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 (cg-RX-API-DMAC7.html) | 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 (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PT3119437T (pt) | 2014-03-21 | 2019-12-12 | Genzyme Corp | Terapia genética para retinopatia pigmentar |
| EA201791805A1 (ru) | 2015-02-10 | 2018-05-31 | Джензим Корпорейшн | ВАРИАНТ СРЕДСТВА ДЛЯ 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 |
| 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 |
| CA3004807C (en) * | 2015-12-04 | 2022-02-22 | Universite Pierre Et Marie Curie (Paris 6) | Promoters and uses thereof |
| CN109154002B (zh) * | 2016-03-01 | 2022-08-30 | 佛罗里达大学研究基金会有限公司 | 用于治疗显性视网膜色素变性的aav载体 |
| SG11201900049QA (en) * | 2016-07-05 | 2019-02-27 | Univ Johns Hopkins | Crispr/cas9-based compositions and methods for treating retinal degenerations |
| JP7257377B2 (ja) * | 2017-03-21 | 2023-04-13 | ミヒャラキス，スティリアノス | Ｃｎｇｂ１連鎖性網膜色素変性症の治療のための遺伝子療法 |
| AU2018335410B2 (en) * | 2017-09-22 | 2025-04-03 | Genzyme Corporation | Variant RNAi |
| 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 |
| US12162994B2 (en) | 2018-03-02 | 2024-12-10 | 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 EDITING 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 |
| 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 |
| US20220143217A1 (en) * | 2019-03-04 | 2022-05-12 | The Trustees Of The University Of Pennsylvania | Neuroprotective gene therapy targeting the akt pathway |
| KR20210145180A (ko) * | 2019-03-21 | 2021-12-01 | 피티씨 테라퓨틱스, 인크. | 엔젤만 증후군을 치료하기 위한 벡터 및 방법 |
| US20220288101A1 (en) * | 2019-08-15 | 2022-09-15 | The Children's Hospital Of Philadelphia | Combined transgene and intron-derived mirna therapy for treatment of sca1 |
| WO2021142447A1 (en) * | 2020-01-10 | 2021-07-15 | Solid Biosciences Inc. | Viral vector for combination therapy |
| MX2022009252A (es) * | 2020-01-29 | 2022-11-09 | Genzyme Corp | Proteinas de la capside de virus adeno-asociados modificadas para la terapia genica ocular y metodos de uso de las mismas. |
| EP4071247A4 (en) * | 2020-07-24 | 2023-08-23 | Rznomics Inc. | TRANSSPLICING OF RIBOZYME SPECIFIC TO RHODOPSIN TRANSCRIPTOME AND ITS USE |
| EP4188450A2 (en) * | 2020-07-29 | 2023-06-07 | 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 |
| 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 |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| CA2379166C (en) | 1999-08-09 | 2013-03-26 | 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 instrastrand base pairs |
| CA2410828C (en) | 2000-06-01 | 2012-01-24 | University Of North Carolina At Chapel Hill | Duplexed parvovirus vectors |
| MX346493B (es) | 2001-11-13 | 2017-03-21 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| 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 |
| WO2006119432A2 (en) | 2005-04-29 | 2006-11-09 | The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & 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 |
| EP2140004B1 (en) * | 2007-04-12 | 2022-01-12 | The Provost, Fellows, Foundation Scholars, & the other members of Board, of the College of the Holy & Undiv. Trinity of Queen Elizabeth near Dublin | 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 |
| CN102573856B (zh) * | 2009-09-10 | 2016-10-26 | 弗莱明·韦林 | 用于制备微小rna 的方法及其治疗性应用 |
| 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 |
| CN103189507A (zh) | 2010-10-27 | 2013-07-03 | 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 |
| DK2675902T3 (da) | 2011-02-17 | 2019-06-03 | Univ Pennsylvania | Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel |
| 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 |
| 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 |
| GB2537000C (en) * | 2012-05-25 | 2019-10-09 | Univ California | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| 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 |
| WO2014066498A1 (en) * | 2012-10-23 | 2014-05-01 | Vivek Mittal | Treatment of metastatic breast cancer |
| US9884125B2 (en) | 2013-10-04 | 2018-02-06 | Merck Sharp & Dohme Corp. | Glucose-responsive insulin conjugates |
| PT3119437T (pt) * | 2014-03-21 | 2019-12-12 | Genzyme Corp | Terapia genética para retinopatia pigmentar |
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2015
- 2015-03-20 PT PT157162322T patent/PT3119437T/pt unknown
- 2015-03-20 HU HUE15716232A patent/HUE046454T2/hu unknown
- 2015-03-20 AU AU2015230942A patent/AU2015230942B2/en active Active
- 2015-03-20 FI FIEP19186929.6T patent/FI3628334T3/fi active
- 2015-03-20 US US15/127,757 patent/US10383953B2/en active Active
- 2015-03-20 CR CR20160480A patent/CR20160480A/es unknown
- 2015-03-20 RS RS20191551A patent/RS59634B1/sr unknown
- 2015-03-20 NZ NZ724622A patent/NZ724622A/en unknown
- 2015-03-20 CA CA2943185A patent/CA2943185C/en active Active
- 2015-03-20 HR HRP20231077TT patent/HRP20231077T1/hr unknown
- 2015-03-20 LT LTEP19186929.6T patent/LT3628334T/lt unknown
- 2015-03-20 LT LTEP15716232.2T patent/LT3119437T/lt unknown
- 2015-03-20 PL PL19186929.6T patent/PL3628334T3/pl unknown
- 2015-03-20 WO PCT/US2015/021896 patent/WO2015143418A2/en not_active Ceased
- 2015-03-20 PE PE2016001643A patent/PE20161252A1/es unknown
- 2015-03-20 CN CN201580026274.0A patent/CN106456660B/zh active Active
- 2015-03-20 UA UAA201610589A patent/UA120050C2/uk unknown
- 2015-03-20 SI SI201531019T patent/SI3119437T1/sl unknown
- 2015-03-20 CN CN202210690697.4A patent/CN115252823A/zh active Pending
- 2015-03-20 CA CA3254798A patent/CA3254798A1/en active Pending
- 2015-03-20 MX MX2016012201A patent/MX376190B/es active IP Right Grant
- 2015-03-20 KR KR1020167028893A patent/KR20160127832A/ko not_active Ceased
- 2015-03-20 JP JP2016557218A patent/JP6669664B2/ja active Active
- 2015-03-20 ES ES15716232T patent/ES2760263T3/es active Active
- 2015-03-20 RS RS20230833A patent/RS64611B1/sr unknown
- 2015-03-20 KR KR1020227045369A patent/KR20230006039A/ko not_active Ceased
- 2015-03-20 DK DK15716232T patent/DK3119437T3/da active
- 2015-03-20 KR KR1020257039649A patent/KR20250172735A/ko active Pending
- 2015-03-20 EP EP23176679.1A patent/EP4345165A3/en active Pending
- 2015-03-20 ES ES19186929T patent/ES2957840T3/es active Active
- 2015-03-20 EP EP15716232.2A patent/EP3119437B1/en active Active
- 2015-03-20 EP EP19186929.6A patent/EP3628334B1/en active Active
- 2015-03-20 DK DK19186929.6T patent/DK3628334T5/da active
- 2015-03-20 PT PT191869296T patent/PT3628334T/pt unknown
- 2015-03-20 MA MA39390A patent/MA39390B2/fr unknown
- 2015-03-20 SG SG10201912968WA patent/SG10201912968WA/en unknown
- 2015-03-20 EA EA201691891A patent/EA201691891A1/ru unknown
- 2015-03-20 MY MYPI2016703077A patent/MY190726A/en unknown
- 2015-03-20 MY MYPI2020002274A patent/MY203654A/en unknown
- 2015-03-20 HR HRP20192141TT patent/HRP20192141T1/hr unknown
- 2015-03-20 SI SI201531966T patent/SI3628334T1/sl unknown
- 2015-03-20 HU HUE19186929A patent/HUE063460T2/hu unknown
- 2015-03-20 SG SG11201607005UA patent/SG11201607005UA/en unknown
- 2015-03-20 PL PL15716232T patent/PL3119437T3/pl 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