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 PDFInfo
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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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- 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
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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
- A61K38/36—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
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- 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)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Immunology (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Zoology (AREA)
- Hematology (AREA)
- Microbiology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- General Chemical & Material Sciences (AREA)
- Diabetes (AREA)
- Gastroenterology & Hepatology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Mycology (AREA)
- Endocrinology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Toxicology (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)
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 (https=) | 2023-06-23 |
Family
ID=71579659
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US20200405883A1 (https=) |
| EP (1) | EP3986481A2 (https=) |
| JP (1) | JP2022537555A (https=) |
| CN (1) | CN114375205A (https=) |
| WO (1) | WO2020257586A2 (https=) |
Families Citing this family (3)
| 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 |
| TW202444398A (zh) * | 2023-01-09 | 2024-11-16 | 美商拜奧馬林製藥公司 | 表觀遺傳修飾劑改善aav基因療法之耐久性 |
Citations (1)
| 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)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5795965A (en) | 1991-04-25 | 1998-08-18 | Chugai Seiyaku Kabushiki Kaisha | Reshaped human to human interleukin-6 receptor |
| US5831005A (en) | 1992-09-24 | 1998-11-03 | Chiron Corporation | Synthesis of N-substituted oligomers |
| US5877278A (en) | 1992-09-24 | 1999-03-02 | Chiron Corporation | Synthesis of N-substituted oligomers |
| US6458563B1 (en) | 1996-06-26 | 2002-10-01 | Emory University | Modified factor VIII |
| US6531298B2 (en) | 1997-07-21 | 2003-03-11 | 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 |
| PT2308888T (pt) | 2001-11-14 | 2017-05-03 | Janssen Biotech Inc | Anticorpos anti-il-6, composições, métodos e utilizações |
| JO3058B1 (ar) | 2005-04-29 | 2017-03-15 | Applied Molecular Evolution Inc | الاجسام المضادة لمضادات -اي ال-6,تركيباتها طرقها واستعمالاتها |
| ATE550353T1 (de) | 2005-12-30 | 2012-04-15 | Merck Patent Gmbh | Die bindung von mit il-6ralpha komplexiertem il-6 an gp130 verhindernde anti-il-6-antikörper |
| JP5307708B2 (ja) | 2006-06-02 | 2013-10-02 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | ヒトil−6受容体に対する高親和性抗体 |
| CN101563365B (zh) | 2006-08-03 | 2012-10-31 | 瓦西尼斯公司 | 抗-il-6单克隆抗体及其用途 |
| WO2008069942A2 (en) | 2006-12-05 | 2008-06-12 | Biogen Idec Ma Inc. | Novel methods of enhancing delivery of a gene therapy vector using steroids |
| MX2010012031A (es) | 2008-05-13 | 2011-01-20 | Novimmune Sa | Anticuerpos anti-il-6/il-6r y metodos de uso de los mismos. |
| MX2011000071A (es) | 2008-07-02 | 2011-05-03 | Emergent Product Dev Seattle | Inmunoterapeuticos de interleucina-6 (il6). |
| TWI440469B (zh) | 2008-09-26 | 2014-06-11 | Chugai Pharmaceutical Co Ltd | Improved antibody molecules |
| AU2009313958B2 (en) | 2008-11-13 | 2015-09-10 | Femta Pharmaceuticals, Inc. | Humanized anti-IL-6 antibodies |
| WO2010115995A2 (en) | 2009-04-10 | 2010-10-14 | Ablynx Nv | Improved amino acid sequences directed against il-6r and polypeptides comprising the same for the treatment of il-6r related diseases and disorders |
| WO2011031657A1 (en) | 2009-09-08 | 2011-03-17 | Centocor Ortho Biotech Inc. | Use of an anti-il6 antibody to decrease hepcidin in cancer patients |
| JO3417B1 (ar) | 2010-01-08 | 2019-10-20 | Regeneron Pharma | الصيغ المستقرة التي تحتوي على الأجسام المضادة لمضاد مستقبل( interleukin-6 (il-6r |
| EP2640745B1 (en) | 2010-09-10 | 2018-11-07 | MedImmune Limited | Bivalent and bispecific anti-il6/anti-il23 antibodies |
| 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 |
| CA2991637C (en) * | 2015-07-31 | 2022-07-05 | Medimmune Limited | Methods for treating hepcidin-mediated disorders |
| TW202446416A (zh) * | 2015-09-24 | 2024-12-01 | 美商拜奧馬林製藥公司 | 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物 |
| WO2017079443A1 (en) | 2015-11-03 | 2017-05-11 | Sanofi Biotechnology | Compositions comprising il6r antibodies for the treatment of uveitis and macular edema and methods of using same |
| EA202190827A1 (ru) * | 2015-11-13 | 2021-11-30 | Баксалта Инкорпорейтед | Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a |
| CA3005565A1 (en) * | 2015-11-13 | 2017-05-18 | Baxalta Incorporated | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a |
| 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 |
| MX2019008143A (es) * | 2017-01-07 | 2020-01-13 | Selecta Biosciences Inc | Dosificación sistemática de inmunosupresores acoplados a nanoportadores sintéticos. |
| KR20200012823A (ko) * | 2017-02-01 | 2020-02-05 | 예일 유니버시티 | 이뇨제 내성의 치료 |
| US11617797B2 (en) * | 2017-04-28 | 2023-04-04 | David T. Tran | Multi-targeted multi-valent ligand drug particles for the treatment and prevention of diseases and conditions |
| CN110945127B (zh) | 2017-05-22 | 2024-07-12 | 武田药品工业株式会社 | 用于血友病b的基因疗法的编码具有增加的表达的重组fix的病毒载体 |
| CN111051343B (zh) | 2017-09-13 | 2022-11-22 | 江苏恒瑞医药股份有限公司 | Il-6r抗体、其抗原结合片段及医药用途 |
| 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 |
-
2020
- 2020-06-19 CN CN202080044892.9A patent/CN114375205A/zh active Pending
- 2020-06-19 US US16/906,349 patent/US20200405883A1/en not_active Abandoned
- 2020-06-19 WO PCT/US2020/038643 patent/WO2020257586A2/en not_active Ceased
- 2020-06-19 EP EP20739798.5A patent/EP3986481A2/en not_active Withdrawn
- 2020-06-19 JP JP2021575397A patent/JP2022537555A/ja active Pending
Patent Citations (1)
| 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)
| 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 * |
Also Published As
| Publication number | Publication date |
|---|---|
| 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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