JP6646282B2 - 多発性硬化症のためのａａｖ系遺伝子療法 - Google Patents

多発性硬化症のためのａａｖ系遺伝子療法 Download PDF

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JP6646282B2
JP6646282B2 JP2016564019A JP2016564019A JP6646282B2 JP 6646282 B2 JP6646282 B2 JP 6646282B2 JP 2016564019 A JP2016564019 A JP 2016564019A JP 2016564019 A JP2016564019 A JP 2016564019A JP 6646282 B2 JP6646282 B2 JP 6646282B2
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nucleic acid
raav
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mice
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JP2017513500A (ja
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ブラッド・イー・ホフマン
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ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド
ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド
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    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/0005—Vertebrate antigens
    • A61K39/0008—Antigens related to auto-immune diseases; Preparations to induce self-tolerance
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/53—DNA (RNA) vaccination
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/57—Medicinal preparations containing antigens or antibodies characterised by the type of response, e.g. Th1, Th2
    • A61K2039/577—Medicinal preparations containing antigens or antibodies characterised by the type of response, e.g. Th1, Th2 tolerising response
    • 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)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
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  • Biotechnology (AREA)
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  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Rheumatology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Mycology (AREA)
  • Pain & Pain Management (AREA)
  • Neurology (AREA)
  • Neurosurgery (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
JP2016564019A 2014-04-24 2015-04-24 多発性硬化症のためのａａｖ系遺伝子療法 Active JP6646282B2 (ja)

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JP2019235107A JP7430898B2 (ja) 2014-04-24 2019-12-25 多発性硬化症のためのａａｖ系遺伝子療法

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US201461983924P 2014-04-24 2014-04-24
US61/983,924 2014-04-24
PCT/US2015/027598 WO2015164789A1 (en) 2014-04-24 2015-04-24 Aav-based gene therapy for multiple sclerosis

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JP2017513500A JP2017513500A (ja) 2017-06-01
JP2017513500A5 JP2017513500A5 (https=) 2018-07-26
JP6646282B2 true JP6646282B2 (ja) 2020-02-14

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JP2016564019A Active JP6646282B2 (ja) 2014-04-24 2015-04-24 多発性硬化症のためのａａｖ系遺伝子療法
JP2019235107A Active JP7430898B2 (ja) 2014-04-24 2019-12-25 多発性硬化症のためのａａｖ系遺伝子療法
JP2022031064A Pending JP2022081549A (ja) 2014-04-24 2022-03-01 多発性硬化症のためのａａｖ系遺伝子療法

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JP2022031064A Pending JP2022081549A (ja) 2014-04-24 2022-03-01 多発性硬化症のためのａａｖ系遺伝子療法

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US (3) US20170043036A1 (https=)
EP (2) EP3133923B1 (https=)
JP (3) JP6646282B2 (https=)
AU (2) AU2015249328B2 (https=)
CA (1) CA2945984C (https=)
CY (1) CY1123312T1 (https=)
DK (1) DK3133923T3 (https=)
ES (1) ES2814280T3 (https=)
HR (1) HRP20201342T1 (https=)
HU (1) HUE050681T2 (https=)
LT (1) LT3133923T (https=)
PL (1) PL3133923T3 (https=)
PT (1) PT3133923T (https=)
RS (1) RS60816B1 (https=)
SI (1) SI3133923T1 (https=)
SM (1) SMT202000453T1 (https=)
WO (1) WO2015164789A1 (https=)

Families Citing this family (8)

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JP7270946B2 (ja) * 2017-08-08 2023-05-11 国立大学法人浜松医科大学 自己免疫疾患の治療又は予防剤
BR112021003399A2 (pt) * 2018-08-24 2021-05-18 Spark Therapeutics, Inc. sequências de promotores otimizadas, construtos de expressão livres de íntron e métodos de uso
CA3142487A1 (en) * 2019-06-04 2020-12-10 Thomas Jefferson University Oligodendrocyte-derived extracellular vesicles for therapy of multiple sclerosis
WO2021211614A1 (en) * 2020-04-14 2021-10-21 University Of Florida Research Foundation, Incorporated Aav-based gene therapies for treatment of autoimmune diseases
WO2021211640A1 (en) * 2020-04-14 2021-10-21 University Of Florida Research Foundation, Incorporated Enhanced effects of gene-immunotherapy and immunosuppressants in multiple sclerosis
JP2023532923A (ja) * 2020-06-30 2023-08-01 チルドレンズ ナショナル メディカル センター 骨格筋線維の修復を改善するためのリコンビナントヒト酸性スフィンゴミエリナーゼの使用
WO2024081570A1 (en) * 2022-10-10 2024-04-18 Eli Lilly And Company Methods and systems for using causal networks to develop models for evaluating biological processes
WO2024258925A1 (en) 2023-06-12 2024-12-19 Children's Hospital Medical Center Aav-cftr vectors and methods of using same

Family Cites Families (13)

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KR960705038A (ko) * 1993-09-03 1996-10-09 스테시 엘. 채닝 자기 면역 질환에 관련된 프로토콜에서 미엘린 올리고덴드로시트 당 단백질 및 그의 펩타이드 부분들의 용도(users of myelin oligodendrocyte glycoprotein and peptide portions thereof in protocols related to autoimmune desease)
AU5588298A (en) * 1996-12-02 1998-06-29 Cell Genesys, Inc. Adeno-associated viral vector-mediated delivery of dna to cells of the liver
KR100449454B1 (ko) * 2000-10-02 2004-09-21 이현철 단일사슬 인슐린 유도체의 유전자를 포함하는 당뇨병치료용 벡터
EP1193272B1 (en) * 2000-10-02 2004-06-30 Yonsei University Single-chain insulin analogs
CA2361462A1 (en) * 2001-11-07 2003-05-08 Katherine A. High Induction of tolerance to a therapeutic polypeptide
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
US20090304726A1 (en) * 2006-02-15 2009-12-10 Ramot At Tel Aviv University Ltd. Viral display vehicles for treating multiple sclerosis
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
EP2364362B1 (en) * 2008-11-12 2015-10-21 Ospedale San Raffaele S.r.l. Gene vector for inducing transgene-specific immune tolerance
JP6189754B2 (ja) * 2011-03-04 2017-08-30 イントレキソン コーポレーション タンパク質を条件的に発現するベクター
JP2015502135A (ja) * 2011-09-26 2015-01-22 ウニヴェルジテート チューリッヒ プロレクトラート エムエヌヴェーＵｎｉｖｅｒｓｉｔａｅｔ Ｚｕｅｒｉｃｈ Ｐｒｏｒｅｋｔｏｒａｔ Ｍｎｗ 多発性硬化症の治療のためのａｐｃ媒介寛容誘導
US20160287685A1 (en) * 2013-11-22 2016-10-06 Amarna Holding B.V. Method for restoring immune tolerance in vivo
WO2021211614A1 (en) * 2020-04-14 2021-10-21 University Of Florida Research Foundation, Incorporated Aav-based gene therapies for treatment of autoimmune diseases

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PT3133923T (pt) 2020-09-04
AU2019202958B2 (en) 2021-08-05
US20170043036A1 (en) 2017-02-16
AU2015249328B2 (en) 2019-01-17
PL3133923T3 (pl) 2021-02-22
LT3133923T (lt) 2020-08-25
JP7430898B2 (ja) 2024-02-14
SMT202000453T1 (it) 2020-09-10
CA2945984A1 (en) 2015-10-29
AU2015249328A1 (en) 2016-10-27
US20200138978A1 (en) 2020-05-07
US20230381342A1 (en) 2023-11-30
JP2017513500A (ja) 2017-06-01
JP2022081549A (ja) 2022-05-31
JP2020062038A (ja) 2020-04-23
HRP20201342T1 (hr) 2020-11-27
EP3133923A1 (en) 2017-03-01
AU2019202958A1 (en) 2019-05-16
ES2814280T3 (es) 2021-03-26
EP3744177A1 (en) 2020-12-02
CY1123312T1 (el) 2021-12-31
RS60816B1 (sr) 2020-10-30
DK3133923T3 (da) 2020-08-10
HUE050681T2 (hu) 2020-12-28
SI3133923T1 (sl) 2020-11-30
EP3133923B1 (en) 2020-06-03
WO2015164789A1 (en) 2015-10-29
EP3133923A4 (en) 2017-12-27
CA2945984C (en) 2023-09-26

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