JP2021526818A - 心筋症のためのａａｖ心臓遺伝子治療 - Google Patents

心筋症のためのａａｖ心臓遺伝子治療 Download PDF

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Publication number
JP2021526818A
JP2021526818A JP2020568431A JP2020568431A JP2021526818A JP 2021526818 A JP2021526818 A JP 2021526818A JP 2020568431 A JP2020568431 A JP 2020568431A JP 2020568431 A JP2020568431 A JP 2020568431A JP 2021526818 A JP2021526818 A JP 2021526818A
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Prior art keywords
raav
heart
gene
aav
cardiac
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JP2020568431A
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Japanese (ja)
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JP2021526818A5 (https=
JPWO2019237067A5 (https=
Inventor
スウィーニー，フー，リー
スリーパー，マーガレット，マリー
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University of Florida Research Foundation Inc
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University of Florida Research Foundation Inc
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Priority to JP2024062211A priority Critical patent/JP2024097779A/ja
Publication of JP2021526818A5 publication Critical patent/JP2021526818A5/ja
Publication of JPWO2019237067A5 publication Critical patent/JPWO2019237067A5/ja
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    • 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
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0375—Animal model for cardiovascular diseases
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Cardiology (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hospice & Palliative Care (AREA)
  • Heart & Thoracic Surgery (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)
JP2020568431A 2018-06-08 2019-06-07 心筋症のためのａａｖ心臓遺伝子治療 Pending JP2021526818A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024062211A JP2024097779A (ja) 2018-06-08 2024-04-08 心筋症のためのａａｖ心臓遺伝子治療

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862682772P 2018-06-08 2018-06-08
US62/682,772 2018-06-08
US201962822015P 2019-03-21 2019-03-21
US62/822,015 2019-03-21
PCT/US2019/036157 WO2019237067A1 (en) 2018-06-08 2019-06-07 Aav cardiac gene therapy for cardiomyopathy

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024062211A Division JP2024097779A (ja) 2018-06-08 2024-04-08 心筋症のためのａａｖ心臓遺伝子治療

Publications (3)

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JP2021526818A true JP2021526818A (ja) 2021-10-11
JP2021526818A5 JP2021526818A5 (https=) 2024-05-15
JPWO2019237067A5 JPWO2019237067A5 (https=) 2024-05-15

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JP2020568431A Pending JP2021526818A (ja) 2018-06-08 2019-06-07 心筋症のためのａａｖ心臓遺伝子治療
JP2024062211A Pending JP2024097779A (ja) 2018-06-08 2024-04-08 心筋症のためのａａｖ心臓遺伝子治療

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Country Link
US (1) US20210260215A1 (https=)
EP (1) EP3814512A4 (https=)
JP (2) JP2021526818A (https=)
KR (1) KR20210018902A (https=)
CN (1) CN112272705A (https=)
AU (1) AU2019282822B2 (https=)
BR (1) BR112020024935A2 (https=)
CA (1) CA3100280A1 (https=)
CL (1) CL2020003190A1 (https=)
CO (1) CO2020016718A2 (https=)
IL (1) IL279225B2 (https=)
MX (1) MX421375B (https=)
SG (1) SG11202011061SA (https=)
WO (1) WO2019237067A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112022000898A2 (pt) * 2019-07-19 2022-06-07 Univ Florida Terapia de gene cardíaco aav para cardiomiopatia em seres humanos
BR112023001336A2 (pt) * 2020-08-05 2023-02-14 Spacecraft Seven Llc Terapia gênica de csrp3 (proteína 3 rica em cisteína e glicina)
MX2023007602A (es) * 2020-12-23 2023-09-21 Univ Florida Mayor eficiencia del empaquetamiento de vectores para terapia genica cardiaca.
CA3252359A1 (en) * 2022-03-02 2023-09-07 Cevec Pharmaceuticals Gmbh IMPROVED CELL LINES AND PROCESSES FOR THE PRODUCTION OF ADENO-ASSOCIATED VECTORS
EP4239063A1 (en) * 2022-03-02 2023-09-06 CEVEC Pharmaceuticals GmbH Improved cell lines and methods for the production of adeno-associated vectors

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016521708A (ja) * 2013-06-07 2016-07-25 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 環状アデノシン一リン酸−インコンピテントアデニリルシクラーゼならびに心不全を処置するおよび心機能を増大させるための組成物および方法
WO2017127565A1 (en) * 2016-01-19 2017-07-27 The Regents Of The University Of California Methods for the treatment of danon disease and other disorders of autophagy

Family Cites Families (5)

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US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
US20170348387A1 (en) * 2016-02-29 2017-12-07 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for nphp5 lca-ciliopathy

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016521708A (ja) * 2013-06-07 2016-07-25 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 環状アデノシン一リン酸−インコンピテントアデニリルシクラーゼならびに心不全を処置するおよび心機能を増大させるための組成物および方法
WO2017127565A1 (en) * 2016-01-19 2017-07-27 The Regents Of The University Of California Methods for the treatment of danon disease and other disorders of autophagy

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
FRONTIERS IN MOLECULAR NEUROSCIENCE, 2015, VOL.8, ARTICLE 36, JPN6023021214, ISSN: 0005214807 *
SCIENCE TRANSLATIONAL MEDICINE, 2011, VOL.3, NO.92, 92RA64, JPN6023021212, ISSN: 0005214805 *
THE JOURNAL OF THORACIC AND CARDIOVASCULAR SURGERY, 2003, VOL.125, NO.6, PP.1461-1469, JPN6023021213, ISSN: 0005214806 *

Also Published As

Publication number Publication date
MX2020013313A (es) 2021-02-22
WO2019237067A1 (en) 2019-12-12
CO2020016718A2 (es) 2021-04-08
CL2020003190A1 (es) 2021-04-30
EP3814512A1 (en) 2021-05-05
EP3814512A4 (en) 2022-03-09
CA3100280A1 (en) 2019-12-12
JP2024097779A (ja) 2024-07-19
CN112272705A (zh) 2021-01-26
AU2019282822B2 (en) 2025-09-04
MX421375B (es) 2025-03-14
IL279225A (en) 2021-01-31
IL279225B1 (en) 2024-07-01
KR20210018902A (ko) 2021-02-18
US20210260215A1 (en) 2021-08-26
AU2019282822A1 (en) 2020-11-26
IL279225B2 (en) 2024-11-01
SG11202011061SA (en) 2020-12-30
BR112020024935A2 (pt) 2021-03-09

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