CA3088897A1 - Immuno-evasive vectors and use for gene therapy - Google Patents

Immuno-evasive vectors and use for gene therapy Download PDF

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Publication number
CA3088897A1
CA3088897A1 CA3088897A CA3088897A CA3088897A1 CA 3088897 A1 CA3088897 A1 CA 3088897A1 CA 3088897 A CA3088897 A CA 3088897A CA 3088897 A CA3088897 A CA 3088897A CA 3088897 A1 CA3088897 A1 CA 3088897A1
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Canada
Prior art keywords
enveloped
vector
viral vector
aav
viral
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Legal status (The legal status 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 status listed.)
Pending
Application number
CA3088897A
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English (en)
French (fr)
Inventor
Genine WINSLOW
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Chameleon Biosciences Inc
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Chameleon Biosciences Inc
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Filing date
Publication date
Application filed by Chameleon Biosciences Inc filed Critical Chameleon Biosciences Inc
Publication of CA3088897A1 publication Critical patent/CA3088897A1/en
Pending legal-status Critical Current

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    • 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
    • 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
    • A61K48/0083—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 administration regime
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C07K14/70521—CD28, CD152
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C07K14/70532—B7 molecules, e.g. CD80, CD86
    • 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
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/00051—Methods of production or purification of viral material
    • C12N2750/00052—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Immunology (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Virology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Hematology (AREA)
  • Mycology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CA3088897A 2018-01-11 2019-01-11 Immuno-evasive vectors and use for gene therapy Pending CA3088897A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862616167P 2018-01-11 2018-01-11
US62/616,167 2018-01-11
US201862768779P 2018-11-16 2018-11-16
US62/768,779 2018-11-16
PCT/US2019/013361 WO2019140311A1 (en) 2018-01-11 2019-01-11 Immuno-evasive vectors and use for gene therapy

Publications (1)

Publication Number Publication Date
CA3088897A1 true CA3088897A1 (en) 2019-07-18

Family

ID=65444323

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3088897A Pending CA3088897A1 (en) 2018-01-11 2019-01-11 Immuno-evasive vectors and use for gene therapy

Country Status (14)

Country Link
US (1) US20200338216A1 (https=)
EP (1) EP3737768A1 (https=)
JP (2) JP7406253B2 (https=)
KR (1) KR20200128519A (https=)
CN (1) CN111836896A (https=)
AU (1) AU2019206639A1 (https=)
BR (1) BR112020014093A2 (https=)
CA (1) CA3088897A1 (https=)
CL (1) CL2020001850A1 (https=)
IL (1) IL275951A (https=)
MX (1) MX2020007390A (https=)
RU (1) RU2020126574A (https=)
SG (1) SG11202006298XA (https=)
WO (1) WO2019140311A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4118219A4 (en) * 2020-03-11 2024-04-17 Shanghai Belief-Delivery Biomed Co., Ltd. Novel use of aspirin compound in increasing nucleic acid expression
US20230355803A1 (en) * 2020-06-24 2023-11-09 Chameleon Biosciences, Inc. Extracellular vesicles with immune modulators
CN112410304A (zh) * 2020-11-12 2021-02-26 天津大学 一种基因修饰的外泌体及其制备方法和应用
WO2023028035A1 (en) * 2021-08-23 2023-03-02 University Of Florida Research Foundation, Incorporated Lipid enveloped recombinant aav particles for gene therapy use
EP4660308A1 (en) * 2023-01-31 2025-12-10 Synplogen Co., Ltd. Helper gene

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU8672198A (en) 1997-07-31 1999-02-22 Chiron Corporation Method enabling readministration of aav vector via immunosuppression of host
WO1999055893A1 (en) 1998-04-29 1999-11-04 University Of Southern California Retroviral vectors including modified envelope escort proteins
DE19827457C1 (de) * 1998-06-19 2000-03-02 Medigene Ag Strukturprotein von AAV, seine Herstellung und Verwendung
EP1769078A1 (en) * 2004-07-01 2007-04-04 VIRxSYS Corporation Vector packaging cell line
WO2006138670A2 (en) 2005-06-16 2006-12-28 Virxsys Corporation Antibody complexes
JP4691611B1 (ja) 2010-01-15 2011-06-01 富士フイルム株式会社 有機電界発光素子
WO2011106376A2 (en) * 2010-02-23 2011-09-01 The General Hospital Corporation Use of microvesicles in the treatment of medical conditions
JP7242180B2 (ja) * 2014-09-07 2023-03-20 セレクタ バイオサイエンシーズ インコーポレーテッド 抗ウイルス導入ベクター免疫応答を減弱化するための方法および組成物
CN104887717B (zh) * 2015-06-04 2019-05-31 爱康得生物医学技术（苏州）有限公司 一种免疫增强试剂
WO2017136764A1 (en) * 2016-02-05 2017-08-10 The General Hospital Corporation Hybrid system for efficient gene delivery to cells of the inner ear

Also Published As

Publication number Publication date
KR20200128519A (ko) 2020-11-13
US20200338216A1 (en) 2020-10-29
JP2021510522A (ja) 2021-04-30
WO2019140311A1 (en) 2019-07-18
BR112020014093A2 (pt) 2020-12-01
EP3737768A1 (en) 2020-11-18
MX2020007390A (es) 2020-10-14
JP7406253B2 (ja) 2023-12-27
AU2019206639A1 (en) 2020-08-20
IL275951A (en) 2020-08-31
JP2023160938A (ja) 2023-11-02
CL2020001850A1 (es) 2020-12-04
SG11202006298XA (en) 2020-07-29
RU2020126574A (ru) 2022-02-11
CN111836896A (zh) 2020-10-27

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