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 (OSRAM) | 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 (OSRAM) |
| EP (1) | EP3194601B1 (OSRAM) |
| JP (2) | JP6673923B2 (OSRAM) |
| KR (2) | KR102612871B1 (OSRAM) |
| CN (2) | CN107001436B (OSRAM) |
| AU (2) | AU2015317756B2 (OSRAM) |
| BR (1) | BR112017005235B1 (OSRAM) |
| IL (1) | IL251178B (OSRAM) |
| MX (2) | MX2017003426A (OSRAM) |
| RU (2) | RU2746991C2 (OSRAM) |
| SG (3) | SG10201913430VA (OSRAM) |
| ZA (1) | ZA201701881B (OSRAM) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102612871B1 (ko) | 2014-09-16 | 2023-12-13 | 젠자임 코포레이션 | 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터 |
| US11845952B2 (en) * | 2018-02-07 | 2023-12-19 | Nippon Medical School Foundation | Adeno-associated virus vector |
| US20210030719A1 (en) * | 2018-02-22 | 2021-02-04 | 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 |
| WO2022120080A1 (en) * | 2020-12-03 | 2022-06-09 | University Of Massachusetts | Development of novel gene therapeutics 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 |
| US20240318175A1 (en) * | 2021-06-28 | 2024-09-26 | Alnylam Pharmaceuticals, Inc. | Compositions and Methods for Silencing MYOC Expression |
| US20250087355A1 (en) * | 2021-12-16 | 2025-03-13 | 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 |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2005514917A (ja) | 2001-10-03 | 2005-05-26 | インサイト・ゲノミックス・インコーポレイテッド | 分泌タンパク質 |
| 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 |
| WO2009046059A1 (en) | 2007-10-01 | 2009-04-09 | Alcon Research, Ltd. | Self complementary aav-mediated delivery of interfering rna molecules to treat or prevent ocular disorders |
| GB201106395D0 (en) * | 2011-04-14 | 2011-06-01 | Hubrecht Inst | Compounds |
| RS66184B1 (sr) | 2011-04-22 | 2024-12-31 | Univ California | Adeno-povezani virioni virusa sa varijantama kapsida i postupci za njihovu primenu |
| EP2554662A1 (en) * | 2011-08-05 | 2013-02-06 | M Maria Pia Cosma | Methods of treatment of retinal degeneration diseases |
| EP2812350B1 (en) * | 2012-02-11 | 2019-04-03 | F.Hoffmann-La Roche Ag | R-spondin translocations and methods using the same |
| EP2847337A4 (en) * | 2012-05-09 | 2016-04-27 | Univ Oregon Health & Science | ADENO ASSOCIATED VIRUS PLASMIDS AND VECTORS |
| KR102612871B1 (ko) | 2014-09-16 | 2023-12-13 | 젠자임 코포레이션 | 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터 |
-
2015
- 2015-09-16 KR KR1020177010012A patent/KR102612871B1/ko active Active
- 2015-09-16 US US15/511,595 patent/US10821193B2/en active Active
- 2015-09-16 BR BR112017005235-0A patent/BR112017005235B1/pt active IP Right Grant
- 2015-09-16 KR KR1020237042363A patent/KR20230172610A/ko active Pending
- 2015-09-16 CN CN201580061882.5A patent/CN107001436B/zh active Active
- 2015-09-16 RU RU2020111038A patent/RU2746991C2/ru active
- 2015-09-16 IL IL251178A patent/IL251178B/en unknown
- 2015-09-16 SG SG10201913430VA patent/SG10201913430VA/en unknown
- 2015-09-16 AU AU2015317756A patent/AU2015317756B2/en active Active
- 2015-09-16 JP JP2017534525A patent/JP6673923B2/ja active Active
- 2015-09-16 SG SG10201902285SA patent/SG10201902285SA/en unknown
- 2015-09-16 RU RU2017112972A patent/RU2718047C2/ru active
- 2015-09-16 EP EP15772133.3A patent/EP3194601B1/en active Active
- 2015-09-16 MX MX2017003426A patent/MX2017003426A/es unknown
- 2015-09-16 CN CN202211533202.3A patent/CN116585492A/zh active Pending
- 2015-09-16 SG SG11201702055PA patent/SG11201702055PA/en unknown
-
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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