JP2022520232A - ダノン病の治療のための遺伝子療法ベクター - Google Patents

ダノン病の治療のための遺伝子療法ベクター Download PDF

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JP2022520232A
JP2022520232A JP2021546891A JP2021546891A JP2022520232A JP 2022520232 A JP2022520232 A JP 2022520232A JP 2021546891 A JP2021546891 A JP 2021546891A JP 2021546891 A JP2021546891 A JP 2021546891A JP 2022520232 A JP2022520232 A JP 2022520232A
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gene therapy
therapy vector
raav
lamp
raav gene
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JP2022520232A5 (OSRAM
Inventor
アナヒタ ケラヴァラ
ラジ プラバカール
ガウラブ シャー
ロデリック ウォン
ナヴィーン ヤラマンチ
ピラチプ プラトゥムスワン
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スペースクラフト セブン リミテッド ライアビリティ カンパニー
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Publication of JP2022520232A publication Critical patent/JP2022520232A/ja
Publication of JP2022520232A5 publication Critical patent/JP2022520232A5/ja
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    • 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
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • 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
    • A61P9/06—Antiarrhythmics
    • 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
    • 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
    • C07K14/70596—Molecules with a "CD"-designation not provided for elsewhere
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/42—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a HA(hemagglutinin)-tag
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/43—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a FLAG-tag
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021546891A 2019-02-12 2020-02-12 ダノン病の治療のための遺伝子療法ベクター Pending JP2022520232A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025007633A JP2025063228A (ja) 2019-02-12 2025-01-20 ダノン病の治療のための遺伝子療法ベクター

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962804521P 2019-02-12 2019-02-12
US62/804,521 2019-02-12
US201962934928P 2019-11-13 2019-11-13
US62/934,928 2019-11-13
PCT/US2020/017987 WO2020167996A1 (en) 2019-02-12 2020-02-12 Gene therapy vectors for treatment of danon disease

Related Child Applications (1)

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JP2025007633A Division JP2025063228A (ja) 2019-02-12 2025-01-20 ダノン病の治療のための遺伝子療法ベクター

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JP2022520232A true JP2022520232A (ja) 2022-03-29
JP2022520232A5 JP2022520232A5 (OSRAM) 2023-02-21

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JP2021546891A Pending JP2022520232A (ja) 2019-02-12 2020-02-12 ダノン病の治療のための遺伝子療法ベクター
JP2025007633A Pending JP2025063228A (ja) 2019-02-12 2025-01-20 ダノン病の治療のための遺伝子療法ベクター

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US (1) US20220143215A1 (OSRAM)
EP (1) EP3924371A4 (OSRAM)
JP (2) JP2022520232A (OSRAM)
KR (1) KR20210125999A (OSRAM)
CN (2) CN120324641A (OSRAM)
AU (3) AU2020221842A1 (OSRAM)
BR (1) BR112021015751A2 (OSRAM)
CA (1) CA3128514A1 (OSRAM)
IL (1) IL285238A (OSRAM)
MX (1) MX2021009696A (OSRAM)
SG (1) SG11202107744SA (OSRAM)
WO (1) WO2020167996A1 (OSRAM)

Families Citing this family (11)

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CN109641063B (zh) 2016-04-20 2022-11-15 能源环境和技术研究中心O.A., M.P. 用于增强pklr的基因表达的组合物和方法
SG11202002263TA (en) 2017-10-16 2020-04-29 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
JP7576754B2 (ja) 2018-04-11 2024-11-01 スペースクラフト セブン リミテッド ライアビリティ カンパニー 幹細胞移植のための組成物および方法
AU2019261646A1 (en) 2018-04-27 2020-11-12 Spacecraft Seven, Llc Gene therapy for CNS degeneration
WO2020014523A1 (en) * 2018-07-12 2020-01-16 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
IL300263A (en) * 2020-08-07 2023-03-01 Spacecraft Seven Llc Plakophilin-2 (PKP2) gene therapy using an AAV vector
KR20230129431A (ko) * 2020-12-07 2023-09-08 스페이스크래프트 세븐, 엘엘씨 다농병의 치료
CN114874990A (zh) * 2021-02-05 2022-08-09 中国科学院苏州纳米技术与纳米仿生研究所 一种功能化外泌体及其制备方法和应用
JP7564368B2 (ja) 2021-09-23 2024-10-08 エルジー エナジー ソリューション リミテッド 高耐熱性コネクター及びこれを含むバッテリーモジュール、バッテリーパック、自動車
CN114457112A (zh) * 2022-02-07 2022-05-10 苏州市立医院 一种特异性神经靶向mr分子探针及其制备方法和应用
CN115975051B (zh) * 2022-11-17 2025-09-12 东南大学 靶向外泌体及制备方法、应用、药物和药物递送系统

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WO2013078316A1 (en) * 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
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
JP2021531752A (ja) * 2018-07-12 2021-11-25 ロケット ファーマシューティカルズ リミテッド ダノン病治療用遺伝子療法ベクター

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US9217155B2 (en) * 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
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WO2017136536A1 (en) * 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
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WO2013078316A1 (en) * 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
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
JP2021531752A (ja) * 2018-07-12 2021-11-25 ロケット ファーマシューティカルズ リミテッド ダノン病治療用遺伝子療法ベクター

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Publication number Publication date
BR112021015751A2 (pt) 2022-02-08
MX2021009696A (es) 2021-09-23
EP3924371A4 (en) 2023-01-11
KR20210125999A (ko) 2021-10-19
AU2023201237A1 (en) 2023-04-13
SG11202107744SA (en) 2021-08-30
US20220143215A1 (en) 2022-05-12
EP3924371A1 (en) 2021-12-22
CN113508130A (zh) 2021-10-15
JP2025063228A (ja) 2025-04-15
CA3128514A1 (en) 2020-08-20
AU2020221842A1 (en) 2021-08-12
WO2020167996A1 (en) 2020-08-20
AU2023201237B2 (en) 2025-04-10
IL285238A (en) 2021-09-30
CN120324641A (zh) 2025-07-18
AU2025205318A1 (en) 2025-07-31

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