JP2017529395A5 - - Google Patents
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- Publication number
- JP2017529395A5 JP2017529395A5 JP2017534525A JP2017534525A JP2017529395A5 JP 2017529395 A5 JP2017529395 A5 JP 2017529395A5 JP 2017534525 A JP2017534525 A JP 2017534525A JP 2017534525 A JP2017534525 A JP 2017534525A JP 2017529395 A5 JP2017529395 A5 JP 2017529395A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- seq
- myoc
- aav2
- amino acid
- 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.)
- Granted
Links
- 239000008194 pharmaceutical composition Substances 0.000 claims 36
- 239000002245 particle Substances 0.000 claims 35
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 20
- 125000003275 alpha amino acid group Chemical group 0.000 claims 20
- 210000000234 capsid Anatomy 0.000 claims 19
- 108091030071 RNAI Proteins 0.000 claims 13
- 230000009368 gene silencing by RNA Effects 0.000 claims 13
- 108020004707 nucleic acids Proteins 0.000 claims 10
- 102000039446 nucleic acids Human genes 0.000 claims 10
- 150000007523 nucleic acids Chemical class 0.000 claims 10
- 239000013598 vector Substances 0.000 claims 10
- 241000124008 Mammalia Species 0.000 claims 9
- 208000010412 Glaucoma Diseases 0.000 claims 8
- 210000001585 trabecular meshwork Anatomy 0.000 claims 7
- 239000013607 AAV vector Substances 0.000 claims 6
- 241000702421 Dependoparvovirus Species 0.000 claims 5
- 238000006467 substitution reaction Methods 0.000 claims 5
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims 4
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 4
- 208000030533 eye disease Diseases 0.000 claims 4
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 3
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 3
- 108090000565 Capsid Proteins Proteins 0.000 claims 3
- 102100023321 Ceruloplasmin Human genes 0.000 claims 3
- 206010030348 Open-Angle Glaucoma Diseases 0.000 claims 3
- 102000014450 RNA Polymerase III Human genes 0.000 claims 3
- 108010078067 RNA Polymerase III Proteins 0.000 claims 3
- 108091027967 Small hairpin RNA Proteins 0.000 claims 3
- 108700019146 Transgenes Proteins 0.000 claims 3
- 102000013814 Wnt Human genes 0.000 claims 3
- 230000002401 inhibitory effect Effects 0.000 claims 3
- 201000006366 primary open angle glaucoma Diseases 0.000 claims 3
- 230000011664 signaling Effects 0.000 claims 3
- 239000004055 small Interfering RNA Substances 0.000 claims 3
- 230000001225 therapeutic effect Effects 0.000 claims 3
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 2
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 2
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 2
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 2
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 2
- 241000649047 Adeno-associated virus 12 Species 0.000 claims 2
- 241000283690 Bos taurus Species 0.000 claims 2
- 101000825954 Homo sapiens R-spondin-1 Proteins 0.000 claims 2
- 101000825960 Homo sapiens R-spondin-3 Proteins 0.000 claims 2
- 101000825962 Homo sapiens R-spondin-4 Proteins 0.000 claims 2
- 101100531974 Homo sapiens RSPO2 gene Proteins 0.000 claims 2
- 208000022873 Ocular disease Diseases 0.000 claims 2
- 239000003795 chemical substances by application Substances 0.000 claims 2
- 102000050526 human RSPO1 Human genes 0.000 claims 2
- 102000054144 human RSPO3 Human genes 0.000 claims 2
- 102000048108 human RSPO4 Human genes 0.000 claims 2
- 238000002347 injection Methods 0.000 claims 2
- 239000007924 injection Substances 0.000 claims 2
- 241000283707 Capra Species 0.000 claims 1
- HTTJABKRGRZYRN-UHFFFAOYSA-N Heparin Chemical compound OC1C(NC(=O)C)C(O)OC(COS(O)(=O)=O)C1OC1C(OS(O)(=O)=O)C(O)C(OC2C(C(OS(O)(=O)=O)C(OC3C(C(O)C(O)C(O3)C(O)=O)OS(O)(=O)=O)C(CO)O2)NS(O)(=O)=O)C(C(O)=O)O1 HTTJABKRGRZYRN-UHFFFAOYSA-N 0.000 claims 1
- OUYCCCASQSFEME-QMMMGPOBSA-N L-tyrosine Chemical compound OC(=O)[C@@H](N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-QMMMGPOBSA-N 0.000 claims 1
- 210000002159 anterior chamber Anatomy 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- 230000002708 enhancing effect Effects 0.000 claims 1
- 230000002068 genetic effect Effects 0.000 claims 1
- 229960002897 heparin Drugs 0.000 claims 1
- 229920000669 heparin Polymers 0.000 claims 1
- 230000000366 juvenile effect Effects 0.000 claims 1
- 230000035772 mutation Effects 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 230000008685 targeting Effects 0.000 claims 1
- OUYCCCASQSFEME-UHFFFAOYSA-N tyrosine Natural products OC(=O)C(N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-UHFFFAOYSA-N 0.000 claims 1
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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017529395A JP2017529395A (ja) | 2017-10-05 |
| JP2017529395A5 true JP2017529395A5 (cg-RX-API-DMAC7.html) | 2018-10-25 |
| JP6673923B2 JP6673923B2 (ja) | 2020-03-25 |
Family
ID=58640637
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017534525A Active JP6673923B2 (ja) | 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 |
| 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 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US10821193B2 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3194601B1 (cg-RX-API-DMAC7.html) |
| JP (2) | JP6673923B2 (cg-RX-API-DMAC7.html) |
| KR (2) | KR102612871B1 (cg-RX-API-DMAC7.html) |
| CN (2) | CN107001436B (cg-RX-API-DMAC7.html) |
| AU (2) | AU2015317756B2 (cg-RX-API-DMAC7.html) |
| BR (1) | BR112017005235B1 (cg-RX-API-DMAC7.html) |
| IL (1) | IL251178B (cg-RX-API-DMAC7.html) |
| MX (2) | MX2017003426A (cg-RX-API-DMAC7.html) |
| RU (2) | RU2718047C2 (cg-RX-API-DMAC7.html) |
| SG (3) | SG10201913430VA (cg-RX-API-DMAC7.html) |
| ZA (1) | ZA201701881B (cg-RX-API-DMAC7.html) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10821193B2 (en) | 2014-09-16 | 2020-11-03 | Genzyme Corporation | Adeno-associated viral vectors for treating myocilin (MYOC) glaucoma |
| WO2019155833A1 (ja) * | 2018-02-07 | 2019-08-15 | 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 |
| WO2019165241A1 (en) * | 2018-02-22 | 2019-08-29 | University Of South Florida | Grp94 inhibitors to treat steroid-induced ocular hypertensions and glaucomas |
| JP2022522756A (ja) | 2019-02-28 | 2022-04-20 | 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 | 治療薬の送達のためのアデノ随伴ウイルスベクター |
| KR20230117177A (ko) * | 2020-12-03 | 2023-08-07 | 유니버시티 오브 매사추세츠 | 진행성 골화성 섬유이형성증을 위한 신규 유전자 치료제의개발 |
| 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 |
| JP2025500291A (ja) * | 2021-12-16 | 2025-01-09 | クーレン ピーター | 初期段階緑内障を検出するための網膜電図を使用した機械学習ベースのフレームワーク |
| 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 |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20050130145A1 (en) | 2001-10-03 | 2005-06-16 | Henry Yue | Secreted proteins |
| GB0403600D0 (en) * | 2004-02-18 | 2004-03-24 | Trinity College Dublin | Methods and reagents for treating disease |
| 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 |
| CN101815536A (zh) * | 2007-10-01 | 2010-08-25 | 爱尔康研究有限公司 | 自补aav介导的干扰rna分子递送以治疗或预防眼病 |
| GB201106395D0 (en) * | 2011-04-14 | 2011-06-01 | Hubrecht Inst | Compounds |
| JP6072772B2 (ja) * | 2011-04-22 | 2017-02-01 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 変異体キャプシドを有するアデノ関連ウイルスビリオンおよびその使用方法 |
| EP2554662A1 (en) * | 2011-08-05 | 2013-02-06 | M Maria Pia Cosma | Methods of treatment of retinal degeneration diseases |
| RU2014133069A (ru) * | 2012-02-11 | 2016-04-10 | Дженентек, Инк. | Транслокации r-спондина и способы с их использованием |
| JP6385920B2 (ja) * | 2012-05-09 | 2018-09-05 | オレゴン ヘルス アンド サイエンス ユニバーシティー | アデノ随伴ウイルスプラスミド及びベクター |
| US10821193B2 (en) | 2014-09-16 | 2020-11-03 | Genzyme Corporation | Adeno-associated viral vectors for treating myocilin (MYOC) glaucoma |
-
2015
- 2015-09-16 US US15/511,595 patent/US10821193B2/en active Active
- 2015-09-16 KR KR1020177010012A patent/KR102612871B1/ko active Active
- 2015-09-16 SG SG10201913430VA patent/SG10201913430VA/en unknown
- 2015-09-16 IL IL251178A patent/IL251178B/en unknown
- 2015-09-16 RU RU2017112972A patent/RU2718047C2/ru active
- 2015-09-16 AU AU2015317756A patent/AU2015317756B2/en active Active
- 2015-09-16 RU RU2020111038A patent/RU2746991C2/ru active
- 2015-09-16 JP JP2017534525A patent/JP6673923B2/ja active Active
- 2015-09-16 CN CN201580061882.5A patent/CN107001436B/zh active Active
- 2015-09-16 MX MX2017003426A patent/MX2017003426A/es unknown
- 2015-09-16 EP EP15772133.3A patent/EP3194601B1/en active Active
- 2015-09-16 SG SG10201902285SA patent/SG10201902285SA/en unknown
- 2015-09-16 BR BR112017005235-0A patent/BR112017005235B1/pt active IP Right Grant
- 2015-09-16 SG SG11201702055PA patent/SG11201702055PA/en unknown
- 2015-09-16 CN CN202211533202.3A patent/CN116585492A/zh active Pending
- 2015-09-16 KR KR1020237042363A patent/KR20230172610A/ko active Pending
-
2017
- 2017-03-15 MX MX2023005998A patent/MX2023005998A/es unknown
- 2017-03-16 ZA ZA2017/01881A patent/ZA201701881B/en unknown
-
2020
- 2020-03-04 JP JP2020036344A patent/JP7097398B2/ja active Active
-
2022
- 2022-04-12 AU AU2022202413A patent/AU2022202413A1/en not_active Abandoned
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