JP2020526514A5 - - Google Patents
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- JP2020526514A5 JP2020526514A5 JP2020500131A JP2020500131A JP2020526514A5 JP 2020526514 A5 JP2020526514 A5 JP 2020526514A5 JP 2020500131 A JP2020500131 A JP 2020500131A JP 2020500131 A JP2020500131 A JP 2020500131A JP 2020526514 A5 JP2020526514 A5 JP 2020526514A5
- Authority
- JP
- Japan
- Prior art keywords
- immunosuppressant
- composition
- use according
- months
- sequence
- 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.)
- Pending
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023108011A JP7670759B2 (ja) | 2017-07-06 | 2023-06-30 | ムコ多糖症ｉ型を治療するための遺伝子治療 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762529385P | 2017-07-06 | 2017-07-06 | |
| US62/529,385 | 2017-07-06 | ||
| US201762530614P | 2017-07-10 | 2017-07-10 | |
| US62/530,614 | 2017-07-10 | ||
| US201862616106P | 2018-01-11 | 2018-01-11 | |
| US62/616,106 | 2018-01-11 | ||
| PCT/US2018/040957 WO2019010335A1 (en) | 2017-07-06 | 2018-07-05 | AAV9 MEDIATED GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023108011A Division JP7670759B2 (ja) | 2017-07-06 | 2023-06-30 | ムコ多糖症ｉ型を治療するための遺伝子治療 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020526514A JP2020526514A (ja) | 2020-08-31 |
| JP2020526514A5 true JP2020526514A5 (enExample) | 2021-08-12 |
Family
ID=63036365
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020500131A Pending JP2020526514A (ja) | 2017-07-06 | 2018-07-05 | ムコ多糖症ｉ型を治療するための遺伝子治療 |
| JP2023108011A Active JP7670759B2 (ja) | 2017-07-06 | 2023-06-30 | ムコ多糖症ｉ型を治療するための遺伝子治療 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023108011A Active JP7670759B2 (ja) | 2017-07-06 | 2023-06-30 | ムコ多糖症ｉ型を治療するための遺伝子治療 |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US11890329B2 (enExample) |
| EP (1) | EP3676385A1 (enExample) |
| JP (2) | JP2020526514A (enExample) |
| KR (2) | KR20240063170A (enExample) |
| AU (1) | AU2018298133A1 (enExample) |
| BR (1) | BR112020000063A2 (enExample) |
| CA (1) | CA3068328A1 (enExample) |
| IL (1) | IL271780A (enExample) |
| MA (1) | MA50016A (enExample) |
| SG (1) | SG11201912631PA (enExample) |
| WO (1) | WO2019010335A1 (enExample) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112015021036A2 (pt) | 2013-03-15 | 2017-10-10 | Univ Pennsylvania | composições e métodos para tratar mps1 |
| JP2016523835A (ja) | 2013-05-15 | 2016-08-12 | リージェンツ オブ ザ ユニバーシティ オブ ミネソタ | 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入 |
| MA43968A (fr) | 2016-02-03 | 2018-12-12 | Univ Pennsylvania | Thérapie génique pour traiter la mucopolysaccharidose de type i |
| CA3068328A1 (en) | 2017-07-06 | 2019-01-10 | The Trustees Of The University Of Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
| EP3684938A1 (en) | 2017-09-22 | 2020-07-29 | The Trustees of the University of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
| EP3886919A1 (en) * | 2018-11-30 | 2021-10-06 | Novartis AG | Aav viral vectors and uses thereof |
| JP7697943B2 (ja) * | 2019-10-23 | 2025-06-24 | 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 | 導入遺伝子発現のｄｒｇ特異的低減のための組成物 |
| KR20220131522A (ko) * | 2020-01-22 | 2022-09-28 | 리젠엑스바이오 인크. | 완전-인간 글리코실화된 인간 알파-l-이두로니다아제 (idua)를 사용한 점액다당류증 i의 치료 |
| CN116157527A (zh) * | 2020-08-10 | 2023-05-23 | 普利维尔治疗公司 | 用于溶酶体病症的基因疗法 |
| AU2021325891A1 (en) * | 2020-08-10 | 2023-04-06 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disorders |
| MX2023008826A (es) | 2021-02-01 | 2023-09-15 | Regenxbio Inc | Terapia génica para lipofuscinosis neuronal ceroidea. |
| US20240316217A1 (en) * | 2021-02-05 | 2024-09-26 | Regents Of The University Of Minnesota | Methods for preventing cardiac or skeletal defects in diseases including mucopolysaccharidoses |
| US20240226331A9 (en) * | 2021-02-22 | 2024-07-11 | Decibel Therapeutics, Inc. | Methods and compositions for reducing nucleic acid vector-induced toxicity in the inner ear |
| WO2024205528A1 (en) * | 2023-03-31 | 2024-10-03 | Erci̇yes Üni̇versi̇tesi̇ Strateji̇ Geli̇şti̇rme Dai̇re Başkanliği | A multi-head for use in electrohydrodynamic |
Family Cites Families (53)
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| EP0578790A4 (en) | 1991-11-14 | 1994-11-02 | Womens & Childrens Hospital | SYNTHETIC ALPHA-L-IDURONIDASE AND THESE ENCODING GENETIC SEQUENCES. |
| US6149909A (en) | 1995-06-23 | 2000-11-21 | Women's And Children's Hospital | Synthetic α-L-iduronidase and genetic sequences encoding same |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| CA2264482A1 (en) | 1996-09-06 | 1998-03-12 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| WO1999058691A2 (en) | 1998-05-13 | 1999-11-18 | Harbor-Ucla | Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof |
| US6596535B1 (en) | 1999-08-09 | 2003-07-22 | Targeted Genetics Corporation | Metabolically activated recombinant viral vectors and methods for the preparation and use |
| US6426208B1 (en) | 1999-11-12 | 2002-07-30 | Harbor-Ucla Research And Education Institute | Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof |
| RU2196988C2 (ru) | 2000-04-19 | 2003-01-20 | Государственный новосибирский областной клинический диагностический центр | Способ диагностики мукополисахаридозов |
| AU2001267337A1 (en) | 2000-06-30 | 2002-01-14 | Maxygen Aps | Peptide extended glycosylated polypeptides |
| 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 |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| AU2003302724A1 (en) | 2002-08-13 | 2004-07-09 | Mcivor, Scott, R. | Methods of using vectors to treat metabolic disorders |
| US7155804B2 (en) | 2003-09-17 | 2007-01-02 | Moog Inc. | Method of forming an electric motor |
| EP1668143B1 (en) | 2003-09-30 | 2013-03-20 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| DE23193606T1 (de) | 2005-04-07 | 2024-02-15 | The Trustees Of The University Of Pennsylvania | Verfahren zur erhöhung der funktion eines aav-vektors |
| JP4495210B2 (ja) | 2005-06-09 | 2010-06-30 | パナソニック株式会社 | 振幅誤差補償装置及び直交度誤差補償装置 |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| US20090062144A1 (en) | 2007-04-03 | 2009-03-05 | Nancy Lan Guo | Gene signature for prognosis and diagnosis of lung cancer |
| US9415121B2 (en) | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
| JP2014503173A (ja) | 2009-11-05 | 2014-02-13 | プロイェクト、デ、ビオメディシナ、シーマ、ソシエダッド、リミターダ | 制御された発現系 |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| DK2561073T3 (en) | 2010-04-23 | 2016-12-12 | Univ Massachusetts | Aav vectors targeted to central nervous system and methods of use thereof |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| EP2394667A1 (en) | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
| 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 |
| DK3693025T3 (da) | 2011-04-22 | 2022-01-10 | Univ California | Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf |
| HUE051612T2 (hu) | 2012-07-11 | 2021-03-01 | Sangamo Therapeutics Inc | Eljárások és készítmények lizoszomális tárolási betegségek kezelésére |
| WO2014125647A1 (ja) | 2013-02-18 | 2014-08-21 | 富士通オプティカルコンポーネンツ株式会社 | 光受信装置 |
| BR112015021036A2 (pt) | 2013-03-15 | 2017-10-10 | Univ Pennsylvania | composições e métodos para tratar mps1 |
| JP2016523835A (ja) | 2013-05-15 | 2016-08-12 | リージェンツ オブ ザ ユニバーシティ オブ ミネソタ | 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入 |
| AU2014310360B2 (en) | 2013-06-11 | 2019-10-24 | PPL (BVI) Limited | Structure, manufacturing and uses of human-derived cell-permeable peptides conjugated with specific biologically active cargo peptides |
| EP3564379A1 (en) | 2013-09-13 | 2019-11-06 | California Institute of Technology | Selective recovery |
| WO2015164723A1 (en) * | 2014-04-25 | 2015-10-29 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treating metastatic breast cancer and other cancers in the brain |
| WO2016049230A1 (en) | 2014-09-24 | 2016-03-31 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| CN107002096A (zh) * | 2014-10-06 | 2017-08-01 | 阿罗根有限公司 | 基于aav的基因疗法 |
| KR20220082100A (ko) * | 2015-05-15 | 2022-06-16 | 리젠츠 오브 더 유니버시티 오브 미네소타 | 중추 신경계로의 아데노-연관 치료적 전달 |
| CN107922932A (zh) | 2015-08-06 | 2018-04-17 | 中央研究院 | 用于酶置换疗法的改造酶 |
| WO2017024198A1 (en) | 2015-08-06 | 2017-02-09 | The Trustees Of The University Of Pennsylvania | Glp-1 and use thereof in compositions for treating metabolic diseases |
| WO2017040254A1 (en) | 2015-08-28 | 2017-03-09 | Laufer Wind Group Llc | Mitigation of small unmanned aircraft systems threats |
| HK1257024A1 (zh) * | 2015-08-31 | 2019-10-11 | 宾夕法尼亚大学 | 用於治疗伴侣动物的aav-epo |
| AU2016341428B2 (en) | 2015-10-23 | 2021-12-02 | University Of Iowa Research Foundation | Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection |
| EP3992283A1 (en) | 2015-12-11 | 2022-05-04 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aavrh10 |
| EP3387138B2 (en) | 2015-12-11 | 2026-05-27 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| US11015174B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV8 |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| EP3402533B1 (en) | 2016-01-15 | 2021-08-04 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of neurologic disease |
| MA43968A (fr) | 2016-02-03 | 2018-12-12 | Univ Pennsylvania | Thérapie génique pour traiter la mucopolysaccharidose de type i |
| IL309808B2 (en) | 2016-11-15 | 2025-04-01 | Univ Minnesota | Method for improving neurological function in MPSI and MPSII and other neurological disorders |
| JP2020508289A (ja) | 2017-01-31 | 2020-03-19 | レジェンクスバイオ インコーポレーテッド | 完全ヒトグリコシル化ヒトα−Ｌ−イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療 |
| CA3068328A1 (en) * | 2017-07-06 | 2019-01-10 | The Trustees Of The University Of Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
| IL280198B2 (en) | 2018-07-18 | 2026-01-01 | Regenxbio Inc | Treatment of mucopolysaccharidosis I with fully glycosylated human iduronidase-alpha-L (idus) |
| KR20220131522A (ko) | 2020-01-22 | 2022-09-28 | 리젠엑스바이오 인크. | 완전-인간 글리코실화된 인간 알파-l-이두로니다아제 (idua)를 사용한 점액다당류증 i의 치료 |
| US20240316217A1 (en) | 2021-02-05 | 2024-09-26 | Regents Of The University Of Minnesota | Methods for preventing cardiac or skeletal defects in diseases including mucopolysaccharidoses |
-
2018
- 2018-07-05 CA CA3068328A patent/CA3068328A1/en active Pending
- 2018-07-05 MA MA050016A patent/MA50016A/fr unknown
- 2018-07-05 BR BR112020000063-9A patent/BR112020000063A2/pt unknown
- 2018-07-05 AU AU2018298133A patent/AU2018298133A1/en not_active Abandoned
- 2018-07-05 WO PCT/US2018/040957 patent/WO2019010335A1/en not_active Ceased
- 2018-07-05 SG SG11201912631PA patent/SG11201912631PA/en unknown
- 2018-07-05 US US16/624,084 patent/US11890329B2/en active Active
- 2018-07-05 EP EP18746390.6A patent/EP3676385A1/en active Pending
- 2018-07-05 KR KR1020247013510A patent/KR20240063170A/ko active Pending
- 2018-07-05 JP JP2020500131A patent/JP2020526514A/ja active Pending
- 2018-07-05 KR KR1020207003304A patent/KR20200053468A/ko not_active Ceased
-
2019
- 2019-12-31 IL IL271780A patent/IL271780A/en unknown
-
2023
- 2023-06-30 JP JP2023108011A patent/JP7670759B2/ja active Active
- 2023-12-13 US US18/538,866 patent/US20240307511A1/en not_active Abandoned
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