JP2019504003A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2019504003A5 JP2019504003A5 JP2018530566A JP2018530566A JP2019504003A5 JP 2019504003 A5 JP2019504003 A5 JP 2019504003A5 JP 2018530566 A JP2018530566 A JP 2018530566A JP 2018530566 A JP2018530566 A JP 2018530566A JP 2019504003 A5 JP2019504003 A5 JP 2019504003A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- composition
- composition according
- human subject
- genome copies
- 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
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022002190A JP2022050575A (ja) | 2015-12-11 | 2022-01-11 | 家族性高コレステロール血症を処置するための遺伝子治療 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562266383P | 2015-12-11 | 2015-12-11 | |
| US62/266,383 | 2015-12-11 | ||
| US201562269440P | 2015-12-18 | 2015-12-18 | |
| US62/269,440 | 2015-12-18 | ||
| PCT/US2016/065984 WO2017100682A1 (en) | 2015-12-11 | 2016-12-09 | Gene therapy for treating familial hypercholesterolemia |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022002190A Division JP2022050575A (ja) | 2015-12-11 | 2022-01-11 | 家族性高コレステロール血症を処置するための遺伝子治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019504003A JP2019504003A (ja) | 2019-02-14 |
| JP2019504003A5 true JP2019504003A5 (https=) | 2020-01-23 |
| JP7328760B2 JP7328760B2 (ja) | 2023-08-17 |
Family
ID=57794341
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018530566A Active JP7328760B2 (ja) | 2015-12-11 | 2016-12-09 | 家族性高コレステロール血症を処置するための遺伝子治療 |
| JP2022002190A Pending JP2022050575A (ja) | 2015-12-11 | 2022-01-11 | 家族性高コレステロール血症を処置するための遺伝子治療 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022002190A Pending JP2022050575A (ja) | 2015-12-11 | 2022-01-11 | 家族性高コレステロール血症を処置するための遺伝子治療 |
Country Status (14)
| Country | Link |
|---|---|
| US (2) | US10889832B2 (https=) |
| EP (2) | EP3400304B1 (https=) |
| JP (2) | JP7328760B2 (https=) |
| KR (1) | KR20180113990A (https=) |
| CN (2) | CN115957350A (https=) |
| AU (2) | AU2016366560A1 (https=) |
| BR (1) | BR112018011687A2 (https=) |
| CA (1) | CA3008142A1 (https=) |
| CO (1) | CO2018007165A2 (https=) |
| ES (1) | ES2921450T3 (https=) |
| IL (2) | IL259850B2 (https=) |
| MA (1) | MA43570A (https=) |
| MX (1) | MX2018007080A (https=) |
| WO (1) | WO2017100682A1 (https=) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3134431B1 (en) | 2014-04-25 | 2021-04-07 | The Trustees Of The University Of Pennsylvania | Ldlr variants and their use in compositions for reducing cholesterol levels |
| AU2016366560A1 (en) | 2015-12-11 | 2018-06-28 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| IL301963A (en) | 2016-07-21 | 2023-06-01 | Spark Therapeutics Inc | Scalable High Recovery Methods for High-Throughput Recombinant Adeno-Associated Viral Vector Production and Recombinant Adeno-Associated Viral Vectors Made So |
| 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 |
| JP2020510648A (ja) * | 2017-02-20 | 2020-04-09 | 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 | 家族性高コレステロール血症を処置するための遺伝子治療 |
| 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 |
| CN113710808A (zh) * | 2018-12-20 | 2021-11-26 | 宾夕法尼亚州大学信托人 | 用于治疗家族性高胆固醇血症的基因疗法 |
| BR112021013807A2 (pt) | 2019-01-18 | 2021-11-30 | Astrazeneca Ab | Inibidores de pcsk9 e seus métodos de uso |
| MX2021008661A (es) | 2019-01-18 | 2021-08-19 | Astrazeneca Ab | Inhibidores de la pcsk9 y metodos de uso de los mismos. |
| TWI908746B (zh) * | 2019-11-28 | 2025-12-21 | 美商銳進科斯生物股份有限公司 | 微小肌縮蛋白基因療法之構築體及其用途 |
| KR20230002594A (ko) * | 2020-04-29 | 2023-01-05 | 살리오젠 테라퓨틱스 인코포레이티드 | 가족성 고콜레스테롤혈증 및 상승된 저밀도 지단백질 콜레스테롤의 치료를 위한 조성물 및 방법 |
| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| CN117887723A (zh) * | 2024-01-08 | 2024-04-16 | 苏州诺洁贝生物技术有限公司 | 密码子优化的ldlr基因及其应用 |
Family Cites Families (44)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| US6548286B1 (en) | 1997-04-14 | 2003-04-15 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant AAV product |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| 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 |
| US7201898B2 (en) | 2000-06-01 | 2007-04-10 | The University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| CN1780910A (zh) * | 2003-01-28 | 2006-05-31 | 拉塞有限公司 | 对与家族性高胆固醇血症有关的分离的低密度脂蛋白受体(ldl－r)的基因序列中的突变进行检测的方法和装置 |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| ES2525067T3 (es) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| 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 |
| US7769466B2 (en) | 2006-02-24 | 2010-08-03 | Kenergy, Inc. | Class-E radio frequency amplifier for use with an implantable medical 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 |
| KR101010352B1 (ko) | 2008-05-30 | 2011-01-25 | 삼성중공업 주식회사 | 전력 제어 장치 및 방법 |
| JP2012516357A (ja) * | 2009-01-29 | 2012-07-19 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 神経学的障害を治療するための皮質全体に亘る高レベルの治療薬の分布方法 |
| US20120252877A1 (en) * | 2009-08-14 | 2012-10-04 | Theramind Research, Llc | Methods and compositions for treatment of tuberous sclerosis complex |
| WO2011094198A1 (en) * | 2010-01-28 | 2011-08-04 | The Children's Hospital Of Philadelphia Research Institute, Abramson Research Center | A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| WO2012177741A1 (en) | 2011-06-20 | 2012-12-27 | Genentech, Inc. | Pcsk9-binding polypeptides and methods of use |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| ES2857773T5 (es) | 2011-08-24 | 2024-06-04 | Univ Leland Stanford Junior | Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos |
| HUE069234T2 (hu) | 2011-09-16 | 2025-02-28 | Regeneron Pharma | Eljárások lipoprotein(a)-szint csökkentésére proprotein-konvertáz szubtilizin/kexin 9 (PCSK9) inhibitorának beadásával |
| MX367100B (es) * | 2012-02-17 | 2019-08-05 | The Children´S Hospital Of Philadelphia | Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos. |
| US9567376B2 (en) | 2013-02-08 | 2017-02-14 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| WO2014127196A1 (en) * | 2013-02-15 | 2014-08-21 | The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services | Aav8 retinoschisin expression vector for treating x-linked retinoschisis |
| US8980864B2 (en) * | 2013-03-15 | 2015-03-17 | Moderna Therapeutics, Inc. | Compositions and methods of altering cholesterol levels |
| AU2014235096B2 (en) * | 2013-03-15 | 2020-03-19 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating MPSI |
| WO2015012924A2 (en) | 2013-04-29 | 2015-01-29 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof |
| EP3052521A1 (en) | 2013-10-03 | 2016-08-10 | Moderna Therapeutics, Inc. | Polynucleotides encoding low density lipoprotein receptor |
| EP3134431B1 (en) | 2014-04-25 | 2021-04-07 | The Trustees Of The University Of Pennsylvania | Ldlr variants and their use in compositions for reducing cholesterol levels |
| ES2833230T3 (es) * | 2014-05-13 | 2021-06-14 | Univ Pennsylvania | Composiciones que comprenden AVV que expresan construcciones y usos de doble anticuerpo del mismo |
| ES2934848T3 (es) | 2015-12-11 | 2023-02-27 | Univ Pennsylvania | Método de purificación escalable para AAV8 |
| AU2016366560A1 (en) | 2015-12-11 | 2018-06-28 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| JP2020510648A (ja) | 2017-02-20 | 2020-04-09 | 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 | 家族性高コレステロール血症を処置するための遺伝子治療 |
-
2016
- 2016-12-09 AU AU2016366560A patent/AU2016366560A1/en not_active Abandoned
- 2016-12-09 KR KR1020187019657A patent/KR20180113990A/ko not_active Ceased
- 2016-12-09 CN CN202211286244.1A patent/CN115957350A/zh active Pending
- 2016-12-09 MX MX2018007080A patent/MX2018007080A/es unknown
- 2016-12-09 MA MA043570A patent/MA43570A/fr unknown
- 2016-12-09 EP EP16825936.4A patent/EP3400304B1/en active Active
- 2016-12-09 JP JP2018530566A patent/JP7328760B2/ja active Active
- 2016-12-09 BR BR112018011687A patent/BR112018011687A2/pt not_active IP Right Cessation
- 2016-12-09 EP EP22165782.8A patent/EP4085934A1/en not_active Withdrawn
- 2016-12-09 CA CA3008142A patent/CA3008142A1/en active Pending
- 2016-12-09 CN CN201680081580.9A patent/CN109121421B/zh active Active
- 2016-12-09 US US16/060,409 patent/US10889832B2/en active Active
- 2016-12-09 IL IL259850A patent/IL259850B2/en unknown
- 2016-12-09 WO PCT/US2016/065984 patent/WO2017100682A1/en not_active Ceased
- 2016-12-09 IL IL303850A patent/IL303850A/en unknown
- 2016-12-09 ES ES16825936T patent/ES2921450T3/es active Active
-
2018
- 2018-07-09 CO CONC2018/0007165A patent/CO2018007165A2/es unknown
-
2020
- 2020-11-17 US US16/950,272 patent/US20210285013A1/en not_active Abandoned
-
2022
- 2022-01-11 JP JP2022002190A patent/JP2022050575A/ja active Pending
-
2023
- 2023-03-16 AU AU2023201657A patent/AU2023201657A1/en not_active Abandoned
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2019504003A5 (https=) | ||
| JP2020510648A5 (https=) | ||
| Raina et al. | An update on LDL apheresis for nephrotic syndrome | |
| Schiedner et al. | Selective depletion or blockade of Kupffer cells leads to enhanced and prolonged hepatic transgene expression using high-capacity adenoviral vectors | |
| Xun et al. | Metformin inhibits hepatitis B virus protein production and replication in human hepatoma cells | |
| Pei et al. | Efficient capsid antigen presentation from adeno-associated virus empty virions in vivo | |
| BR112021023692A2 (pt) | Partícula viral de vírus adeno-associado recombinante, proteína de capsídeo de vírus adeno-associado, molécula de ácido nucleico, célula empacotadora, métodos de produção de uma partícula viral e de distribuição de um nucleotídeo, e, composição farmacêutica | |
| JP2021523198A5 (https=) | ||
| JP2018535929A5 (https=) | ||
| JP2020537637A5 (https=) | ||
| RU2020126034A (ru) | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции | |
| JP2016538885A5 (https=) | ||
| RU2022102472A (ru) | Фиксированные комбинации и составы, содержащие etc-1002 и эзетимиб, и способы лечения или уменьшения риска развития сердечно-сосудистого заболевания | |
| Descamps et al. | Two key challenges for effective adenovirus-mediated liver gene therapy: innate immune responses and hepatocyte-specific transduction | |
| EP3134113A1 (en) | Methods of permitting a subject to receive multiple doses of recombinant adeno-associated virus | |
| JP2024045289A (ja) | メチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルスの髄腔内送達 | |
| Ayoobi et al. | Reduced expression of TRIF in chronic HBV infected Iranian patients | |
| JP2018502112A5 (https=) | ||
| Notarte et al. | Infusion reactions to adeno‐associated virus (AAV)‐based gene therapy: Mechanisms, diagnostics, treatment and review of the literature | |
| Pipe et al. | First-in-human dose-finding study of AAVhu37 vector-based gene therapy: BAY 2599023 has stable and sustained expression of FVIII over 2 years | |
| JP2003501114A (ja) | 治療用ウイルス免疫原性剤投与の有効性を増強する方法 | |
| JP2022060514A (ja) | ガバペンチノイドからの干渉が減少し、ｈｇｆ異型体を発現するｄｎａコンストラクトを用いる神経病症の治療 | |
| King et al. | High-capacity adenovirus vector-mediated anti-glioma gene therapy in the presence of systemic antiadenovirus immunity | |
| JP5665899B2 (ja) | 癌の処置のための粘液腫ウイルス変異体 | |
| JP2021500880A5 (https=) |