JP2016512683A5 - - Google Patents
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- JP2016512683A5 JP2016512683A5 JP2016501864A JP2016501864A JP2016512683A5 JP 2016512683 A5 JP2016512683 A5 JP 2016512683A5 JP 2016501864 A JP2016501864 A JP 2016501864A JP 2016501864 A JP2016501864 A JP 2016501864A JP 2016512683 A5 JP2016512683 A5 JP 2016512683A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- raav
- enhancer
- change
- hidua
- 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 |
|---|---|---|---|
| US201361788724P | 2013-03-15 | 2013-03-15 | |
| US61/788,724 | 2013-03-15 | ||
| PCT/US2014/025509 WO2014151341A1 (en) | 2013-03-15 | 2014-03-13 | Compositions and methods for treating mpsi |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019079881A Division JP2019134717A (ja) | 2013-03-15 | 2019-04-19 | Ｍｐｓ１を治療するための組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016512683A JP2016512683A (ja) | 2016-05-09 |
| JP2016512683A5 true JP2016512683A5 (enExample) | 2017-04-13 |
| JP6591956B2 JP6591956B2 (ja) | 2019-10-16 |
Family
ID=51580934
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016501864A Active JP6591956B2 (ja) | 2013-03-15 | 2014-03-13 | Ｍｐｓ１を治療するための組成物および方法 |
| JP2019079881A Pending JP2019134717A (ja) | 2013-03-15 | 2019-04-19 | Ｍｐｓ１を治療するための組成物および方法 |
| JP2020148103A Active JP7168621B2 (ja) | 2013-03-15 | 2020-09-03 | Ｍｐｓ１を治療するための組成物および方法 |
| JP2022172314A Active JP7553527B2 (ja) | 2013-03-15 | 2022-10-27 | Ｍｐｓ１を治療するための組成物および方法 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019079881A Pending JP2019134717A (ja) | 2013-03-15 | 2019-04-19 | Ｍｐｓ１を治療するための組成物および方法 |
| JP2020148103A Active JP7168621B2 (ja) | 2013-03-15 | 2020-09-03 | Ｍｐｓ１を治療するための組成物および方法 |
| JP2022172314A Active JP7553527B2 (ja) | 2013-03-15 | 2022-10-27 | Ｍｐｓ１を治療するための組成物および方法 |
Country Status (21)
| Country | Link |
|---|---|
| US (3) | US10137176B2 (enExample) |
| EP (2) | EP2984166B1 (enExample) |
| JP (4) | JP6591956B2 (enExample) |
| KR (2) | KR102581652B1 (enExample) |
| CN (1) | CN105026554B (enExample) |
| AU (2) | AU2014235096B2 (enExample) |
| BR (1) | BR112015021036A2 (enExample) |
| CA (1) | CA2901328C (enExample) |
| CY (1) | CY1123169T1 (enExample) |
| DK (1) | DK2984166T3 (enExample) |
| ES (2) | ES2991471T3 (enExample) |
| HR (1) | HRP20201063T1 (enExample) |
| HU (1) | HUE051373T2 (enExample) |
| LT (1) | LT2984166T (enExample) |
| MX (2) | MX386059B (enExample) |
| PL (1) | PL2984166T3 (enExample) |
| PT (1) | PT2984166T (enExample) |
| RU (1) | RU2708318C2 (enExample) |
| SG (2) | SG10201707319UA (enExample) |
| SI (1) | SI2984166T1 (enExample) |
| WO (1) | WO2014151341A1 (enExample) |
Families Citing this family (42)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG10201707319UA (en) * | 2013-03-15 | 2017-10-30 | Univ Pennsylvania | Compositions and methods for treating mpsi |
| US11555059B2 (en) * | 2014-04-25 | 2023-01-17 | The Trustees Of The University Of Pennsylvania | LDLR variants and their use in compositions for reducing cholesterol levels |
| CA3008142A1 (en) * | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| JP7061067B2 (ja) | 2015-12-14 | 2022-04-27 | 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 | クリグラー・ナジャー症候群の処置のための組成物 |
| WO2017136500A1 (en) * | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| WO2017136533A1 (en) * | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Methods for treating diagnosing, and monitoring treatment of mucopolysaccharidoses |
| MX2018009426A (es) | 2016-02-22 | 2018-12-19 | Univ North Carolina Chapel Hill | Vector aav-idua para el tratamiento de ceguera asociada con mucopolisacaridosis i (mps i). |
| WO2017181113A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsyvania | Gene therapy for treating mucopolysaccharidosis type ii |
| IL322403A (en) | 2016-04-15 | 2025-09-01 | Univ Pennsylvania | Preparations for the treatment of wet age-related macular degeneration |
| WO2018022511A1 (en) | 2016-07-25 | 2018-02-01 | The Trustees Of The University Of Pennsylvania | Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof |
| IL263801B2 (en) | 2016-07-26 | 2024-01-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| AU2017313917B2 (en) | 2016-08-18 | 2023-12-21 | The Regents Of The University Of California | CRISPR-Cas genome engineering via a modular AAV delivery system |
| EP3562494A4 (en) | 2016-12-30 | 2020-08-19 | The Trustees Of The University Of Pennsylvania | GENE THERAPY FOR THE TREATMENT OF PHENYLKETONURIS |
| AU2018298133A1 (en) | 2017-07-06 | 2020-01-23 | The Trustees Of The University Of Pennsylvania | AAV9-mediated gene therapy for treating mucopolysaccharidosis type I |
| WO2019060662A1 (en) | 2017-09-22 | 2019-03-28 | The Trustees Of The University Of Pennsylvania | GENE THERAPY FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSE TYPE II |
| CN107699590A (zh) * | 2017-10-13 | 2018-02-16 | 成都中医药大学 | 一种制备重组人α‑L‑艾杜糖醛酸酶的方法 |
| CN111989396A (zh) | 2017-11-30 | 2020-11-24 | 宾夕法尼亚州大学信托人 | 用于iiib型粘多糖贮积病的基因疗法 |
| US20190256867A1 (en) | 2018-02-01 | 2019-08-22 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring pah gene function and methods of use thereof |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| CN112041437A (zh) | 2018-02-19 | 2020-12-04 | 同源药物公司 | 用于恢复f8基因功能的腺相关病毒组合物和其使用方法 |
| US12558434B2 (en) | 2018-02-20 | 2026-02-24 | The Trustees Of The University Of Pennsylvania | Compositions for treatment of wet age-related macular degeneration |
| US12460226B2 (en) | 2018-04-16 | 2025-11-04 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating duchenne muscular dystrophy |
| JP2021522811A (ja) | 2018-05-09 | 2021-09-02 | ビオマリン プハルマセウトイカル インコーポレイテッド | フェニルケトン尿症の治療方法 |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| AU2019304569B2 (en) | 2018-07-17 | 2023-07-06 | Helixmith Co., Ltd | Treatment of neuropathy with DNA constructs expressing IGF-1 isoforms |
| KR20210130158A (ko) | 2019-01-31 | 2021-10-29 | 오레곤 헬스 앤드 사이언스 유니버시티 | Aav 캡시드의 전사 의존적 유도 진화를 사용하는 방법 |
| TW202140791A (zh) | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
| EP4162059A1 (en) | 2020-05-12 | 2023-04-12 | The Trustees of The University of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
| IL299167A (en) | 2020-06-17 | 2023-02-01 | Univ Pennsylvania | Compositions and methods for treating patients with gene therapy |
| AU2021356684A1 (en) | 2020-10-09 | 2023-05-11 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
| KR102590276B1 (ko) * | 2021-01-04 | 2023-10-16 | 부산대학교 산학협력단 | 조직 특이적 발현을 위한 융합 프로모터 및 이의 용도 |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| EP4148078B1 (de) | 2021-09-08 | 2025-11-05 | Covestro Deutschland AG | Auf bernsteinsäure basierende polyesterpolyole |
| CN114181318B (zh) * | 2021-11-08 | 2023-08-01 | 四川大学 | 一种组织特异性表达穿透血脑屏障的idua融合蛋白的重组腺相关病毒及应用 |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| WO2023102517A1 (en) | 2021-12-02 | 2023-06-08 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
| CN119317645A (zh) | 2022-04-06 | 2025-01-14 | 宾夕法尼亚州大学信托人 | 用于治疗her2阳性转移性乳腺癌及其他癌症的组合物和方法 |
| WO2023196892A1 (en) | 2022-04-06 | 2023-10-12 | The Trustees Of The University Of Pennsylvania | Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors |
| WO2024258961A1 (en) | 2023-06-12 | 2024-12-19 | The Trustees Of The University Of Pennsylvania | Aav gene therapy for mucopolysaccharidosis iiib |
| WO2025035143A1 (en) | 2023-08-10 | 2025-02-13 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of spinal muscular atrophy |
| WO2025129157A1 (en) | 2023-12-15 | 2025-06-19 | The Trustees Of The University Of Pennsylvania | Gene therapy for treatment of canavan disease |
Family Cites Families (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| AU4072499A (en) | 1998-05-13 | 1999-11-29 | Harbor-Ucla | Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof |
| ATE403715T1 (de) | 1999-08-09 | 2008-08-15 | Targeted Genetics Corp | Erhöhung der expression einer einzelsträngigen, heterologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen |
| 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 |
| CN103555677B (zh) | 2001-11-13 | 2018-01-30 | 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 |
| WO2003052051A2 (en) | 2001-12-17 | 2003-06-26 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences |
| WO2004055157A2 (en) * | 2002-08-13 | 2004-07-01 | Whitley Chester B | Methods of using vectors to treat metabolic disorders |
| US7155804B2 (en) | 2003-09-17 | 2007-01-02 | Moog Inc. | Method of forming an electric motor |
| DK3211085T3 (da) | 2003-09-30 | 2021-06-21 | Univ Pennsylvania | Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf |
| US8999678B2 (en) | 2005-04-07 | 2015-04-07 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an AAV vector |
| US7456683B2 (en) | 2005-06-09 | 2008-11-25 | Panasonic Corporation | Amplitude error compensating device and quadrature skew error compensating device |
| 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 |
| US20120225933A1 (en) * | 2009-11-05 | 2012-09-06 | Gonzalez Aseguinolaza Gloria | Regulated expression systems |
| EP2333074A1 (en) * | 2009-12-14 | 2011-06-15 | Robert Steinfeld | Substances and methods for the treatment of lysosmal storage diseases |
| SG10201908848RA (en) * | 2010-03-29 | 2019-10-30 | Univ Pennsylvania | Pharmacologically induced transgene ablation system |
| DK2826860T3 (en) | 2010-04-23 | 2018-12-03 | Univ Massachusetts | CNS targeting AAV vectors and methods for their use |
| 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 |
| US9222088B2 (en) * | 2010-10-22 | 2015-12-29 | Curna, Inc. | Treatment of alpha-L-iduronidase (IDUA) related diseases by inhibition of natural antisense transcript to IDUA |
| ES2724800T3 (es) | 2011-02-17 | 2019-09-16 | Univ Pennsylvania | Composiciones y métodos para alterar la especificidad de tejido y mejorar la transferencia génica mediada por AAV9 |
| DK4005603T3 (da) | 2011-04-22 | 2024-11-25 | Univ California | Adeno-associerede virus-virioner med variant kapsid og fremgangsmåder til anvendelse heraf |
| HUE051612T2 (hu) | 2012-07-11 | 2021-03-01 | Sangamo Therapeutics Inc | Eljárások és készítmények lizoszomális tárolási betegségek kezelésére |
| JP6437443B2 (ja) | 2012-11-21 | 2018-12-12 | アールキューエックス ファーマシューティカルズ，インク． | 大環状広域抗生物質 |
| 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 |
| SG10201707319UA (en) | 2013-03-15 | 2017-10-30 | Univ Pennsylvania | Compositions and methods for treating mpsi |
| CA2912678C (en) | 2013-05-15 | 2023-10-10 | Regents Of The University Of Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
| KR20230067694A (ko) | 2014-09-24 | 2023-05-16 | 시티 오브 호프 | 고효율 게놈 편집을 위한 아데노-관련 바이러스 벡터 변이체 및 이의 방법 |
| CA2963168A1 (en) | 2014-10-06 | 2016-04-14 | Arthrogen B.V. | Aav-based gene therapy |
| HRP20220050T1 (hr) | 2015-05-15 | 2022-10-14 | Regents Of The University Of Minnesota | Adenoasocirani virus, namijenjen terapijskom unosu u središnji živčani sustav |
| CN107922932A (zh) | 2015-08-06 | 2018-04-17 | 中央研究院 | 用于酶置换疗法的改造酶 |
| CN108601771B (zh) | 2015-10-23 | 2023-02-21 | 衣阿华大学研究基金会 | 使用基因疗法以推迟疾病发生和发展同时提供认知保护来治疗神经变性疾病的方法 |
| US11098286B2 (en) | 2015-12-11 | 2021-08-24 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV9 |
| EP3387118B1 (en) | 2015-12-11 | 2022-04-06 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aavrh10 |
| WO2017100676A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| US11015173B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV1 |
| ES2895430T3 (es) | 2016-01-15 | 2022-02-21 | Univ Minnesota | Métodos y composiciones para el tratamiento de enfermedad neurológica |
| WO2017136500A1 (en) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| AU2018298133A1 (en) | 2017-07-06 | 2020-01-23 | The Trustees Of The University Of Pennsylvania | AAV9-mediated gene therapy for treating mucopolysaccharidosis type I |
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2014
- 2014-03-13 SG SG10201707319UA patent/SG10201707319UA/en unknown
- 2014-03-13 SI SI201431605T patent/SI2984166T1/sl unknown
- 2014-03-13 RU RU2015144234A patent/RU2708318C2/ru active
- 2014-03-13 WO PCT/US2014/025509 patent/WO2014151341A1/en not_active Ceased
- 2014-03-13 EP EP14770186.6A patent/EP2984166B1/en active Active
- 2014-03-13 JP JP2016501864A patent/JP6591956B2/ja active Active
- 2014-03-13 ES ES20170476T patent/ES2991471T3/es active Active
- 2014-03-13 KR KR1020217043023A patent/KR102581652B1/ko active Active
- 2014-03-13 SG SG11201507507PA patent/SG11201507507PA/en unknown
- 2014-03-13 US US14/769,596 patent/US10137176B2/en active Active
- 2014-03-13 PL PL14770186T patent/PL2984166T3/pl unknown
- 2014-03-13 KR KR1020157022504A patent/KR102346455B1/ko active Active
- 2014-03-13 AU AU2014235096A patent/AU2014235096B2/en active Active
- 2014-03-13 DK DK14770186.6T patent/DK2984166T3/da active
- 2014-03-13 BR BR112015021036A patent/BR112015021036A2/pt not_active Application Discontinuation
- 2014-03-13 CN CN201480010987.3A patent/CN105026554B/zh active Active
- 2014-03-13 HR HRP20201063TT patent/HRP20201063T1/hr unknown
- 2014-03-13 LT LTEP14770186.6T patent/LT2984166T/lt unknown
- 2014-03-13 EP EP20170476.4A patent/EP3747998B1/en active Active
- 2014-03-13 ES ES14770186T patent/ES2805355T3/es active Active
- 2014-03-13 MX MX2015012739A patent/MX386059B/es unknown
- 2014-03-13 CA CA2901328A patent/CA2901328C/en active Active
- 2014-03-13 HU HUE14770186A patent/HUE051373T2/hu unknown
- 2014-03-13 PT PT147701866T patent/PT2984166T/pt unknown
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2015
- 2015-09-14 MX MX2021010918A patent/MX2021010918A/es unknown
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2018
- 2018-10-22 US US16/166,346 patent/US10792343B2/en active Active
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2019
- 2019-04-19 JP JP2019079881A patent/JP2019134717A/ja active Pending
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2020
- 2020-06-10 AU AU2020203826A patent/AU2020203826B2/en active Active
- 2020-07-07 CY CY20201100628T patent/CY1123169T1/el unknown
- 2020-09-03 JP JP2020148103A patent/JP7168621B2/ja active Active
- 2020-09-10 US US17/017,150 patent/US20200397872A1/en not_active Abandoned
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2022
- 2022-10-27 JP JP2022172314A patent/JP7553527B2/ja active Active