JP6673923B2 - ミオシリン（ｍｙｏｃ）緑内障を処置するためのアデノ随伴ウイルスベクター - Google Patents

ミオシリン（ｍｙｏｃ）緑内障を処置するためのアデノ随伴ウイルスベクター Download PDF

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JP6673923B2
JP6673923B2 JP2017534525A JP2017534525A JP6673923B2 JP 6673923 B2 JP6673923 B2 JP 6673923B2 JP 2017534525 A JP2017534525 A JP 2017534525A JP 2017534525 A JP2017534525 A JP 2017534525A JP 6673923 B2 JP6673923 B2 JP 6673923B2
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myoc
seq
aav
capsid
raav
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JP2017529395A (ja
JP2017529395A5 (enExample
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ピーター・ペカン
エイブラハム・スケーリア
ジェフリー・アーディンジャー
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Genzyme Corp
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Genzyme Corp
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    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
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    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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JP2017534525A 2014-09-16 2015-09-16 ミオシリン（ｍｙｏｃ）緑内障を処置するためのアデノ随伴ウイルスベクター Active JP6673923B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462051299P 2014-09-16 2014-09-16
US62/051,299 2014-09-16
PCT/US2015/050515 WO2016044478A1 (en) 2014-09-16 2015-09-16 Adeno-associated viral vectors for treating myocilin (myoc) glaucoma

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JP2020036344A Division JP7097398B2 (ja) 2014-09-16 2020-03-04 ミオシリン（ｍｙｏｃ）緑内障を処置するためのアデノ随伴ウイルスベクター

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JP2017529395A JP2017529395A (ja) 2017-10-05
JP2017529395A5 JP2017529395A5 (enExample) 2018-10-25
JP6673923B2 true JP6673923B2 (ja) 2020-03-25

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JP2020036344A Active JP7097398B2 (ja) 2014-09-16 2020-03-04 ミオシリン（ｍｙｏｃ）緑内障を処置するためのアデノ随伴ウイルスベクター

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US (1) US10821193B2 (enExample)
EP (1) EP3194601B1 (enExample)
JP (2) JP6673923B2 (enExample)
KR (3) KR20260019013A (enExample)
CN (2) CN116585492A (enExample)
AU (2) AU2015317756B2 (enExample)
BR (1) BR112017005235B1 (enExample)
CA (2) CA3280614A1 (enExample)
IL (1) IL251178B (enExample)
MX (2) MX2017003426A (enExample)
RU (2) RU2718047C2 (enExample)
SG (3) SG10201913430VA (enExample)
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Cited By (1)

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JP7097398B2 (ja) 2014-09-16 2022-07-07 ジェンザイム・コーポレーション ミオシリン（ｍｙｏｃ）緑内障を処置するためのアデノ随伴ウイルスベクター

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JP7287611B2 (ja) * 2018-02-07 2023-06-06 学校法人日本医科大学 改良型アデノ随伴ウイルスベクター
US20210030719A1 (en) * 2018-02-22 2021-02-04 University Of South Florida Grp94 inhibitors to treat steroid-induced ocular hypertensions and glaucomas
EP3930694A1 (en) 2019-02-28 2022-01-05 Regeneron Pharmaceuticals, Inc. Adeno-associated virus vectors for the delivery of therapeutics
EP4255457A4 (en) * 2020-12-03 2025-05-21 University of Massachusetts DEVELOPMENT OF NEW GENE THERAPEUTIC AGENTS FOR FIBRODYSPLASIA OSSIFICANS PROGRESSIVA
CN113032010B (zh) * 2021-03-12 2022-09-20 歌尔科技有限公司 命令的传输控制方法、终端及计算机可读存储介质
CA3224868A1 (en) * 2021-06-28 2023-01-05 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing myoc expression
AU2022408167A1 (en) 2021-12-08 2024-06-06 Regeneron Pharmaceuticals, Inc. Mutant myocilin disease model and uses thereof
WO2023114446A2 (en) * 2021-12-16 2023-06-22 Peter Koulen A machine learning-based framework using electroretinography for detecting early-stage glaucoma
CN117343152B (zh) * 2023-04-18 2024-06-04 上海本导基因技术有限公司 一种用于治疗青光眼疾病的慢病毒样颗粒
CN118546217B (zh) * 2024-07-25 2024-10-29 广州译码基因科技有限公司 靶向小梁网、角膜缘及rpe的aav衣壳蛋白及其应用
CN121449764B (zh) * 2026-01-07 2026-05-19 山东伯桢生物科技有限公司 抑制Wnt信号通路的融合蛋白及其应用

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WO2005117938A2 (en) 2004-04-13 2005-12-15 Regents Of The University Of Minnesota Methods of treating ocular conditions
WO2008075796A1 (ja) 2006-12-21 2008-06-26 Kyowa Hakko Kirin Co., Ltd. 血球回復促進剤
AU2008308784B2 (en) * 2007-10-01 2013-07-18 Arrowhead Research Corporation Self complementary AAV-mediated delivery of interfering RNA molecules to treat or prevent ocular disorders
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DK3693025T3 (da) * 2011-04-22 2022-01-10 Univ California Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf
EP2554662A1 (en) * 2011-08-05 2013-02-06 M Maria Pia Cosma Methods of treatment of retinal degeneration diseases
BR112014019741A2 (pt) * 2012-02-11 2020-12-22 Genentech, Inc Usos de um antagonista da via wnt, uso de uma terapia anticâncer, método de identificação de um indivíduo com câncer, métodos para prever, método de inibição da proliferação de uma célula de câncer, uso de um antagonista da translocação de r-spondina e antagonista da translocação de r-spondina isolado
EP3470523A1 (en) * 2012-05-09 2019-04-17 Oregon Health & Science University Adeno associated virus plasmids and vectors
IL251178B (en) 2014-09-16 2022-07-01 Genzyme Corp Adeno-associated viral vector for the treatment of glaucoma myocilin (myoc)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7097398B2 (ja) 2014-09-16 2022-07-07 ジェンザイム・コーポレーション ミオシリン（ｍｙｏｃ）緑内障を処置するためのアデノ随伴ウイルスベクター

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US20170304465A1 (en) 2017-10-26
BR112017005235A2 (pt) 2017-12-12
KR20260019013A (ko) 2026-02-09
SG10201902285SA (en) 2019-04-29
IL251178A0 (en) 2017-05-29
CA3280614A1 (en) 2026-03-02
RU2746991C2 (ru) 2021-04-23
RU2017112972A (ru) 2018-10-17
JP2017529395A (ja) 2017-10-05
RU2020111038A3 (enExample) 2020-10-15
EP3194601A1 (en) 2017-07-26
AU2015317756A1 (en) 2017-04-13
ZA201701881B (en) 2023-09-27
KR20170054493A (ko) 2017-05-17
JP7097398B2 (ja) 2022-07-07
CN116585492A (zh) 2023-08-15
SG11201702055PA (en) 2017-04-27
CN107001436A (zh) 2017-08-01
CA2961523C (en) 2026-01-13
AU2022202413A1 (en) 2022-05-05
KR102916865B1 (ko) 2026-01-23
KR20230172610A (ko) 2023-12-22
KR102612871B1 (ko) 2023-12-13
MX2023005998A (es) 2023-06-08
RU2017112972A3 (enExample) 2019-04-16
IL251178B (en) 2022-07-01
BR112017005235B1 (pt) 2023-10-31
RU2718047C2 (ru) 2020-03-30
JP2020105194A (ja) 2020-07-09
EP3194601B1 (en) 2021-10-27
CA2961523A1 (en) 2016-03-24
AU2015317756B2 (en) 2022-01-13
RU2020111038A (ru) 2020-04-29
CN107001436B (zh) 2023-01-06
SG10201913430VA (en) 2020-03-30
MX2017003426A (es) 2017-07-28
US10821193B2 (en) 2020-11-03

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