inactivation of virus replication and methods of making and using the same
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US20110105593A1 (en) * 2006-10-11 2011-05-05 Drexel University MicroRNA-Formatted Multitarget Interfering RNA Vector Constructs and Methods of Using The Same
US20140271550A1 (en) * 2013-03-14 2014-09-18 The Trustees Of The University Of Pennsylvania Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses
US20160317677A1 (en) * 2013-12-12 2016-11-03 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hbv and viral diseases and disorders
US20170049909A1 (en) * 2014-02-18 2017-02-23 Duke University Compositions for the inactivation of virus replication and methods of making and using the same
WO2017096237A1 (fr) * 2015-12-02 2017-06-08 Ceres, Inc. Procédés de modification génétique de plantes
CN106520824A (zh) * 2016-09-30 2017-03-22 北京大北农科技集团股份有限公司 多靶点编辑系统及其用途

Non-Patent Citations (3)

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WANG ET AL.: "The gRNA-miRNA-gRNA Ternary Cassette Combining CRISPR/Cas9 with RNAi Approach Strongly Inhibits Hepatitis B Virus Replication", THERANOSTICS, vol. 7, no. 12, 22 July 2017 (2017-07-22), pages 3090 - 3105, XP055482499, DOI: doi:10.7150/thno.18114 *

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Publication number Publication date
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