JP2012519008A - 集合活性化タンパク質（ａａｐ）およびｖｐ３から本質的になるパルボウイルス粒子を製造するためのａａｐの使用 - Google Patents

集合活性化タンパク質（ａａｐ）およびｖｐ３から本質的になるパルボウイルス粒子を製造するためのａａｐの使用 Download PDF

Info

Publication number
JP2012519008A
JP2012519008A JP2011552364A JP2011552364A JP2012519008A JP 2012519008 A JP2012519008 A JP 2012519008A JP 2011552364 A JP2011552364 A JP 2011552364A JP 2011552364 A JP2011552364 A JP 2011552364A JP 2012519008 A JP2012519008 A JP 2012519008A
Authority
JP
Japan
Prior art keywords
seq
parvovirus
protein
nucleic acid
aav
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2011552364A
Other languages
English (en)
Japanese (ja)
Other versions
JP2012519008A5 (https=
Inventor
フロリアン ゾンターク
ユルゲン クラインシュミット
マーカス ヘーラー
ケルスティン ルクス
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Deutsches Krebsforschungszentrum DKFZ
Original Assignee
Deutsches Krebsforschungszentrum DKFZ
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Deutsches Krebsforschungszentrum DKFZ filed Critical Deutsches Krebsforschungszentrum DKFZ
Publication of JP2012519008A publication Critical patent/JP2012519008A/ja
Publication of JP2012519008A5 publication Critical patent/JP2012519008A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/04—Drugs for disorders of the alimentary tract or the digestive system for ulcers, gastritis or reflux esophagitis, e.g. antacids, inhibitors of acid secretion, mucosal protectants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A61P11/02—Nasal agents, e.g. decongestants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A61P11/06—Antiasthmatics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/06—Antipsoriatics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • A61P25/30—Drugs for disorders of the nervous system for treating abuse or dependence
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • 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
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • 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
    • A61P37/08—Antiallergic agents
    • 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
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • A61K2039/525—Virus
    • A61K2039/5256—Virus expressing foreign proteins
    • 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
    • A61K2039/525—Virus
    • A61K2039/5258—Virus-like particles
    • 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
    • C12N2750/14123—Virus like particles [VLP]
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Virology (AREA)
  • Wood Science & Technology (AREA)
  • Pulmonology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Psychiatry (AREA)
  • Rheumatology (AREA)
  • Mycology (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Pain & Pain Management (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
JP2011552364A 2009-03-04 2010-03-04 集合活性化タンパク質（ａａｐ）およびｖｐ３から本質的になるパルボウイルス粒子を製造するためのａａｐの使用 Pending JP2012519008A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US15743609P 2009-03-04 2009-03-04
US61/157,436 2009-03-04
US30620510P 2010-02-19 2010-02-19
US61/306,205 2010-02-19
PCT/EP2010/001343 WO2010099960A2 (en) 2009-03-04 2010-03-04 Assembly activating protein (aap) and its use for the manufacture of parvovirus particles essential consisting of vp3

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2015200962A Division JP6151328B2 (ja) 2009-03-04 2015-10-09 集合活性化タンパク質（ａａｐ）およびｖｐ３から本質的になるパルボウイルス粒子を製造するためのａａｐの使用

Publications (2)

Publication Number Publication Date
JP2012519008A true JP2012519008A (ja) 2012-08-23
JP2012519008A5 JP2012519008A5 (https=) 2013-04-11

Family

ID=42313642

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2011552364A Pending JP2012519008A (ja) 2009-03-04 2010-03-04 集合活性化タンパク質（ａａｐ）およびｖｐ３から本質的になるパルボウイルス粒子を製造するためのａａｐの使用
JP2015200962A Expired - Fee Related JP6151328B2 (ja) 2009-03-04 2015-10-09 集合活性化タンパク質（ａａｐ）およびｖｐ３から本質的になるパルボウイルス粒子を製造するためのａａｐの使用

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2015200962A Expired - Fee Related JP6151328B2 (ja) 2009-03-04 2015-10-09 集合活性化タンパク質（ａａｐ）およびｖｐ３から本質的になるパルボウイルス粒子を製造するためのａａｐの使用

Country Status (12)

Country Link
US (3) US9464119B2 (https=)
EP (1) EP2403867B1 (https=)
JP (2) JP2012519008A (https=)
CN (1) CN102341406B (https=)
BR (1) BRPI1013364A2 (https=)
CA (1) CA2754335C (https=)
DK (1) DK2403867T3 (https=)
ES (1) ES2742180T3 (https=)
MX (2) MX2011009193A (https=)
PL (1) PL2403867T3 (https=)
PT (1) PT2403867T (https=)
WO (1) WO2010099960A2 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021516048A (ja) * 2018-02-28 2021-07-01 ユニバーシティ オブ ピッツバーグ −オブ ザ コモンウェルス システム オブ ハイヤー エデュケイション 遺伝子及びタンパク質の送達のためのａａｖに基づくモジュールシステム

Families Citing this family (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2742180T3 (es) 2009-03-04 2020-02-13 Deutsches Krebsforsch Proteína activadora del ensamblaje (AAP) y su uso para la fabricación de partículas de parvovirus que consisten esencialmente en VP3
EP2534172A2 (en) * 2010-02-12 2012-12-19 The U.S.A. as represented by the Secretary, Department of Health and Human Services Compositions and methods for preventing or treating a human parvovirus infection
AR082925A1 (es) * 2010-09-08 2013-01-16 Medigene Ag Proteinas estructurales mutadas por parvovirus con epitopo de celulas b de proteccion cruzada, producto y metodos relacionados
RU2487708C1 (ru) * 2012-03-12 2013-07-20 Государственное бюджетное образовательное учреждение высшего профессионального образования "Уральская государственная медицинская академия Министерства здравоохранения и социального развития Российской Федерации" (ГБОУ ВПО УГМА Минздравсоцразвития России) Способ лечения парвовирусной инфекции в19 у детей раннего возраста
TWI698240B (zh) 2012-05-15 2020-07-11 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
EP3800191B1 (en) 2014-03-17 2025-08-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
RU2716991C2 (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
US11021519B2 (en) 2015-03-02 2021-06-01 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
CN108603235A (zh) 2015-07-30 2018-09-28 马萨诸塞眼科耳科诊所 祖先病毒序列及其用途
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
KR102427379B1 (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2018044933A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ハンチントン病治療組成物および方法
JP2020518258A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
EP3645021A4 (en) 2017-06-30 2021-04-21 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
EP3808849A1 (en) 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
AU2018338728B2 (en) 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery
US20200231986A1 (en) * 2017-09-29 2020-07-23 Massachusetts Eye And Ear Infirmary Production of adeno-associated viruses in insect cells
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
US20190202866A1 (en) * 2017-12-22 2019-07-04 William Marsh Rice University Viral nanoparticles and methods of use thereof
EP3527223A1 (en) 2018-02-16 2019-08-21 2A Pharma AB Mutated parvovirus structural protein
AU2019220386A1 (en) 2018-02-16 2020-08-20 2A Pharma Ab Parvovirus structural protein for the treatment of autoimmune diseases
US20210147487A1 (en) * 2018-04-04 2021-05-20 The University Of North Carolina At Chapel Hill Methods and compositions for engineered assembly activating proteins (eaaps)
US12037707B2 (en) 2018-04-05 2024-07-16 Massachusetts Eye And Ear Infirmary Methods of making and using combinatorial barcoded nucleic acid libraries having defined variation
EP3784697A4 (en) * 2018-04-27 2022-07-06 Spark Therapeutics, Inc. GMO AAV CAPSIDS WITH INCREASED TROPISM AND AAV VECTORS CONTAINING THE GMO CAPSIDS, AND METHODS FOR THEIR MANUFACTURE AND USE
KR20210019996A (ko) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 조성물 및 방법
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
WO2020142236A1 (en) * 2019-01-02 2020-07-09 Intima Bioscience, Inc. Modified adeno-associated viral vectors for use in genetic engineering
KR20210148101A (ko) 2019-02-04 2021-12-07 프리라인 테라퓨틱스 리미티드 폴리뉴클레오티드
CN113728108A (zh) 2019-02-15 2021-11-30 桑格摩生物治疗股份有限公司 用于生产重组aav的组合物和方法
KR20220019669A (ko) * 2019-04-12 2022-02-17 프리라인 테라퓨틱스 리미티드 플라스미드 시스템
EP4100056A4 (en) * 2020-02-07 2024-03-06 Vecprobio, Inc. Recombinant adeno-associated viral vectors in plants
CN116249771A (zh) * 2020-06-25 2023-06-09 辉凌企业有限公司 改进的腺相关病毒基因治疗载体
WO2023083964A1 (en) 2021-11-11 2023-05-19 2A Pharma Ab Parvovirus structural protein against beta- and gamma-hpv
EP4603505A1 (en) * 2022-10-13 2025-08-20 Kanglin Biotech (Hangzhou) Co., Ltd. Method for modifying capsid protein coding gene of adeno-associated virus
CN117126270B (zh) * 2023-10-25 2024-02-13 首都儿科研究所 一种2型人博卡病毒型别特异性抗体及其应用
WO2025125222A1 (en) 2023-12-11 2025-06-19 Leukocare Ag Compositions comprising viruses, viral vectors or virus-like particles

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4168062A (en) 1977-12-05 1979-09-18 Mccarthy Gerald F Automated goalie
US6027722A (en) * 1990-10-25 2000-02-22 Nature Technology Corporation Vectors for gene transfer
DE19905501B4 (de) 1999-02-10 2005-05-19 MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels
US20020052040A1 (en) * 1999-06-30 2002-05-02 Nicholas Hunt Virus like particles, their preparation and their use preferably in pharmaceutical screening and functional genomics
DE19933288A1 (de) 1999-07-15 2001-01-18 Medigene Ag Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung
DE19933719A1 (de) 1999-07-19 2001-01-25 Medigene Ag Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung
JP2001169777A (ja) 1999-07-30 2001-06-26 Hiroshi Handa アデノ随伴ウイルスのｖｐ３キャプシド蛋白質からウイルス様粒子を形成する方法
US7638120B2 (en) * 2000-03-14 2009-12-29 Thomas Jefferson University High transgene expression of a pseudotyped adeno-associated virus type
DE10024334B4 (de) 2000-05-17 2006-06-01 Medigene Ag Verfahren zum Einbringen von Nukleinsäure in eine Zelle (Transfektion) mittels Calciumphosphat
WO2002053703A2 (en) * 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
US7749492B2 (en) * 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
CA2453405A1 (en) * 2001-07-13 2003-01-23 University Of Iowa Research Foundation Pseudotyped adeno-associated viruses and uses thereof
SI1496944T1 (sl) 2002-05-01 2009-02-28 Univ Florida Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov
US20060093589A1 (en) * 2004-02-19 2006-05-04 Warrington Kenneth H Vp2-modified raav vector compositions and uses therefor
WO2004000220A2 (en) * 2002-06-21 2003-12-31 Genetix Pharmaceuticals, Inc. Methods for purifying viral particles for gene therapy
ES2602610T3 (es) 2007-05-31 2017-02-21 Medigene Ag Proteína estructural mutada de un parvovirus
EP2012122A1 (en) * 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
ES2742180T3 (es) 2009-03-04 2020-02-13 Deutsches Krebsforsch Proteína activadora del ensamblaje (AAP) y su uso para la fabricación de partículas de parvovirus que consisten esencialmente en VP3

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
JPN6014029203; Ｊ　Ｖｉｒｏｌ．，　１９９９年，　第７３巻，　７９１２-７９１５ページ *
JPN6014029205; ＰＮＡＳ，　２０１０年６月，　第１０７巻，　１０２２０-１０２２５ページ *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021516048A (ja) * 2018-02-28 2021-07-01 ユニバーシティ オブ ピッツバーグ −オブ ザ コモンウェルス システム オブ ハイヤー エデュケイション 遺伝子及びタンパク質の送達のためのａａｖに基づくモジュールシステム
US12421525B2 (en) 2018-02-28 2025-09-23 University of Pittsburgh—of the Commonwealth System of Higher Education Conjugates comprising AAVs and CAS9 polypeptides

Also Published As

Publication number Publication date
JP2016063810A (ja) 2016-04-28
DK2403867T3 (da) 2019-08-12
US20120135022A1 (en) 2012-05-31
PT2403867T (pt) 2019-08-29
US11267847B2 (en) 2022-03-08
CN102341406A (zh) 2012-02-01
US20200087352A1 (en) 2020-03-19
EP2403867A2 (en) 2012-01-11
EP2403867B1 (en) 2019-05-22
US9464119B2 (en) 2016-10-11
CA2754335C (en) 2019-12-24
US10344057B2 (en) 2019-07-09
JP6151328B2 (ja) 2017-06-21
ES2742180T3 (es) 2020-02-13
US20170226160A1 (en) 2017-08-10
MX345729B (es) 2017-02-14
CA2754335A1 (en) 2010-09-10
WO2010099960A2 (en) 2010-09-10
CN102341406B (zh) 2016-06-08
BRPI1013364A2 (pt) 2016-03-29
MX2011009193A (es) 2011-12-16
WO2010099960A3 (en) 2011-01-13
PL2403867T3 (pl) 2019-12-31

Similar Documents

Publication Publication Date Title
JP6151328B2 (ja) 集合活性化タンパク質（ａａｐ）およびｖｐ３から本質的になるパルボウイルス粒子を製造するためのａａｐの使用
CN110770346B (zh) 多倍体腺相关病毒载体及其制备和使用方法
KR102572449B1 (ko) 곤충 세포에서 생산된 더욱 향상된 aav 벡터
JP4842471B2 (ja) 修飾された抗原性を有するアデノ随伴ウイルスの構造タンパク質と、その製造及び使用
JP4559429B2 (ja) 空キャプシドを実質的に含まない組換えａａｖビリオン調製物を生成するための方法
JP2024026105A (ja) 抗体回避ウイルスベクターのための方法および組成物
WO2011122950A1 (en) Monomeric duplex aav vectors
EP2297185A1 (en) Parvoviral capsid with incorporated gly-ala repeat region
CN108697774A (zh) 治疗mps i-相关失明的aav-idua载体
US20230365942A1 (en) Engineered aav vectors
JP2023540085A (ja) ＳＡＲＳ－ＣｏＶ－２に対するＡＡＶ５に基づくワクチン
JP2024515626A (ja) 血液脳関門を横断し低減された液性応答を惹起する合理的ポリプロイドａａｖビリオン
WO2019195549A1 (en) Methods and compositions for engineered assembly activating proteins (eaaps)
WO2026037757A1 (en) Modified aav particles
CN119143858A (zh) 一种kcc2截短体或其编码序列及其应用

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20130225

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20130225

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20140710

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20141002

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20141009

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20150113

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20150526

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20150610