JP2020510648A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2020510648A5 JP2020510648A5 JP2019545363A JP2019545363A JP2020510648A5 JP 2020510648 A5 JP2020510648 A5 JP 2020510648A5 JP 2019545363 A JP2019545363 A JP 2019545363A JP 2019545363 A JP2019545363 A JP 2019545363A JP 2020510648 A5 JP2020510648 A5 JP 2020510648A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- suspension
- raav
- patient
- genome
- 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
Links
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023030651A JP2023071829A (ja) | 2017-02-20 | 2023-03-01 | 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 (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762461015P | 2017-02-20 | 2017-02-20 | |
| US62/461,015 | 2017-02-20 | ||
| PCT/US2018/018678 WO2018152485A1 (en) | 2017-02-20 | 2018-02-20 | Gene therapy for treating familial hypercholesterolemia |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023030651A Division JP2023071829A (ja) | 2017-02-20 | 2023-03-01 | 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 (2)
| Publication Number | Publication Date |
|---|---|
| JP2020510648A JP2020510648A (ja) | 2020-04-09 |
| JP2020510648A5 true JP2020510648A5 (https=) | 2021-03-25 |
Family
ID=63170484
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019545363A Pending JP2020510648A (ja) | 2017-02-20 | 2018-02-20 | 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 |
| JP2023030651A Pending JP2023071829A (ja) | 2017-02-20 | 2023-03-01 | 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 |
|---|---|---|---|
| JP2023030651A Pending JP2023071829A (ja) | 2017-02-20 | 2023-03-01 | 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 (8)
| Country | Link |
|---|---|
| US (2) | US11554147B2 (https=) |
| EP (1) | EP3595688A4 (https=) |
| JP (2) | JP2020510648A (https=) |
| KR (1) | KR20190118163A (https=) |
| AU (1) | AU2018220212A1 (https=) |
| CA (1) | CA3051011A1 (https=) |
| IL (1) | IL268276A (https=) |
| WO (1) | WO2018152485A1 (https=) |
Families Citing this family (6)
| 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 |
| 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 |
| WO2021080975A1 (en) * | 2019-10-21 | 2021-04-29 | The Trustees Of The University Of Pennsylvania | Compositions and methods for reducing cholesterol levels |
| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| WO2023081807A1 (en) * | 2021-11-04 | 2023-05-11 | The Trustees of the University of Pennsylvania Penn Center for Innovation | Compositions and methods for reducing pcsk9 levels in a subject |
Family Cites Families (41)
| 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 |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| 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 |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| 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 |
| 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 |
| 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. |
| US9255250B2 (en) | 2012-12-05 | 2016-02-09 | Sangamo Bioscience, Inc. | Isolated mouse or human cell having an exogenous transgene in an endogenous albumin gene |
| 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 |
| US9254332B2 (en) | 2013-03-15 | 2016-02-09 | Arecor Limited | Stable aqueous formulations of adenovirus vectors |
| 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 |
| 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 |
-
2018
- 2018-02-20 JP JP2019545363A patent/JP2020510648A/ja active Pending
- 2018-02-20 EP EP18754347.5A patent/EP3595688A4/en not_active Withdrawn
- 2018-02-20 CA CA3051011A patent/CA3051011A1/en active Pending
- 2018-02-20 KR KR1020197024773A patent/KR20190118163A/ko not_active Ceased
- 2018-02-20 WO PCT/US2018/018678 patent/WO2018152485A1/en not_active Ceased
- 2018-02-20 AU AU2018220212A patent/AU2018220212A1/en not_active Abandoned
- 2018-02-20 US US16/486,981 patent/US11554147B2/en active Active
-
2019
- 2019-07-25 IL IL268276A patent/IL268276A/en unknown
-
2022
- 2022-11-22 US US18/058,126 patent/US20230190837A1/en not_active Abandoned
-
2023
- 2023-03-01 JP JP2023030651A patent/JP2023071829A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2020510648A5 (https=) | ||
| JP2019504003A5 (https=) | ||
| JP7808674B2 (ja) | Ａａｖウイルスベクター及びその使用 | |
| Bharucha-Goebel et al. | Intrathecal gene therapy for giant axonal neuropathy | |
| Naveed et al. | Onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy | |
| EP4427808A2 (en) | Compositions and methods for reducing ocular neovascularization | |
| JP2018535929A5 (https=) | ||
| Pei et al. | Efficient capsid antigen presentation from adeno-associated virus empty virions in vivo | |
| CN110325219B (zh) | 编码甲基-CpG结合蛋白2的重组腺相关病毒的鞘内递送 | |
| JP2008055195A (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 | |
| EP3134113A1 (en) | Methods of permitting a subject to receive multiple doses of recombinant adeno-associated virus | |
| JP2022523766A5 (https=) | ||
| Notarte et al. | Infusion reactions to adeno‐associated virus (AAV)‐based gene therapy: Mechanisms, diagnostics, treatment and review of the literature | |
| 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 | |
| Lauver et al. | The CXCR6-CXCL16 axis mediates T cell control of polyomavirus infection in the kidney | |
| Zhao et al. | The therapeutic potential of Rho kinase inhibitor fasudil derivative FaD-1 in experimental autoimmune encephalomyelitis | |
| Wang et al. | Suppression of interleukin-6 increases enterovirus A71 lethality in mice | |
| US20200062820A1 (en) | Method for Treating Ischemic Tissue | |
| Kumar et al. | AAV viral vectors as therapeutic interventions for inherited or non-inherited cardiac disorders: current aspects and future prospects | |
| Kuragano et al. | Effectiveness of β2-microglobulin adsorption column in treating dialysis-related amyloidosis: a multicenter study | |
| Rebrikov et al. | TWO-STEP AAV8 GENE DELIVERY IN A CHILD WITH CRIGLER–NAJJAR SYNDROME TYPE I | |
| JP2022518354A5 (https=) | ||
| AlMohammedi et al. | Pulse‐steroid therapy in a 37‐year‐old man with acute motor and sensory axonal neuropathy: A case report | |
| Mittal et al. | Therapeutic plasma exchange in Guillain Barr’e Syndrome, an experience of our centre |