JP2022537555A - ウイルスベースの遺伝子療法による治療方法 - Google Patents

ウイルスベースの遺伝子療法による治療方法 Download PDF

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JP2022537555A
JP2022537555A JP2021575397A JP2021575397A JP2022537555A JP 2022537555 A JP2022537555 A JP 2022537555A JP 2021575397 A JP2021575397 A JP 2021575397A JP 2021575397 A JP2021575397 A JP 2021575397A JP 2022537555 A JP2022537555 A JP 2022537555A
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patient
gene therapy
viral
smrt
ncor2
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JP2022537555A5 (OSRAM
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イヴァン ビリク
バージット ライペルト
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Takeda Pharmaceutical Co Ltd
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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
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
    • A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
    • 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
    • A61K38/37—Factors VIII
    • 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
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/3955—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against proteinaceous materials, e.g. enzymes, hormones, lymphokines
    • 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
    • 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
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • 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
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/24—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
    • C07K16/244—Interleukins [IL]
    • C07K16/248—IL-6
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2866—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against receptors for cytokines, lymphokines, interferons
    • 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
    • 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)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Zoology (AREA)
  • Hematology (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Endocrinology (AREA)
  • Mycology (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2021575397A 2019-06-20 2020-06-19 ウイルスベースの遺伝子療法による治療方法 Pending JP2022537555A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962864404P 2019-06-20 2019-06-20
US62/864,404 2019-06-20
US201962867172P 2019-06-26 2019-06-26
US62/867,172 2019-06-26
PCT/US2020/038643 WO2020257586A2 (en) 2019-06-20 2020-06-19 Method of treatment with viral-based gene therapy

Publications (2)

Publication Number Publication Date
JP2022537555A true JP2022537555A (ja) 2022-08-26
JP2022537555A5 JP2022537555A5 (OSRAM) 2023-06-23

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JP2021575397A Pending JP2022537555A (ja) 2019-06-20 2020-06-19 ウイルスベースの遺伝子療法による治療方法

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US (1) US20200405883A1 (OSRAM)
EP (1) EP3986481A2 (OSRAM)
JP (1) JP2022537555A (OSRAM)
CN (1) CN114375205A (OSRAM)
WO (1) WO2020257586A2 (OSRAM)

Families Citing this family (3)

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Publication number Priority date Publication date Assignee Title
CN114277057B (zh) * 2021-07-09 2023-10-13 上海天泽云泰生物医药有限公司 用于治疗或预防b型血友病的重组腺相关病毒载体和方法
CN115772214B (zh) * 2022-11-30 2023-09-29 湖南家辉生物技术有限公司 F8突变体蛋白、f8基因突变体、检测f8基因突变体的引物组合、试剂及应用
WO2024151568A1 (en) * 2023-01-09 2024-07-18 Biomarin Pharmaceutical Inc. Epigenetic modifiers improve aav gene therapy durability

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017531652A (ja) * 2014-10-06 2017-10-26 アルスロジェン ビー.ブイ.Arthrogen B.V. Aavに基づく遺伝子治療

Family Cites Families (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2134212T3 (es) 1991-04-25 1999-10-01 Chugai Pharmaceutical Co Ltd Anticuerpo humano reconstituido contra el receptor de la interleuquina 6 humano.
US5877278A (en) 1992-09-24 1999-03-02 Chiron Corporation Synthesis of N-substituted oligomers
US5831005A (en) 1992-09-24 1998-11-03 Chiron Corporation Synthesis of N-substituted oligomers
US6458563B1 (en) 1996-06-26 2002-10-01 Emory University Modified factor VIII
WO1999003496A1 (en) 1997-07-21 1999-01-28 The University Of North Carolina At Chapel Hill Factor ix antihemophilic factor with increased clotting activity
US20020014242A1 (en) * 2000-07-31 2002-02-07 Abraham Scaria Use of rapamycin to inhibit immune response and induce tolerance to gene therapy vector and encoded transgene products
RU2318829C2 (ru) 2001-11-14 2008-03-10 Сентокор, Инк. Антитела против il-6, композиции, способы и применение
PA8672101A1 (es) 2005-04-29 2006-12-07 Centocor Inc Anticuerpos anti-il-6, composiciones, métodos y usos
EA200870130A1 (ru) 2005-12-30 2009-02-27 Мерк Патент Гмбх Антитела против ил-6, предотвращающие связывание с gp130 комплекса ил-6 и альфа-рецептора ил-6
NZ587107A (en) 2006-06-02 2012-05-25 Regeneron Pharma High affinity antibodies to human il-6 receptor
AU2007282023B2 (en) 2006-08-03 2012-09-13 Vaccinex, Inc. Anti-IL-6 monoclonal antibodies and uses thereof
WO2008069942A2 (en) 2006-12-05 2008-06-12 Biogen Idec Ma Inc. Novel methods of enhancing delivery of a gene therapy vector using steroids
CA2724279C (en) 2008-05-13 2017-03-07 Novimmune S.A. Anti-il-6/il-6r antibodies and methods of use thereof
EA019512B1 (ru) 2008-07-02 2014-04-30 Эмерджент Продакт Дивелопмент Сиэтл, Ллс Il-6-опосредованная иммунотерапия
TWI440469B (zh) 2008-09-26 2014-06-11 Chugai Pharmaceutical Co Ltd Improved antibody molecules
KR20110094307A (ko) 2008-11-13 2011-08-23 펨타 파마슈티컬스, 인크. 인간화된 항-il-6 항체
US10618964B2 (en) 2009-04-10 2020-04-14 Ablynx N.V. Nanobody against IL-6R
JP2013503919A (ja) 2009-09-08 2013-02-04 ヤンセン バイオテツク,インコーポレーテツド 癌患者においてヘプシジンを減少させるための抗il−6抗体の使用
JO3417B1 (ar) 2010-01-08 2019-10-20 Regeneron Pharma الصيغ المستقرة التي تحتوي على الأجسام المضادة لمضاد مستقبل( interleukin-6 (il-6r
WO2012032181A2 (en) 2010-09-10 2012-03-15 Allozyne, Inc Novel antibody derivatives
US9017678B1 (en) 2014-07-15 2015-04-28 Kymab Limited Method of treating rheumatoid arthritis using antibody to IL6R
EP3283126B1 (en) * 2015-04-16 2019-11-06 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
CA3119498A1 (en) * 2015-07-31 2017-02-09 Medimmune Limited Methods for treating hepcidin-mediated disorders
PE20181338A1 (es) * 2015-09-24 2018-08-21 Biomarin Pharm Inc Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones terapeuticas que los comprenden
SG11201803654TA (en) 2015-11-03 2018-05-30 Sanofi Biotechnology Compositions comprising il6r antibodies for the treatment of uveitis and macular edema and methods of using same
CN116479001A (zh) * 2015-11-13 2023-07-25 武田药品工业株式会社 用于血友病a的基因治疗的具有增加的表达的编码重组fviii变体的病毒载体
TWI823830B (zh) * 2015-11-13 2023-12-01 日商武田藥品工業股份有限公司 用於a型血友病基因治療之編碼表現增加之重組fviii變異體之病毒載體
WO2018029182A1 (en) 2016-08-08 2018-02-15 Ablynx N.V. Il-6r single variable domain antibodies for treatment of il-6r related diseases
WO2018112237A1 (en) 2016-12-14 2018-06-21 Progenity Inc. Treatment of a disease of the gastrointestinal tract with an il-6r inhibitor
WO2018129268A1 (en) * 2017-01-07 2018-07-12 Selecta Biosciences, Inc. Patterned dosing of immunosuppressants coupled to synthetic nanocarriers
TWI716668B (zh) * 2017-02-01 2021-01-21 耶魯大學 心臟衰竭及心腎症候群之精準治療
WO2018200951A1 (en) * 2017-04-28 2018-11-01 Tran David T Multi-targeted multi-valent ligand-drug particles for the treatment and prevention of diseases and conditions
KR102386890B1 (ko) 2017-05-22 2022-04-15 다케다 야쿠힌 고교 가부시키가이샤 B형 혈우병 유전자 요법을 위한 증가된 발현을 가지는 재조합 fix 변이체를 암호화하는 바이러스 벡터
MX2020002750A (es) 2017-09-13 2020-07-20 Jiangsu Hengrui Medicine Co Anticuerpo il-6r y fragmento de union a antigeno y uso medico.
CN109867723B (zh) 2017-12-05 2022-06-17 南京金斯瑞生物科技有限公司 抗人il6单克隆抗体及其制备方法和用途

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017531652A (ja) * 2014-10-06 2017-10-26 アルスロジェン ビー.ブイ.Arthrogen B.V. Aavに基づく遺伝子治療

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
KLAUDIA KURANDA ET AL.: ""Exposure to wild-type AAV drives distinct capsid immunity profiles in humans"", JOURNAL OF CLINICAL INVESTIGATION, vol. 128, no. 12, JPN6024027726, 22 October 2018 (2018-10-22), pages 5267 - 5279, ISSN: 0005613709 *
VICTORIA M. VELAZQUEZ ET AL.: ""Effective Depletion of Pre-existing Anti-AAV Antibodies Requires Broad Immune Targeting"", MOLECULAR THERAPY - METHODS & CLINICAL DEVELOPMENT, vol. 4, JPN6024027727, March 2017 (2017-03-01), pages 159 - 168, ISSN: 0005613710 *

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EP3986481A2 (en) 2022-04-27
CN114375205A (zh) 2022-04-19
WO2020257586A3 (en) 2021-03-04
US20200405883A1 (en) 2020-12-31
WO2020257586A2 (en) 2020-12-24

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