JP2017509632A5 - - Google Patents
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- Publication number
- 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
Links
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 (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PT3628334T (pt) | 2014-03-21 | 2023-09-26 | Genzyme Corp | Terapia genética para retinopatia pigmentar |
| CA3255657A1 (en) | 2015-02-10 | 2025-12-01 | Genzyme Corporation | VARIANT RNAi |
| US10994026B2 (en) | 2015-12-03 | 2021-05-04 | Friedrich Miescher Institute For Biomedical Research | SynP160, a promoter for the specific expression of genes in rod photoreceptors |
| US11059871B2 (en) | 2015-12-03 | 2021-07-13 | Friedrich Miescher Institute For Biomedical Research | SYNP162, a promoter for the specific expression of genes in rod photoreceptors |
| JP6966442B2 (ja) * | 2015-12-04 | 2021-11-17 | ソルボンヌ・ユニヴェルシテSorbonne Universite | プロモーター及びその使用 |
| IL302455A (en) * | 2016-03-01 | 2023-06-01 | Univ Florida | Adeno-associated virus vectors for the treatment of dominant retinitis pigmentosa |
| SG10202109385QA (en) * | 2016-07-05 | 2021-10-28 | Univ Johns Hopkins | Crispr/cas9-based compositions and methods for treating retinal degenerations |
| CA3056211A1 (en) * | 2017-03-21 | 2018-09-27 | Stylianos MICHALAKIS | Gene therapy for the treatment of cngb1-linked retinitis pigmentosa |
| MX2020003095A (es) | 2017-09-22 | 2020-10-15 | Genzyme Corp | Arni variante. |
| KR20200057051A (ko) * | 2017-09-27 | 2020-05-25 | 시질론 테라퓨틱스, 인크. | 활성 세포를 포함하는 방법, 조성물 및 이식 가능한 요소 |
| 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 |
| WO2019169333A1 (en) | 2018-03-02 | 2019-09-06 | Sigilon Therapeutics, Inc. | Afibrotic compounds, devices, and uses thereof |
| EP3759154A1 (en) | 2018-03-02 | 2021-01-06 | Sigilon Therapeutics, Inc. | Biocompatible hydrogel capsules and process for preparing same |
| EP3768327A4 (en) * | 2018-03-23 | 2022-04-13 | The Trustees of Columbia University in the City of New York | GENE EDIT FOR AUTOSOMAL DOMINANT DISEASES |
| CN112203646A (zh) | 2018-04-04 | 2021-01-08 | 西吉隆医疗股份有限公司 | 可植入颗粒和相关方法 |
| EP3802836A4 (en) | 2018-06-01 | 2022-10-19 | University of Florida Research Foundation, Incorporated | COMPOSITIONS AND METHODS FOR THE 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 |
| AU2020232692A1 (en) * | 2019-03-04 | 2021-09-30 | The Trustees Of The University Of Pennsylvania | Neuroprotective gene therapy targeting the AKT pathway |
| EP3941530A4 (en) * | 2019-03-21 | 2022-12-14 | PTC Therapeutics, Inc. | ANGELMAN SYNDROME TREATMENT VECTOR AND PROCEDURES |
| US12649930B2 (en) | 2019-03-27 | 2026-06-09 | Sigilon Therapeutics, Inc. | Compositions, devices, and methods for treating Fabry disease |
| WO2021030745A1 (en) * | 2019-08-15 | 2021-02-18 | The Children's Hospital Of Philadelphia | Combined transgene and intron-derived mirna therapy for treatment of sca1 |
| CN115279421A (zh) * | 2020-01-10 | 2022-11-01 | 坚固生物科技公司 | 用于联合疗法的病毒载体 |
| WO2021155137A1 (en) * | 2020-01-29 | 2021-08-05 | Genzyme Corporation | Modified adeno-associated viral capsid proteins for ocular gene therapy and methods of use thereof |
| 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 |
| IL299927A (en) * | 2020-07-29 | 2023-03-01 | Univ Florida | Improved adeno-associated virus-mediated therapies for X-linked retinoschisis |
| US20220331241A1 (en) * | 2021-04-16 | 2022-10-20 | 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 |
| WO2024211480A2 (en) * | 2023-04-05 | 2024-10-10 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
| CN120098992A (zh) * | 2023-12-05 | 2025-06-06 | 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 |
| US6995006B2 (en) | 1997-09-05 | 2006-02-07 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6596535B1 (en) | 1999-08-09 | 2003-07-22 | Targeted Genetics Corporation | Metabolically activated recombinant viral vectors and methods for the preparation and use |
| US7465583B2 (en) | 2000-06-01 | 2008-12-16 | The University Of North Carolina At Chapel Hill | Duplexed parvovirus vectors |
| CN103589692B (zh) | 2001-11-13 | 2018-07-24 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| EP2434020B1 (en) * | 2004-01-22 | 2014-11-05 | Dnavec Research Inc. | Method of producing Paramyxoviridae viral vectors using a hybrid promoter comprising a cytomegalovirus enhancer and a chicken beta-actin promoter |
| 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 |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| US8257969B2 (en) * | 2007-04-12 | 2012-09-04 | 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 |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| AU2010294197C9 (en) | 2009-09-10 | 2018-09-13 | Velin-Pharma A/S | Method for the preparation of micro-RNA and its therapeutic application |
| EP2529020B1 (en) * | 2010-01-28 | 2018-04-25 | The Children's Hospital of Philadelphia | A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy |
| 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 |
| US10738326B2 (en) | 2010-10-27 | 2020-08-11 | Jichi Medical University | Adeno-associated virus vector for gene transfer to nervous system cells |
| CN103502458B (zh) | 2011-02-17 | 2016-11-16 | 宾夕法尼亚大学托管会 | 用于改变组织特异性和改善aav9-介导的基因转移的组合物和方法 |
| 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 |
| BR112014020325A2 (pt) * | 2012-02-17 | 2017-08-08 | Childrens Hospital Philadelphia | composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos |
| WO2013151664A1 (en) * | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins |
| US9127274B2 (en) * | 2012-04-26 | 2015-09-08 | Alnylam Pharmaceuticals, Inc. | Serpinc1 iRNA compositions and methods of use thereof |
| ES2728782T3 (es) * | 2012-05-25 | 2019-10-28 | Univ California | Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN |
| CN105120901A (zh) | 2012-07-11 | 2015-12-02 | 宾夕法尼亚大学托管会 | Rpgr x染色体连锁视网膜退化的aav介导的基因治疗 |
| EP2912177A1 (en) * | 2012-10-23 | 2015-09-02 | Cornell University | Treatment of metastatic breast cancer |
| US9884125B2 (en) | 2013-10-04 | 2018-02-06 | Merck Sharp & Dohme Corp. | Glucose-responsive insulin conjugates |
| PT3628334T (pt) * | 2014-03-21 | 2023-09-26 | Genzyme Corp | Terapia genética para retinopatia pigmentar |
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2015
- 2015-03-20 PT PT191869296T patent/PT3628334T/pt unknown
- 2015-03-20 UA UAA201610589A patent/UA120050C2/uk unknown
- 2015-03-20 HU HUE15716232A patent/HUE046454T2/hu unknown
- 2015-03-20 PE PE2016001643A patent/PE20161252A1/es unknown
- 2015-03-20 KR KR1020227045369A patent/KR20230006039A/ko not_active Ceased
- 2015-03-20 SI SI201531019T patent/SI3119437T1/sl unknown
- 2015-03-20 MY MYPI2016703077A patent/MY190726A/en unknown
- 2015-03-20 CA CA2943185A patent/CA2943185C/en active Active
- 2015-03-20 MY MYPI2020002274A patent/MY203654A/en unknown
- 2015-03-20 CN CN201580026274.0A patent/CN106456660B/zh active Active
- 2015-03-20 LT LTEP15716232.2T patent/LT3119437T/lt unknown
- 2015-03-20 NZ NZ724622A patent/NZ724622A/en unknown
- 2015-03-20 KR KR1020257039649A patent/KR20250172735A/ko active Pending
- 2015-03-20 KR KR1020167028893A patent/KR20160127832A/ko not_active Ceased
- 2015-03-20 SI SI201531966T patent/SI3628334T1/sl unknown
- 2015-03-20 HR HRP20192141TT patent/HRP20192141T1/hr unknown
- 2015-03-20 FI FIEP19186929.6T patent/FI3628334T3/fi active
- 2015-03-20 MX MX2016012201A patent/MX376190B/es active IP Right Grant
- 2015-03-20 RS RS20230833A patent/RS64611B1/sr unknown
- 2015-03-20 EP EP15716232.2A patent/EP3119437B1/en active Active
- 2015-03-20 SG SG11201607005UA patent/SG11201607005UA/en unknown
- 2015-03-20 ES ES19186929T patent/ES2957840T3/es active Active
- 2015-03-20 EP EP23176679.1A patent/EP4345165A3/en active Pending
- 2015-03-20 MA MA39390A patent/MA39390B2/fr unknown
- 2015-03-20 CR CR20160480A patent/CR20160480A/es unknown
- 2015-03-20 RS RS20191551A patent/RS59634B1/sr unknown
- 2015-03-20 ES ES15716232T patent/ES2760263T3/es active Active
- 2015-03-20 CN CN202210690697.4A patent/CN115252823A/zh active Pending
- 2015-03-20 DK DK19186929.6T patent/DK3628334T5/da active
- 2015-03-20 SG SG10201808218YA patent/SG10201808218YA/en unknown
- 2015-03-20 US US15/127,757 patent/US10383953B2/en active Active
- 2015-03-20 AU AU2015230942A patent/AU2015230942B2/en active Active
- 2015-03-20 WO PCT/US2015/021896 patent/WO2015143418A2/en not_active Ceased
- 2015-03-20 CA CA3254798A patent/CA3254798A1/en active Pending
- 2015-03-20 PL PL15716232T patent/PL3119437T3/pl unknown
- 2015-03-20 LT LTEP19186929.6T patent/LT3628334T/lt unknown
- 2015-03-20 HU HUE19186929A patent/HUE063460T2/hu unknown
- 2015-03-20 JP JP2016557218A patent/JP6669664B2/ja active Active
- 2015-03-20 PL PL19186929.6T patent/PL3628334T3/pl unknown
- 2015-03-20 SG SG10201912968WA patent/SG10201912968WA/en unknown
- 2015-03-20 EP EP19186929.6A patent/EP3628334B1/en active Active
- 2015-03-20 DK DK15716232T patent/DK3119437T3/da active
- 2015-03-20 EA EA201691891A patent/EA201691891A1/ru unknown
- 2015-03-20 HR HRP20231077TT patent/HRP20231077T1/hr unknown
- 2015-03-20 PT PT157162322T patent/PT3119437T/pt unknown
- 2015-03-23 TW TW104109152A patent/TWI706789B/zh active
- 2015-03-23 UY UY0001036044A patent/UY36044A/es not_active Application Discontinuation
- 2015-03-23 TW TW109103078A patent/TWI780401B/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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