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) | EP3747998B1 (enExample) |
| JP (4) | JP6591956B2 (enExample) |
| KR (2) | KR102346455B1 (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) | SG11201507507PA (enExample) |
| SI (1) | SI2984166T1 (enExample) |
| WO (1) | WO2014151341A1 (enExample) |
Families Citing this family (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112015021036A2 (pt) | 2013-03-15 | 2017-10-10 | Univ Pennsylvania | composições e métodos para tratar mps1 |
| 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 |
| EP4085934A1 (en) * | 2015-12-11 | 2022-11-09 | 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 |
| SG10201912935WA (en) | 2015-12-15 | 2020-02-27 | Genzyme Corp | Adeno-associated viral vectors for treating mucolipidosis type ii |
| MA43968A (fr) | 2016-02-03 | 2018-12-12 | Univ Pennsylvania | Thérapie génique pour traiter la mucopolysaccharidose de 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 |
| CN108697774A (zh) * | 2016-02-22 | 2018-10-23 | 北卡罗来纳大学教堂山分校 | 治疗mps i-相关失明的aav-idua载体 |
| US11253612B2 (en) | 2016-04-15 | 2022-02-22 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type II |
| IL262207B2 (en) | 2016-04-15 | 2026-01-01 | 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 |
| JP6994018B2 (ja) | 2016-07-26 | 2022-01-14 | バイオマリン ファーマシューティカル インコーポレイテッド | 新規アデノ随伴ウイルスキャプシドタンパク質 |
| CA3034089A1 (en) | 2016-08-18 | 2018-02-22 | 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 |
| 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 |
| 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 |
| WO2019152841A1 (en) | 2018-02-01 | 2019-08-08 | 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 |
| US11306329B2 (en) | 2018-02-19 | 2022-04-19 | City Of Hope | Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof |
| 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 |
| KR20210008501A (ko) | 2018-05-09 | 2021-01-22 | 바이오마린 파머수티컬 인크. | 페닐케톤뇨증을 치료하는 방법 |
| 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 |
| CA3106078A1 (en) | 2018-07-17 | 2020-01-23 | Junghun Lee | Treatment of neuropathy with dna constructs expressing igf-1 isoforms |
| CN119390787A (zh) | 2019-01-31 | 2025-02-07 | 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 |
| IL298138A (en) | 2020-05-12 | 2023-01-01 | Univ 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 |
| CA3183153A1 (en) | 2020-06-17 | 2021-12-23 | Christian HINDERER | Compositions and methods for treatment of gene therapy patients |
| 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 |
| MX2023008826A (es) | 2021-02-01 | 2023-09-15 | Regenxbio Inc | Terapia génica para lipofuscinosis neuronal ceroidea. |
| ES3061188T3 (en) | 2021-09-08 | 2026-03-31 | Covestro Deutschland Ag | Succinic acid based polyester polyols |
| 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 |
| CA3247109A1 (en) | 2022-04-06 | 2023-10-12 | The Trustees Of The University Of Pennsylvania | Compositions and treatment methods for metastatic breast cancer that is human epidermal growth factor receptor 2 positive and other cancers |
| US20250250326A1 (en) | 2022-04-06 | 2025-08-07 | The Trustees Of The University Of Pennsylvania | Passive immunization with anti-aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors |
| JP2026522354A (ja) | 2023-06-12 | 2026-07-07 | 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ｉｉｉｂのためのａａｖ遺伝子療法 |
| IL326390A (en) | 2023-08-10 | 2026-04-01 | Univ 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 |
| 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 |
| 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 |
| EP2333074A1 (en) * | 2009-12-14 | 2011-06-15 | Robert Steinfeld | Substances and methods for the treatment of lysosmal storage diseases |
| 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 |
| CN103180445B (zh) * | 2010-10-22 | 2018-02-16 | 库尔纳公司 | 通过抑制α‑L‑艾杜糖醛酸酶(IDUA)的天然反义转录物而治疗IDUA相关疾病 |
| 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 |
| CA2892027A1 (en) | 2012-11-21 | 2014-05-30 | Rqx Pharmaceuticals, Inc. | Macrocyclic broad spectrum antibiotics |
| 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 |
| 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 |
| 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 |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| US11015174B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV8 |
| 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 |
| CA3068328A1 (en) | 2017-07-06 | 2019-01-10 | The Trustees Of The University Of Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
-
2014
- 2014-03-13 BR BR112015021036A patent/BR112015021036A2/pt not_active Application Discontinuation
- 2014-03-13 AU AU2014235096A patent/AU2014235096B2/en active Active
- 2014-03-13 EP EP20170476.4A patent/EP3747998B1/en active Active
- 2014-03-13 RU RU2015144234A patent/RU2708318C2/ru active
- 2014-03-13 ES ES14770186T patent/ES2805355T3/es active Active
- 2014-03-13 KR KR1020157022504A patent/KR102346455B1/ko active Active
- 2014-03-13 DK DK14770186.6T patent/DK2984166T3/da active
- 2014-03-13 CA CA2901328A patent/CA2901328C/en active Active
- 2014-03-13 HU HUE14770186A patent/HUE051373T2/hu unknown
- 2014-03-13 ES ES20170476T patent/ES2991471T3/es active Active
- 2014-03-13 US US14/769,596 patent/US10137176B2/en active Active
- 2014-03-13 PL PL14770186T patent/PL2984166T3/pl unknown
- 2014-03-13 CN CN201480010987.3A patent/CN105026554B/zh active Active
- 2014-03-13 JP JP2016501864A patent/JP6591956B2/ja active Active
- 2014-03-13 PT PT147701866T patent/PT2984166T/pt unknown
- 2014-03-13 SG SG11201507507PA patent/SG11201507507PA/en unknown
- 2014-03-13 SI SI201431605T patent/SI2984166T1/sl unknown
- 2014-03-13 SG SG10201707319UA patent/SG10201707319UA/en unknown
- 2014-03-13 EP EP14770186.6A patent/EP2984166B1/en active Active
- 2014-03-13 MX MX2015012739A patent/MX386059B/es unknown
- 2014-03-13 LT LTEP14770186.6T patent/LT2984166T/lt unknown
- 2014-03-13 WO PCT/US2014/025509 patent/WO2014151341A1/en not_active Ceased
- 2014-03-13 HR HRP20201063TT patent/HRP20201063T1/hr unknown
- 2014-03-13 KR KR1020217043023A patent/KR102581652B1/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