CN103562396A - 用于无脉络膜的基因疗法的aav载体 - Google Patents

用于无脉络膜的基因疗法的aav载体 Download PDF

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Publication number
CN103562396A
CN103562396A CN201280019547.5A CN201280019547A CN103562396A CN 103562396 A CN103562396 A CN 103562396A CN 201280019547 A CN201280019547 A CN 201280019547A CN 103562396 A CN103562396 A CN 103562396A
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aav
carrier
rep1
sequence
vector
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Chinese (zh)
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R·麦克拉伦
M·塞亚夫拉
M·J·杜日能
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Oxford University Innovation Ltd
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Oxford University Innovation Ltd
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Application filed by Oxford University Innovation Ltd filed Critical Oxford University Innovation Ltd
Priority to CN201710784145.9A priority Critical patent/CN107881198A/zh
Publication of CN103562396A publication Critical patent/CN103562396A/zh
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    • 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
    • A61K39/12—Viral antigens
    • 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
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1085—Transferases (2.) transferring alkyl or aryl groups other than methyl groups (2.5)
    • 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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
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  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
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  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN201280019547.5A 2011-02-22 2012-02-21 用于无脉络膜的基因疗法的aav载体 Pending CN103562396A (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN201710784145.9A CN107881198A (zh) 2011-02-22 2012-02-21 用于无脉络膜的基因疗法的aav载体

Applications Claiming Priority (3)

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GBGB1103062.4A GB201103062D0 (en) 2011-02-22 2011-02-22 Method
GB1103062.4 2011-02-22
PCT/GB2012/050376 WO2012114090A1 (en) 2011-02-22 2012-02-21 Aav -vectors for use in gene therapy of choroideremia

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CN201710784145.9A Division CN107881198A (zh) 2011-02-22 2012-02-21 用于无脉络膜的基因疗法的aav载体

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CN201710784145.9A Pending CN107881198A (zh) 2011-02-22 2012-02-21 用于无脉络膜的基因疗法的aav载体

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US (4) US9834788B2 (https=)
EP (2) EP3428283A1 (https=)
JP (4) JP2014512171A (https=)
KR (3) KR102139203B1 (https=)
CN (2) CN103562396A (https=)
AU (2) AU2012220404B2 (https=)
BR (1) BR112013021318B1 (https=)
CA (1) CA2827975C (https=)
DK (1) DK2678435T3 (https=)
ES (1) ES2676550T5 (https=)
GB (1) GB201103062D0 (https=)
HK (2) HK1258917A1 (https=)
IL (1) IL228031B (https=)
MX (2) MX356525B (https=)
PL (1) PL2678435T3 (https=)
PT (1) PT2678435T (https=)
SG (1) SG192911A1 (https=)
WO (1) WO2012114090A1 (https=)

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CN105940109A (zh) * 2013-12-06 2016-09-14 国立健康与医学研究所 用于在受试者的视网膜色素上皮中表达目的多核苷酸的方法和药物组合物
CN112501208A (zh) * 2017-10-03 2021-03-16 普利维尔治疗公司 用于溶酶体障碍的基因疗法

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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)
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US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
EP3748007B1 (en) * 2015-03-06 2024-06-26 Massachusetts Eye & Ear Infirmary Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene
CA3008264A1 (en) * 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
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KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
KR102427379B1 (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
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
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PT3568483T (pt) * 2017-01-10 2025-11-18 Childrens Medical Res Institute Polinucleótidos e vetores para a expressão de transgenes
US10550405B2 (en) 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
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
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
JP7766393B2 (ja) 2017-06-14 2025-11-10 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 眼疾患のための遺伝子療法
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KR20200119782A (ko) * 2017-12-06 2020-10-20 오비드 테라퓨틱스 인크. 발작 장애 치료 시 mir101 또는 mir128의 용도
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JP7244547B2 (ja) 2018-02-19 2023-03-22 ホモロジー・メディシンズ・インコーポレイテッド Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法
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EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
JP7585233B2 (ja) * 2019-04-30 2024-11-18 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ポンペ病の治療のために有用な組成物
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
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CN112501208A (zh) * 2017-10-03 2021-03-16 普利维尔治疗公司 用于溶酶体障碍的基因疗法

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KR20140044793A (ko) 2014-04-15
BR112013021318B1 (pt) 2021-12-07
JP2014512171A (ja) 2014-05-22
PT2678435T (pt) 2018-07-17
GB201103062D0 (en) 2011-04-06
EP3428283A1 (en) 2019-01-16
MX356525B (es) 2018-06-01
AU2012220404A1 (en) 2013-09-12
KR102139203B1 (ko) 2020-07-29
US20180135078A1 (en) 2018-05-17
ES2676550T5 (es) 2024-06-27
JP2020054345A (ja) 2020-04-09
JP2023036618A (ja) 2023-03-14
AU2012220404B2 (en) 2017-04-20
MX2013009604A (es) 2013-09-16
US20210032656A1 (en) 2021-02-04
CN107881198A (zh) 2018-04-06
US9834788B2 (en) 2017-12-05
KR102299430B1 (ko) 2021-09-07
HK1249548A1 (zh) 2018-11-02
WO2012114090A1 (en) 2012-08-30
US20230332176A1 (en) 2023-10-19
IL228031B (en) 2018-02-28
PL2678435T3 (pl) 2018-11-30
US20140107185A1 (en) 2014-04-17
SG192911A1 (en) 2013-09-30
JP2017221192A (ja) 2017-12-21
DK2678435T3 (en) 2018-08-27
ES2676550T3 (es) 2018-07-20
HK1258917A1 (en) 2019-11-22
KR20210112407A (ko) 2021-09-14
KR20200092422A (ko) 2020-08-03
MX388146B (es) 2025-03-19
BR112013021318A2 (pt) 2016-10-25
CA2827975A1 (en) 2012-08-30
CA2827975C (en) 2023-06-13
EP2678435A1 (en) 2014-01-01
IL228031A0 (en) 2013-09-30
EP2678435B2 (en) 2024-02-07
AU2017206254A1 (en) 2017-08-10
EP2678435B1 (en) 2018-05-16

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Application publication date: 20140205