JP2014512171A - コロイデレミアの遺伝子治療に使用するためのａａｖベクター - Google Patents

コロイデレミアの遺伝子治療に使用するためのａａｖベクター Download PDF

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JP2014512171A
JP2014512171A JP2013554938A JP2013554938A JP2014512171A JP 2014512171 A JP2014512171 A JP 2014512171A JP 2013554938 A JP2013554938 A JP 2013554938A JP 2013554938 A JP2013554938 A JP 2013554938A JP 2014512171 A JP2014512171 A JP 2014512171A
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vector
aav
rep1
sequence
seq
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JP2014512171A5 (enExample
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マクラーレン、ロバート
シーブラ、ミゲル
ジョン デュアリング、マシュウ
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ISISINNOVATION LIMITED
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ISISINNOVATION LIMITED
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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)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
JP2013554938A 2011-02-22 2012-02-21 コロイデレミアの遺伝子治療に使用するためのａａｖベクター Pending JP2014512171A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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

Related Child Applications (1)

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JP2017117286A Division JP2017221192A (ja) 2011-02-22 2017-06-14 コロイデレミアの遺伝子治療に使用するためのａａｖベクター

Publications (2)

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JP2014512171A true JP2014512171A (ja) 2014-05-22
JP2014512171A5 JP2014512171A5 (enExample) 2015-04-16

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Family Applications (4)

Application Number Title Priority Date Filing Date
JP2013554938A Pending JP2014512171A (ja) 2011-02-22 2012-02-21 コロイデレミアの遺伝子治療に使用するためのａａｖベクター
JP2017117286A Pending JP2017221192A (ja) 2011-02-22 2017-06-14 コロイデレミアの遺伝子治療に使用するためのａａｖベクター
JP2019201092A Pending JP2020054345A (ja) 2011-02-22 2019-11-05 コロイデレミアの遺伝子治療に使用するためのａａｖベクター
JP2022193831A Pending JP2023036618A (ja) 2011-02-22 2022-12-02 コロイデレミアの遺伝子治療に使用するためのａａｖベクター

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JP2017117286A Pending JP2017221192A (ja) 2011-02-22 2017-06-14 コロイデレミアの遺伝子治療に使用するためのａａｖベクター
JP2019201092A Pending JP2020054345A (ja) 2011-02-22 2019-11-05 コロイデレミアの遺伝子治療に使用するためのａａｖベクター
JP2022193831A Pending JP2023036618A (ja) 2011-02-22 2022-12-02 コロイデレミアの遺伝子治療に使用するためのａａｖベクター

Country Status (18)

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US (4) US9834788B2 (enExample)
EP (2) EP3428283A1 (enExample)
JP (4) JP2014512171A (enExample)
KR (3) KR102299430B1 (enExample)
CN (2) CN107881198A (enExample)
AU (2) AU2012220404B2 (enExample)
BR (1) BR112013021318B1 (enExample)
CA (1) CA2827975C (enExample)
DK (1) DK2678435T3 (enExample)
ES (1) ES2676550T5 (enExample)
GB (1) GB201103062D0 (enExample)
HK (2) HK1258917A1 (enExample)
IL (1) IL228031B (enExample)
MX (2) MX388146B (enExample)
PL (1) PL2678435T3 (enExample)
PT (1) PT2678435T (enExample)
SG (1) SG192911A1 (enExample)
WO (1) WO2012114090A1 (enExample)

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018511652A (ja) * 2015-03-06 2018-04-26 マサチューセッツ アイ アンド イヤー インファーマリー Ｐｒｐｆ３１遺伝子における変異により引き起こされる遺伝性網膜変性に対する遺伝子増強療法
JP2019502378A (ja) * 2015-12-14 2019-01-31 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
JP2019509762A (ja) * 2016-03-10 2019-04-11 ナイトスタアールエックス リミテッド プレニル化アッセイ
JP2021505619A (ja) * 2017-12-06 2021-02-18 オービッド・セラピューティクス・インコーポレイテッドＯｖｉｄ Ｔｈｅｒａｐｅｕｔｉｃｓ Ｉｎｃ． 発作障害の治療におけるｍｉｒ１０１またはｍｉｒ１２８の使用
JP2021520233A (ja) * 2018-04-05 2021-08-19 ナイトスター リミテッド Ａａｖ組成物、作製する方法及び使用の方法

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WO2015082690A1 (en) * 2013-12-06 2015-06-11 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the retinal pigment epithelium of a subject
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
CN112553229A (zh) 2014-11-05 2021-03-26 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
RU2020108189A (ru) 2014-11-14 2020-03-11 Вояджер Терапьютикс, Инк. Композиции и способы лечения бокового амиотрофического склероза (als)
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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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Cited By (11)

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Publication number Priority date Publication date Assignee Title
JP2018511652A (ja) * 2015-03-06 2018-04-26 マサチューセッツ アイ アンド イヤー インファーマリー Ｐｒｐｆ３１遺伝子における変異により引き起こされる遺伝性網膜変性に対する遺伝子増強療法
JP2021059561A (ja) * 2015-03-06 2021-04-15 マサチューセッツ アイ アンド イヤー インファーマリー Ｐｒｐｆ３１遺伝子における変異により引き起こされる遺伝性網膜変性に対する遺伝子増強療法
JP7239550B2 (ja) 2015-03-06 2023-03-14 マサチューセッツ アイ アンド イヤー インファーマリー Ｐｒｐｆ３１遺伝子における変異により引き起こされる遺伝性網膜変性に対する遺伝子増強療法
JP2023071867A (ja) * 2015-03-06 2023-05-23 マサチューセッツ アイ アンド イヤー インファーマリー Ｐｒｐｆ３１遺伝子における変異により引き起こされる遺伝性網膜変性に対する遺伝子増強療法
JP2019502378A (ja) * 2015-12-14 2019-01-31 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
JP7057281B2 (ja) 2015-12-14 2022-04-19 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
JP2022088656A (ja) * 2015-12-14 2022-06-14 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
JP2019509762A (ja) * 2016-03-10 2019-04-11 ナイトスタアールエックス リミテッド プレニル化アッセイ
JP7021122B2 (ja) 2016-03-10 2022-02-16 ナイトスタアールエックス リミテッド プレニル化アッセイ
JP2021505619A (ja) * 2017-12-06 2021-02-18 オービッド・セラピューティクス・インコーポレイテッドＯｖｉｄ Ｔｈｅｒａｐｅｕｔｉｃｓ Ｉｎｃ． 発作障害の治療におけるｍｉｒ１０１またはｍｉｒ１２８の使用
JP2021520233A (ja) * 2018-04-05 2021-08-19 ナイトスター リミテッド Ａａｖ組成物、作製する方法及び使用の方法

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