JP2017509632A5 - - Google Patents
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- JP2017509632A5 JP2017509632A5 JP2016557218A JP2016557218A JP2017509632A5 JP 2017509632 A5 JP2017509632 A5 JP 2017509632A5 JP 2016557218 A JP2016557218 A JP 2016557218A JP 2016557218 A JP2016557218 A JP 2016557218A JP 2017509632 A5 JP2017509632 A5 JP 2017509632A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- raav
- seq
- acid encoding
- mir
- 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
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Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461969027P | 2014-03-21 | 2014-03-21 | |
| US61/969,027 | 2014-03-21 | ||
| PCT/US2015/021896 WO2015143418A2 (en) | 2014-03-21 | 2015-03-20 | Gene therapy for retinitis pigmentosa |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019224202A Division JP7048563B2 (ja) | 2014-03-21 | 2019-12-12 | 網膜色素変性症のための遺伝子治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017509632A JP2017509632A (ja) | 2017-04-06 |
| JP2017509632A5 true JP2017509632A5 (enExample) | 2018-04-26 |
| JP6669664B2 JP6669664B2 (ja) | 2020-03-18 |
Family
ID=52829343
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016557218A Active JP6669664B2 (ja) | 2014-03-21 | 2015-03-20 | 網膜色素変性症のための遺伝子治療 |
| JP2019224202A Active JP7048563B2 (ja) | 2014-03-21 | 2019-12-12 | 網膜色素変性症のための遺伝子治療 |
| JP2022046280A Active JP7534348B2 (ja) | 2014-03-21 | 2022-03-23 | 網膜色素変性症のための遺伝子治療 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019224202A Active JP7048563B2 (ja) | 2014-03-21 | 2019-12-12 | 網膜色素変性症のための遺伝子治療 |
| JP2022046280A Active JP7534348B2 (ja) | 2014-03-21 | 2022-03-23 | 網膜色素変性症のための遺伝子治療 |
Country Status (36)
Families Citing this family (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK3628334T5 (da) | 2014-03-21 | 2024-09-02 | Genzyme Corp | Genterapi til behandling af retinitis pigmentosa |
| US10450563B2 (en) | 2015-02-10 | 2019-10-22 | Genzyme Corporation | Variant RNAi |
| CN108474001B (zh) * | 2015-12-03 | 2022-04-08 | 弗里德里克·米谢尔生物医学研究所 | SynP160，用于基因在视杆光感受器中特异性表达的启动子 |
| EP3383440B1 (en) * | 2015-12-03 | 2021-06-30 | Friedrich Miescher Institute for Biomedical Research | Synp162, a promoter for the specific expression of genes in rod photoreceptors |
| EP3384036B1 (en) * | 2015-12-04 | 2021-02-24 | Sorbonne Université | Promoters and uses thereof |
| US11118185B2 (en) * | 2016-03-01 | 2021-09-14 | University Of Florida Research Foundation, Incorporated | AAV vectors for treatment of dominant retinitis pigmentosa |
| SG11201900049QA (en) * | 2016-07-05 | 2019-02-27 | Univ Johns Hopkins | Crispr/cas9-based compositions and methods for treating retinal degenerations |
| UY37636A (es) * | 2017-03-21 | 2018-10-31 | Univ Muenchen Ludwig Maximilians | Terapia génica para el tratamiento de retinitis pigmentosa ligada a cngb1 |
| IL273394B2 (en) | 2017-09-22 | 2025-04-01 | Genzyme Corp | RNAI variant |
| CN111372612A (zh) * | 2017-09-27 | 2020-07-03 | 西吉隆医疗股份有限公司 | 包含活性细胞的方法、组合物和可植入元件 |
| CN107961380B (zh) * | 2017-11-23 | 2020-05-05 | 清华大学 | 试剂在制备药物中的用途、筛选药物的方法以及药物组合物 |
| EP3759154A1 (en) | 2018-03-02 | 2021-01-06 | Sigilon Therapeutics, Inc. | Biocompatible hydrogel capsules and process for preparing same |
| AU2019226587A1 (en) | 2018-03-02 | 2020-09-03 | Sigilon Therapeutics, Inc. | Afibrotic compounds, devices, and uses thereof |
| US20210017509A1 (en) * | 2018-03-23 | 2021-01-21 | The Trustees Of Columbia University In The City Of New York | Gene Editing for Autosomal Dominant Diseases |
| WO2019195055A1 (en) | 2018-04-04 | 2019-10-10 | Sigilon Therapeutics, Inc. | Implantable particles and related methods |
| WO2019232517A1 (en) | 2018-06-01 | 2019-12-05 | University Of Florida Research Foundation, Incorporated | Compositions and methods for treatment of dominant retinitis pigmentosa |
| GB201817469D0 (en) * | 2018-10-26 | 2018-12-12 | Univ Oxford Innovation Ltd | Gene therapy for retinal disease |
| CN111518813B (zh) * | 2019-02-03 | 2023-04-28 | 武汉纽福斯生物科技有限公司 | 视紫红质的编码序列、其表达载体构建及其应用 |
| JP7637060B2 (ja) * | 2019-03-04 | 2025-02-27 | 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 | Ａｋｔ経路を標的とする神経保護遺伝子療法 |
| JP2022525564A (ja) * | 2019-03-21 | 2022-05-17 | ピーティーシー セラピューティクス， インコーポレイテッド | アンジェルマン症候群を治療するためのベクターおよび方法 |
| JP2022545378A (ja) * | 2019-08-15 | 2022-10-27 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア | ＳＣＡ１の治療のための、導入遺伝子およびイントロン由来ｍｉＲＮＡを組み合わせた治療法 |
| EP4087617A4 (en) * | 2020-01-10 | 2024-05-01 | Solid Biosciences Inc. | VIRAL VECTOR FOR COMBINATION THERAPY |
| JP2023513004A (ja) * | 2020-01-29 | 2023-03-30 | ジェンザイム・コーポレーション | 眼科遺伝子療法のための改変されたアデノ随伴ウイルスカプシドタンパク質およびその使用方法 |
| CN114521214A (zh) * | 2020-07-24 | 2022-05-20 | 尔知渃米斯股份有限公司 | 视紫红质转录体特异性反式剪接核酶及其用途 |
| CA3190214A1 (en) * | 2020-07-29 | 2022-02-03 | University Of Florida Research Foundation, Incorporated | Improved aav-mediated x-linked retinoschisis therapies |
| EP4323390A1 (en) * | 2021-04-16 | 2024-02-21 | Regeneron Pharmaceuticals, Inc. | Treatment of x-linked juvenile retinoschisis |
| TWI832531B (zh) * | 2022-11-02 | 2024-02-11 | 慈濟學校財團法人慈濟大學 | Rip1抑制劑或mlkl抑制劑用於治療或預防遺傳性視網膜失養症的用途 |
| CN117625619B (zh) * | 2023-12-05 | 2024-11-12 | 云舟生物科技(广州)股份有限公司 | 核酸分子及其作为特异启动子的应用 |
| CN120230798A (zh) * | 2023-12-29 | 2025-07-01 | 上海朗昇生物科技有限公司 | 用于视网膜基因递送的重组腺相关病毒载体及其应用 |
Family Cites Families (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5262529A (en) * | 1990-01-24 | 1993-11-16 | President And Fellows Of Harvard College | Diagnosis of hereditary retinal degenerative diseases |
| US5985583A (en) * | 1992-06-23 | 1999-11-16 | Mount Sinai School Of Medicine Of The City University Of New York | Cloning and expression of gonadotropin-releasing hormone receptor |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| EP2369002A1 (en) | 1999-08-09 | 2011-09-28 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs |
| ATE318923T1 (de) | 2000-06-01 | 2006-03-15 | Univ North Carolina | Doppelsträngige parvovirus-vektoren |
| SG10202108118RA (en) | 2001-11-13 | 2021-08-30 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| WO2005071092A1 (ja) * | 2004-01-22 | 2005-08-04 | Dnavec Research Inc. | サイトメガロウイルスエンハンサーおよびニワトリβ-アクチンプロモーターを含むハイブリッドプロモーターを利用したマイナス鎖RNAウイルスベクターの製造方法 |
| US7765583B2 (en) | 2005-02-28 | 2010-07-27 | France Telecom | System and method for managing virtual user domains |
| US8283151B2 (en) | 2005-04-29 | 2012-10-09 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes |
| WO2007120542A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid library and aav capsid proteins |
| CA2683469C (en) * | 2007-04-12 | 2019-11-26 | The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth | Genetic suppression and replacement |
| US20090214478A1 (en) | 2008-02-21 | 2009-08-27 | Alberto Auricchio | Method of treating ocular diseases by gene therapy |
| US9217155B2 (en) * | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| WO2010138263A2 (en) * | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| WO2011029903A1 (en) | 2009-09-10 | 2011-03-17 | Flemming Velin | Method for the preparation of micro-rna and its therapeutic application |
| ES2680915T3 (es) | 2010-01-28 | 2018-09-11 | The Children's Hospital Of Philadelphia | Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica |
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| CN107828820B (zh) | 2010-10-27 | 2022-06-07 | 学校法人自治医科大学 | 用于向神经系统细胞导入基因的腺相关病毒粒子 |
| SG10202007803QA (en) | 2011-02-17 | 2020-09-29 | Univ Pennsylvania | Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer |
| JP5884152B2 (ja) | 2011-07-29 | 2016-03-15 | シャープ株式会社 | 基地局、端末、通信システムおよび通信方法 |
| ITRM20110685A1 (it) * | 2011-12-23 | 2013-06-24 | Internat Ct For Genetic En Gineering And | Microrna per la rigenerazione cardiaca attraverso l induzione della proliferazione dei miociti cardiaci |
| KR102057540B1 (ko) * | 2012-02-17 | 2019-12-19 | 더 칠드런스 호스피탈 오브 필라델피아 | 세포, 기관 및 조직으로의 유전자 전이를 위한 ａａｖ 벡터 조성물 및 방법 |
| WO2013151663A1 (en) * | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of membrane proteins |
| US9127274B2 (en) * | 2012-04-26 | 2015-09-08 | Alnylam Pharmaceuticals, Inc. | Serpinc1 iRNA compositions and methods of use thereof |
| LT3401400T (lt) * | 2012-05-25 | 2019-06-10 | The Regents Of The University Of California | Būdai ir kompozicijos, skirtos rnr molekulės nukreipiamai tikslinės dnr modifikacijai ir rnr molekulės nukreipiamam transkripcijos moduliavimui |
| EP2872183B1 (en) | 2012-07-11 | 2018-09-26 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for rpgr x-linked retinal degeneration |
| CA2889020A1 (en) * | 2012-10-23 | 2014-05-01 | Cornell University | Treatment of metastatic breast cancer |
| AU2014329567B2 (en) | 2013-10-04 | 2019-07-25 | Merck Sharp & Dohme Corp. | Glucose-responsive insulin conjugates |
| DK3628334T5 (da) | 2014-03-21 | 2024-09-02 | Genzyme Corp | Genterapi til behandling af retinitis pigmentosa |
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2015
- 2015-03-20 DK DK19186929.6T patent/DK3628334T5/da active
- 2015-03-20 HU HUE19186929A patent/HUE063460T2/hu unknown
- 2015-03-20 HR HRP20192141TT patent/HRP20192141T1/hr unknown
- 2015-03-20 LT LTEP19186929.6T patent/LT3628334T/lt unknown
- 2015-03-20 SG SG10201912968WA patent/SG10201912968WA/en unknown
- 2015-03-20 SG SG11201607005UA patent/SG11201607005UA/en unknown
- 2015-03-20 AU AU2015230942A patent/AU2015230942B2/en active Active
- 2015-03-20 PE PE2016001643A patent/PE20161252A1/es unknown
- 2015-03-20 US US15/127,757 patent/US10383953B2/en active Active
- 2015-03-20 EA EA201691891A patent/EA201691891A1/ru unknown
- 2015-03-20 MY MYPI2016703077A patent/MY190726A/en unknown
- 2015-03-20 HR HRP20231077TT patent/HRP20231077T1/hr unknown
- 2015-03-20 PL PL19186929.6T patent/PL3628334T3/pl unknown
- 2015-03-20 EP EP19186929.6A patent/EP3628334B1/en active Active
- 2015-03-20 RS RS20191551A patent/RS59634B1/sr unknown
- 2015-03-20 UA UAA201610589A patent/UA120050C2/uk unknown
- 2015-03-20 FI FIEP19186929.6T patent/FI3628334T3/fi active
- 2015-03-20 DK DK15716232T patent/DK3119437T3/da active
- 2015-03-20 HU HUE15716232A patent/HUE046454T2/hu unknown
- 2015-03-20 PL PL15716232T patent/PL3119437T3/pl unknown
- 2015-03-20 CA CA2943185A patent/CA2943185C/en active Active
- 2015-03-20 KR KR1020227045369A patent/KR20230006039A/ko not_active Ceased
- 2015-03-20 NZ NZ724622A patent/NZ724622A/en unknown
- 2015-03-20 CN CN202210690697.4A patent/CN115252823A/zh active Pending
- 2015-03-20 LT LTEP15716232.2T patent/LT3119437T/lt unknown
- 2015-03-20 EP EP23176679.1A patent/EP4345165A3/en active Pending
- 2015-03-20 MX MX2016012201A patent/MX376190B/es active IP Right Grant
- 2015-03-20 KR KR1020257039649A patent/KR20250172735A/ko active Pending
- 2015-03-20 CA CA3254798A patent/CA3254798A1/en active Pending
- 2015-03-20 PT PT191869296T patent/PT3628334T/pt unknown
- 2015-03-20 EP EP15716232.2A patent/EP3119437B1/en active Active
- 2015-03-20 CN CN201580026274.0A patent/CN106456660B/zh active Active
- 2015-03-20 ES ES15716232T patent/ES2760263T3/es active Active
- 2015-03-20 MY MYPI2020002274A patent/MY203654A/en unknown
- 2015-03-20 SI SI201531019T patent/SI3119437T1/sl unknown
- 2015-03-20 ES ES19186929T patent/ES2957840T3/es active Active
- 2015-03-20 RS RS20230833A patent/RS64611B1/sr unknown
- 2015-03-20 MA MA39390A patent/MA39390B2/fr unknown
- 2015-03-20 SI SI201531966T patent/SI3628334T1/sl unknown
- 2015-03-20 WO PCT/US2015/021896 patent/WO2015143418A2/en not_active Ceased
- 2015-03-20 CR CR20160480A patent/CR20160480A/es unknown
- 2015-03-20 JP JP2016557218A patent/JP6669664B2/ja active Active
- 2015-03-20 KR KR1020167028893A patent/KR20160127832A/ko not_active Ceased
- 2015-03-20 PT PT157162322T patent/PT3119437T/pt unknown
- 2015-03-20 SG SG10201808218YA patent/SG10201808218YA/en unknown
- 2015-03-23 TW TW109103078A patent/TWI780401B/zh active
- 2015-03-23 UY UY0001036044A patent/UY36044A/es not_active Application Discontinuation
- 2015-03-23 TW TW104109152A patent/TWI706789B/zh active
- 2015-03-25 AR ARP150100879A patent/AR099837A1/es active IP Right Grant
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2016
- 2016-08-24 PH PH1/2016/501684A patent/PH12016501684B1/en unknown
- 2016-08-25 ZA ZA2016/05924A patent/ZA201605924B/en unknown
- 2016-08-30 IL IL247543A patent/IL247543B/en active IP Right Grant
- 2016-09-09 DO DO2016000237A patent/DOP2016000237A/es unknown
- 2016-09-15 CL CL2016002333A patent/CL2016002333A1/es unknown
- 2016-09-20 MX MX2020010694A patent/MX2020010694A/es unknown
- 2016-10-21 EC ECIEPI201683000A patent/ECSP16083000A/es unknown
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2019
- 2019-07-03 US US16/503,091 patent/US11103598B2/en active Active
- 2019-12-12 JP JP2019224202A patent/JP7048563B2/ja active Active
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2020
- 2020-07-08 IL IL275918A patent/IL275918B/en unknown
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2021
- 2021-02-15 AU AU2021200988A patent/AU2021200988B2/en active Active
- 2021-07-11 IL IL284741A patent/IL284741B2/en unknown
- 2021-07-22 US US17/383,052 patent/US12201698B2/en active Active
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2022
- 2022-03-23 JP JP2022046280A patent/JP7534348B2/ja active Active
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2024
- 2024-10-30 AU AU2024227776A patent/AU2024227776A1/en active Pending
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