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
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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) | ES2805355T3 (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 |
|---|---|---|---|---|
| JP6591956B2 (ja) | 2013-03-15 | 2019-10-16 | 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 | Ｍｐｓ１を治療するための組成物および方法 |
| CA2946392A1 (en) * | 2014-04-25 | 2015-10-29 | James M. Wilson | 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 |
| AU2016370590B2 (en) * | 2015-12-14 | 2023-11-02 | The Trustees Of The University Of Pennsylvania | Composition for treatment of Crigler-Najjar syndrome |
| AU2017215211C1 (en) * | 2016-02-03 | 2023-11-16 | 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 |
| AU2017223465A1 (en) * | 2016-02-22 | 2018-08-09 | The University Of North Carolina At Chapel Hill | AAV-IDUA vector for treatment of MPS I-associated blindness |
| WO2017180936A1 (en) * | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsylvania | Compositions for treatment of wet age-related macular degeneration |
| AU2017250298B2 (en) | 2016-04-15 | 2024-10-03 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type II |
| US11883470B2 (en) * | 2016-07-25 | 2024-01-30 | The Trustees Of The University Of Pennsylvania | Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof |
| AU2017301600B2 (en) | 2016-07-26 | 2022-08-04 | Biomarin Pharmaceutical Inc. | Novel adeno-associated virus capsid proteins |
| IL315358A (en) | 2016-08-18 | 2024-11-01 | Univ California | CRISPR-CAS genome engineering using a modular AAV delivery system |
| BR112019013576A2 (pt) * | 2016-12-30 | 2020-02-04 | The Trustees Of The University Of Pennsylvania | terapia genica para o tratamento da fenilcetonuria |
| EP3676385A1 (en) | 2017-07-06 | 2020-07-08 | The Trustees of The University of Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
| JP7449223B2 (ja) | 2017-09-22 | 2024-03-13 | 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 | ムコ多糖症ｉｉ型を治療するための遺伝子療法 |
| 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 |
| EA202091352A1 (ru) | 2017-11-30 | 2020-08-27 | Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания | Генотерапия мукополисахаридоза iiib |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| AU2019215152A1 (en) | 2018-02-01 | 2020-08-20 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring PAH gene function and methods of use thereof |
| JP7244547B2 (ja) | 2018-02-19 | 2023-03-22 | ホモロジー・メディシンズ・インコーポレイテッド | Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法 |
| US12558434B2 (en) | 2018-02-20 | 2026-02-24 | The Trustees Of The University Of Pennsylvania | Compositions for treatment of wet age-related macular degeneration |
| CA3099958A1 (en) | 2018-04-16 | 2019-10-24 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating duchenne muscular dystrophy |
| CA3099704A1 (en) | 2018-05-09 | 2019-11-14 | Biomarin Pharmaceutical Inc. | Methods of treating phenylketonuria |
| 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 |
| US11510999B2 (en) | 2018-07-17 | 2022-11-29 | Helixmith Co., Ltd | Treatment of neuropathy with DNA constructs expressing IGF-1 isoforms |
| CN113924115A (zh) | 2019-01-31 | 2022-01-11 | 俄勒冈健康与科学大学 | 用于aav衣壳的使用转录依赖性定向进化的方法 |
| 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 |
| CA3177407A1 (en) | 2020-05-12 | 2021-11-18 | James M. Wilson | 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 |
| JP2023531451A (ja) | 2020-06-17 | 2023-07-24 | 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 | 遺伝子療法患者の治療のための組成物及び方法 |
| 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 |
| PL4148078T3 (pl) | 2021-09-08 | 2026-03-30 | Covestro Deutschland Ag | Poliole poliestrowe na bazie kwasu bursztynowego |
| 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 |
| WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| US20240408157A1 (en) | 2021-12-02 | 2024-12-12 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
| 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 |
| WO2023196893A1 (en) | 2022-04-06 | 2023-10-12 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating her2 positive metastatic breast cancer and other cancers |
| EP4724085A1 (en) | 2023-06-12 | 2026-04-15 | 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 |
| JP2001500015A (ja) | 1996-09-06 | 2001-01-09 | 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 | Ｔ７ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法 |
| 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 |
| 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 |
| 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 |
| SG10202108118RA (en) | 2001-11-13 | 2021-08-30 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| ES2975413T3 (es) | 2001-12-17 | 2024-07-05 | Univ Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas |
| 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 |
| HUE054805T2 (hu) | 2003-09-30 | 2021-09-28 | Univ Pennsylvania | Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik |
| EP3085389A1 (en) | 2005-04-07 | 2016-10-26 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| WO2006132118A1 (ja) | 2005-06-09 | 2006-12-14 | Matsushita Electric Industrial Co., Ltd. | 振幅誤差補償装置及び直交度誤差補償装置 |
| WO2007120542A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford 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 |
| BR112012010755A2 (pt) | 2009-11-05 | 2015-09-22 | Proyecto Biomedicina Cima Sl | constructo de gene que permite a expressão hepato-específica induzível de um polinucleotídeo de interesse em resposta a um agente indutor, vetor, genoma viral recombinante, vírion, método in vitro para a expressão de um polinucleotídeo de interesse em uma célula de origem hepática, composição farmacêutica e operador-promotor bi-direcional induzível adequado para a expressão hepato-específica induzível de dois polinucleotídeos de interesse por um agente indutor |
| EP2333074A1 (en) * | 2009-12-14 | 2011-06-15 | Robert Steinfeld | Substances and methods for the treatment of lysosmal storage diseases |
| KR20130040844A (ko) * | 2010-03-29 | 2013-04-24 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 약학적으로 유발된 전이유전자 제거 시스템 |
| EP3567106A1 (en) | 2010-04-23 | 2019-11-13 | University of Massachusetts | Cns targeting aav vectors 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 |
| JP6049623B2 (ja) * | 2010-10-22 | 2016-12-21 | カッパーアールエヌエー，インコーポレイテッド | α‐Ｌ‐イズロニダーゼ（ＩＤＵＡ）への天然アンチセンス転写物の阻害によるＩＤＵＡ関連疾患の治療 |
| SG10202007803QA (en) | 2011-02-17 | 2020-09-29 | Univ Pennsylvania | Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer |
| CN107012171B (zh) | 2011-04-22 | 2021-07-13 | 加利福尼亚大学董事会 | 具有变异衣壳的腺相关病毒病毒体及其使用方法 |
| CA2875618C (en) | 2012-07-11 | 2021-04-27 | Sangamo Biosciences, Inc. | Methods and compositions for the treatment of lysosomal storage diseases |
| 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 |
| JP6591956B2 (ja) | 2013-03-15 | 2019-10-16 | 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 | Ｍｐｓ１を治療するための組成物および方法 |
| KR20160010526A (ko) | 2013-05-15 | 2016-01-27 | 리젠츠 오브 더 유니버시티 오브 미네소타 | 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달 |
| AU2015320694B2 (en) | 2014-09-24 | 2021-11-11 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| WO2016055437A1 (en) | 2014-10-06 | 2016-04-14 | Arthrogen B.V. | Aav-based gene therapy |
| CA2986252A1 (en) | 2015-05-15 | 2016-11-24 | Regents Of The University Of Minnesota | Adeno-associated virus vector for delivery of iduronate-2-sulfatase to the central nervous system |
| US10604747B2 (en) | 2015-08-06 | 2020-03-31 | Academia Sinica | Engineered enzyme for enzyme replacement therapy |
| SG11201803218PA (en) | 2015-10-23 | 2018-05-30 | Univ Iowa Res Found | Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection |
| US11015173B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV1 |
| US11098286B2 (en) | 2015-12-11 | 2021-08-24 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV9 |
| ES2918998T3 (es) | 2015-12-11 | 2022-07-21 | Univ Pennsylvania | Método de purificación escalable para AAVrh10 |
| WO2017100676A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| AU2017207818B2 (en) | 2016-01-15 | 2024-03-28 | Regents Of The University Of Minnesota | Methods and compositions for the treatment of neurologic disease |
| AU2017215211C1 (en) | 2016-02-03 | 2023-11-16 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| EP3676385A1 (en) | 2017-07-06 | 2020-07-08 | The Trustees of The University of Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
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2014
- 2014-03-13 JP JP2016501864A patent/JP6591956B2/ja active Active
- 2014-03-13 PT PT147701866T patent/PT2984166T/pt unknown
- 2014-03-13 SI SI201431605T patent/SI2984166T1/sl unknown
- 2014-03-13 HR HRP20201063TT patent/HRP20201063T1/hr unknown
- 2014-03-13 WO PCT/US2014/025509 patent/WO2014151341A1/en not_active Ceased
- 2014-03-13 HU HUE14770186A patent/HUE051373T2/hu unknown
- 2014-03-13 CA CA2901328A patent/CA2901328C/en active Active
- 2014-03-13 US US14/769,596 patent/US10137176B2/en active Active
- 2014-03-13 CN CN201480010987.3A patent/CN105026554B/zh active Active
- 2014-03-13 SG SG10201707319UA patent/SG10201707319UA/en unknown
- 2014-03-13 DK DK14770186.6T patent/DK2984166T3/da active
- 2014-03-13 ES ES14770186T patent/ES2805355T3/es active Active
- 2014-03-13 SG SG11201507507PA patent/SG11201507507PA/en unknown
- 2014-03-13 BR BR112015021036A patent/BR112015021036A2/pt not_active Application Discontinuation
- 2014-03-13 LT LTEP14770186.6T patent/LT2984166T/lt unknown
- 2014-03-13 KR KR1020217043023A patent/KR102581652B1/ko active Active
- 2014-03-13 EP EP14770186.6A patent/EP2984166B1/en active Active
- 2014-03-13 ES ES20170476T patent/ES2991471T3/es active Active
- 2014-03-13 AU AU2014235096A patent/AU2014235096B2/en active Active
- 2014-03-13 EP EP20170476.4A patent/EP3747998B1/en active Active
- 2014-03-13 PL PL14770186T patent/PL2984166T3/pl unknown
- 2014-03-13 MX MX2015012739A patent/MX386059B/es unknown
- 2014-03-13 RU RU2015144234A patent/RU2708318C2/ru active
- 2014-03-13 KR KR1020157022504A patent/KR102346455B1/ko active Active
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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