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WO2018237369A3 - Lipid nanoparticle (lnp)-mediated delivery of a crispr-expressing plasmid dna for treating chronic hepatitis b virus infection - Google Patents

Lipid nanoparticle (lnp)-mediated delivery of a crispr-expressing plasmid dna for treating chronic hepatitis b virus infection 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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WO
WIPO (PCT)
Prior art keywords
plasmid dna
crispr
expressing plasmid
lnp
virus infection
Prior art date
Application number
PCT/US2018/039179
Other languages
French (fr)
Other versions
WO2018237369A2 (en
Inventor
Sean M. Sullivan
Larry Smith
Brenda CLEMENTE
Qun Wei
Ming Ye
Yasuhiro Hayashi
Original Assignee
Vical Incorporated
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 Vical Incorporated filed Critical Vical Incorporated
Publication of WO2018237369A2 publication Critical patent/WO2018237369A2/en
Publication of WO2018237369A3 publication Critical patent/WO2018237369A3/en

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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

Provided herein is a CRISPR-expressing plasmid DNA and a gene delivery system targeted to hepatocytes infected with hepatitis B virus. Also provided are methods of directing CRISPR system formation in such cells to ensure enhanced specificity for target recognition to alter, improve, or treat chronic HBV infection. In addition the CpG content of the expressing plasmid DNA has been minimized to reduce inflammation and maximize gene expression.
PCT/US2018/039179 2017-06-23 2018-06-23 Lipid nanoparticle (lnp)-mediated delivery of a crispr-expressing plasmid dna for treating chronic hepatitis b virus infection WO2018237369A2 (en)

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 (en) 2018-12-27
WO2018237369A3 true WO2018237369A3 (en) 2019-02-14

Family

ID=64735812

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2018/039179 WO2018237369A2 (en) 2017-06-23 2018-06-23 Lipid nanoparticle (lnp)-mediated delivery of a crispr-expressing plasmid dna for treating chronic hepatitis b virus infection

Country Status (1)

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WO (1) WO2018237369A2 (en)

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 (en) * 2019-03-06 2020-09-10 Generation Bio Co. Closed-ended dna (cedna) and immune modulating compounds
JP7650242B2 (en) 2019-04-18 2025-03-24 ツールゲン インコーポレイテッド Composition and method for inhibiting proliferation of hepatitis B virus
BR112021021979A2 (en) 2019-05-03 2022-12-06 Specific Biologics Inc POLYPEPTIDES, CHIMERIC NUCLEASES, PHARMACEUTICALLY ACCEPTABLE FORMULATIONS, METHODS FOR EDITING GENOMIC DNA, DELETING DEFINED LENGTHS OF A DNA MOLECULE AND REPLACING SELECTED SEQUENCES OF A DNA MOLECULE, PROCESS, METHODS OF ADMINISTRATION OF THE FORMULATION, OF TREATMENT OF A DISEASE DISEASE LUNG AND TREATMENT OF MONOGENETIC AND INFECTIOUS DISEASES, LIGAND AND MODIFIED DONOR DNA MOLECULES
CA3150452A1 (en) * 2019-09-06 2021-03-11 Generation Bio Co. Lipid nanoparticle compositions comprising closed-ended dna and cleavable lipids and methods of use thereof
US20230090515A1 (en) * 2019-12-20 2023-03-23 Curevac Ag Lipid nanoparticles for delivery of nucleic acids
EP4308087A1 (en) * 2021-03-17 2024-01-24 Board of Regents of the University of Nebraska Lipid nanoparticle formulations and methods of use thereof
CN113368261A (en) * 2021-06-17 2021-09-10 苏州大学 Non-viral vector and preparation method and application thereof
CN114903987A (en) * 2022-05-31 2022-08-16 复旦大学 A kind of mRNA medicine for treating chronic hepatitis B virus infection and its preparation method and application
WO2024186890A1 (en) * 2023-03-06 2024-09-12 Intellia Therapeutics, Inc. Compositions and methods for hepatitis b virus (hbv) genome editing

Citations (7)

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US20100159585A1 (en) * 2006-09-08 2010-06-24 Ambrx, Inc. Suppressor tRNA Transcription in Vertebrate Cells
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
CN106520824A (en) * 2016-09-30 2017-03-22 北京大北农科技集团股份有限公司 Multi-target-point editing system and application thereof
WO2017096237A1 (en) * 2015-12-02 2017-06-08 Ceres, Inc. Methods for genetic modification of plants

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US20100159585A1 (en) * 2006-09-08 2010-06-24 Ambrx, Inc. Suppressor tRNA Transcription in Vertebrate Cells
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 (en) * 2015-12-02 2017-06-08 Ceres, Inc. Methods for genetic modification of plants
CN106520824A (en) * 2016-09-30 2017-03-22 北京大北农科技集团股份有限公司 Multi-target-point editing system and application thereof

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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