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 (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2943185C (en) | 2014-03-21 | 2025-09-16 | Genzyme Corp | Gene therapy for retinitis pigmentosa |
| HK1247641A1 (zh) | 2015-02-10 | 2018-09-28 | Genzyme Corporation | 变体RNAi |
| ES2886664T3 (es) * | 2015-12-03 | 2021-12-20 | Friedrich Miescher Institute For Biomedical Res | SynP162, un promotor para la expresión específica de genes en fotorreceptores de bastón |
| 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 |
| AU2016363830C1 (en) * | 2015-12-04 | 2022-03-24 | Centre National De La Recherche Scientifique | Promoters and uses thereof |
| AU2017227776C1 (en) * | 2016-03-01 | 2024-01-25 | The Trustees Of The University Of Pennsylvania | AAV vectors for treatment of dominant retinitis pigmentosa |
| EP3481434A4 (en) * | 2016-07-05 | 2020-06-24 | The Johns Hopkins University | 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 |
| EP3684932A1 (en) | 2017-09-22 | 2020-07-29 | Genzyme Corporation | Variant rnai |
| BR112020006149A2 (pt) * | 2017-09-27 | 2020-10-20 | Sigilon Therapeutics, Inc. | célula ativa manipulada, elemento implantável, e, composição farmacêutica. |
| 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 |
| US12162994B2 (en) | 2018-03-02 | 2024-12-10 | Sigilon Therapeutics, Inc. | Biocompatible hydrogel capsules and process for preparing same |
| 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 |
| UY38160A (es) | 2018-04-04 | 2019-11-29 | Sigilon Therapeutics Inc | Partículas implantables y métodos relacionados |
| 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 |
| WO2020180886A1 (en) * | 2019-03-04 | 2020-09-10 | The Trustees Of The University Of Pennsylvania | Neuroprotective gene therapy targeting the akt pathway |
| BR112021018354A2 (pt) * | 2019-03-21 | 2021-11-23 | Ptc Therapeutics Inc | Vetor e método para tratar síndrome de angelman |
| CN114555084A (zh) * | 2019-08-15 | 2022-05-27 | 费城儿童医院 | 用于治疗SCA1的转基因和内含子衍生miRNA联合疗法 |
| AU2021205965A1 (en) * | 2020-01-10 | 2022-09-01 | Solid Biosciences Inc. | Viral vector for combination therapy |
| KR20220133941A (ko) * | 2020-01-29 | 2022-10-05 | 젠자임 코포레이션 | 안과 유전자 치료를 위한 변형 아데노-관련 바이러스 캡시드 단백질 및 이의 사용 방법 |
| 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 |
| WO2022221405A1 (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 |
| 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 |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | 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 |
| ES2256265T3 (es) | 2000-06-01 | 2006-07-16 | University Of North Carolina At Chapel Hill | Vectores de parvovirus duplicados. |
| NZ618298A (en) | 2001-11-13 | 2015-04-24 | 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 |
| US8741650B2 (en) * | 2004-01-22 | 2014-06-03 | Dnavec Research Inc. | Methods for producing minus-strand RNA viral vectors using hybrid promoter comprising cytomegalovirus enhancer and chicken β-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 |
| 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 |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| DK2475372T4 (da) * | 2009-09-10 | 2020-11-30 | Velin Pharma As | Fremgangsmåde til fremstilling af micro-RNA og dets terapeutiske anvendelse |
| CN102947453A (zh) * | 2010-01-28 | 2013-02-27 | 费城儿童医院 | 用于病毒载体纯化的可扩缩生产平台和用于基因治疗中的如此纯化的病毒载体 |
| 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 |
| JP5704361B2 (ja) | 2010-10-27 | 2015-04-22 | 学校法人自治医科大学 | 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン |
| SG10201601110VA (en) | 2011-02-17 | 2016-03-30 | 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 |
| RU2653444C2 (ru) * | 2012-02-17 | 2018-05-08 | Дзе Чилдрен'З Хоспитал Оф Филадельфия | Композиции вектора aav и способы переноса генов в клетки, органы и ткани |
| 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 |
| WO2013176772A1 (en) | 2012-05-25 | 2013-11-28 | The Regents Of The University Of California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
| AU2013287281B2 (en) * | 2012-07-11 | 2018-04-26 | The Trustees Of The University Of Pennsylvania | AAV-mediated gene therapy for RPGR x-linked retinal degeneration |
| EP2912177A1 (en) * | 2012-10-23 | 2015-09-02 | Cornell University | Treatment of metastatic breast cancer |
| EP3052134B1 (en) | 2013-10-04 | 2021-05-05 | Merck Sharp & Dohme Corp. | Glucose-responsive insulin conjugates |
| CA2943185C (en) | 2014-03-21 | 2025-09-16 | Genzyme Corp | Gene therapy for retinitis pigmentosa |
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2015
- 2015-03-20 CA CA2943185A patent/CA2943185C/en active Active
- 2015-03-20 RS RS20230833A patent/RS64611B1/sr unknown
- 2015-03-20 MX MX2016012201A patent/MX376190B/es active IP Right Grant
- 2015-03-20 SG SG10201808218YA patent/SG10201808218YA/en unknown
- 2015-03-20 CA CA3254798A patent/CA3254798A1/en active Pending
- 2015-03-20 SG SG10201912968WA patent/SG10201912968WA/en unknown
- 2015-03-20 ES ES19186929T patent/ES2957840T3/es active Active
- 2015-03-20 DK DK19186929.6T patent/DK3628334T5/da active
- 2015-03-20 HU HUE15716232A patent/HUE046454T2/hu unknown
- 2015-03-20 PE PE2016001643A patent/PE20161252A1/es unknown
- 2015-03-20 WO PCT/US2015/021896 patent/WO2015143418A2/en not_active Ceased
- 2015-03-20 PL PL15716232T patent/PL3119437T3/pl unknown
- 2015-03-20 KR KR1020167028893A patent/KR20160127832A/ko not_active Ceased
- 2015-03-20 EP EP23176679.1A patent/EP4345165A3/en active Pending
- 2015-03-20 PT PT157162322T patent/PT3119437T/pt unknown
- 2015-03-20 RS RS20191551A patent/RS59634B1/sr unknown
- 2015-03-20 EP EP15716232.2A patent/EP3119437B1/en active Active
- 2015-03-20 AU AU2015230942A patent/AU2015230942B2/en active Active
- 2015-03-20 HU HUE19186929A patent/HUE063460T2/hu unknown
- 2015-03-20 HR HRP20192141TT patent/HRP20192141T1/hr unknown
- 2015-03-20 HR HRP20231077TT patent/HRP20231077T1/hr unknown
- 2015-03-20 EP EP19186929.6A patent/EP3628334B1/en active Active
- 2015-03-20 CR CR20160480A patent/CR20160480A/es unknown
- 2015-03-20 DK DK15716232T patent/DK3119437T3/da active
- 2015-03-20 US US15/127,757 patent/US10383953B2/en active Active
- 2015-03-20 LT LTEP15716232.2T patent/LT3119437T/lt unknown
- 2015-03-20 JP JP2016557218A patent/JP6669664B2/ja active Active
- 2015-03-20 KR KR1020227045369A patent/KR20230006039A/ko not_active Ceased
- 2015-03-20 SI SI201531019T patent/SI3119437T1/sl unknown
- 2015-03-20 NZ NZ724622A patent/NZ724622A/en unknown
- 2015-03-20 PT PT191869296T patent/PT3628334T/pt unknown
- 2015-03-20 MA MA39390A patent/MA39390B2/fr unknown
- 2015-03-20 UA UAA201610589A patent/UA120050C2/uk unknown
- 2015-03-20 MY MYPI2020002274A patent/MY203654A/en unknown
- 2015-03-20 SI SI201531966T patent/SI3628334T1/sl unknown
- 2015-03-20 FI FIEP19186929.6T patent/FI3628334T3/fi active
- 2015-03-20 SG SG11201607005UA patent/SG11201607005UA/en unknown
- 2015-03-20 EA EA201691891A patent/EA201691891A1/ru unknown
- 2015-03-20 CN CN202210690697.4A patent/CN115252823A/zh active Pending
- 2015-03-20 PL PL19186929.6T patent/PL3628334T3/pl unknown
- 2015-03-20 CN CN201580026274.0A patent/CN106456660B/zh active Active
- 2015-03-20 LT LTEP19186929.6T patent/LT3628334T/lt unknown
- 2015-03-20 ES ES15716232T patent/ES2760263T3/es active Active
- 2015-03-20 MY MYPI2016703077A patent/MY190726A/en unknown
- 2015-03-23 TW TW109103078A patent/TWI780401B/zh active
- 2015-03-23 TW TW104109152A patent/TWI706789B/zh active
- 2015-03-23 UY UY0001036044A patent/UY36044A/es not_active Application Discontinuation
- 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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