JP2017513504A5 - - Google Patents
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- Publication number
- JP2017513504A5 JP2017513504A5 JP2016564200A JP2016564200A JP2017513504A5 JP 2017513504 A5 JP2017513504 A5 JP 2017513504A5 JP 2016564200 A JP2016564200 A JP 2016564200A JP 2016564200 A JP2016564200 A JP 2016564200A JP 2017513504 A5 JP2017513504 A5 JP 2017513504A5
- Authority
- JP
- Japan
- Prior art keywords
- hldlr
- modified
- amino acid
- raav vector
- expression
- 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 (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461984620P | 2014-04-25 | 2014-04-25 | |
| US61/984,620 | 2014-04-25 | ||
| US201462022627P | 2014-07-09 | 2014-07-09 | |
| US62/022,627 | 2014-07-09 | ||
| PCT/US2015/027572 WO2015164778A1 (en) | 2014-04-25 | 2015-04-24 | Ldlr variants and their use in compositions for reducing cholesterol levels |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020074046A Division JP7280853B2 (ja) | 2014-04-25 | 2020-04-17 | コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017513504A JP2017513504A (ja) | 2017-06-01 |
| JP2017513504A5 true JP2017513504A5 (enExample) | 2018-06-07 |
| JP6741590B2 JP6741590B2 (ja) | 2020-08-19 |
Family
ID=53055129
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016564200A Active JP6741590B2 (ja) | 2014-04-25 | 2015-04-24 | コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用 |
| JP2020074046A Active JP7280853B2 (ja) | 2014-04-25 | 2020-04-17 | コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用 |
| JP2023078988A Pending JP2023100960A (ja) | 2014-04-25 | 2023-05-12 | コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020074046A Active JP7280853B2 (ja) | 2014-04-25 | 2020-04-17 | コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用 |
| JP2023078988A Pending JP2023100960A (ja) | 2014-04-25 | 2023-05-12 | コレステロールレベルを低下させるためのｌｄｌｒ変異体および組成物中でのそれらの使用 |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US11555059B2 (enExample) |
| EP (2) | EP3134431B1 (enExample) |
| JP (3) | JP6741590B2 (enExample) |
| BR (1) | BR112016024379A2 (enExample) |
| CA (1) | CA2946392A1 (enExample) |
| ES (1) | ES2876409T3 (enExample) |
| WO (1) | WO2015164778A1 (enExample) |
Families Citing this family (49)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20160067219A (ko) | 2013-10-03 | 2016-06-13 | 모더나 세라퓨틱스, 인코포레이티드 | 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드 |
| CA2946392A1 (en) | 2014-04-25 | 2015-10-29 | James M. Wilson | Ldlr variants and their use in compositions for reducing cholesterol levels |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| CN107106689A (zh) | 2014-11-05 | 2017-08-29 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| IL292999A (en) | 2014-11-14 | 2022-07-01 | Voyager Therapeutics Inc | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| CN112410338A (zh) | 2014-11-14 | 2021-02-26 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| WO2016094783A1 (en) | 2014-12-12 | 2016-06-16 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| WO2016164762A1 (en) * | 2015-04-08 | 2016-10-13 | Moderna Therapeutics, Inc. | Polynucleotides encoding low density lipoprotein receptor egf-a and intracellular domain mutants and methods of using the same |
| EA201792500A1 (ru) | 2015-05-13 | 2018-04-30 | Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания | Aav-опосредованная экспрессия антител против гриппа и способы их использования |
| CA3008142A1 (en) * | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| WO2017180936A1 (en) * | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsylvania | Compositions for treatment of wet age-related macular degeneration |
| WO2017189964A2 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| US11326182B2 (en) | 2016-04-29 | 2022-05-10 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| KR20220108216A (ko) | 2016-05-18 | 2022-08-02 | 보이저 테라퓨틱스, 인크. | 헌팅톤 질환을 치료하기 위한 조성물 및 방법 |
| SG11201809699XA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| 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 |
| CN110650673B (zh) | 2016-08-30 | 2024-04-09 | 加利福尼亚大学董事会 | 用于生物医学靶向和递送的方法以及用于实践该方法的装置和系统 |
| BR112019013576A2 (pt) * | 2016-12-30 | 2020-02-04 | The Trustees Of The University Of Pennsylvania | terapia genica para o tratamento da fenilcetonuria |
| KR20190118163A (ko) * | 2017-02-20 | 2019-10-17 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법 |
| MX2019010275A (es) | 2017-02-28 | 2019-10-15 | Univ Pennsylvania | Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este. |
| BR112019017697A2 (pt) | 2017-02-28 | 2020-04-07 | The Trustees Of The University Of Pennsylvania | vacinas contra influenza baseadas em vetores de vírus adenoassociado (aav) |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| AU2018254576B2 (en) * | 2017-04-21 | 2022-12-22 | Precision Biosciences, Inc. | Engineered meganucleases specific for recognition sequences in the PCSK9 gene |
| JP2020518259A (ja) | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | ハンチントン病治療組成物および方法 |
| SG11201909870SA (en) | 2017-05-05 | 2019-11-28 | Voyager Therapeutics Inc | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| JP7229989B2 (ja) | 2017-07-17 | 2023-02-28 | ボイジャー セラピューティクス インコーポレイテッド | 軌道アレイガイドシステム |
| TWI832036B (zh) | 2017-08-03 | 2024-02-11 | 美商航海家醫療公司 | 用於aav之遞送之組合物及方法 |
| MX2020003042A (es) | 2017-09-29 | 2020-11-18 | Voyager Therapeutics Inc | Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa. |
| US11434502B2 (en) | 2017-10-16 | 2022-09-06 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
| EP4454654A3 (en) | 2017-10-16 | 2025-02-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| US12558434B2 (en) | 2018-02-20 | 2026-02-24 | The Trustees Of The University Of Pennsylvania | Compositions for treatment of wet age-related macular degeneration |
| EP3794126A1 (en) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| CN113383010B (zh) | 2018-09-28 | 2025-09-09 | 沃雅戈治疗公司 | 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法 |
| AU2019401638A1 (en) * | 2018-12-20 | 2021-06-24 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| JP2023518415A (ja) * | 2020-03-19 | 2023-05-01 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | 組換えａａｖにおいてｃａｐおよびｒｅｐ配列の逆パッケージングを低減するための組成物および方法 |
| IL297756A (en) * | 2020-04-29 | 2022-12-01 | Saliogen Therapeutics Inc | Compositions and methods for treatment of familial hypercholesterolemia and elevated low-density lipoprotein cholesterol |
| CA3177407A1 (en) | 2020-05-12 | 2021-11-18 | James M. Wilson | Compositions for drg-specific reduction of transgene expression |
| TW202221127A (zh) | 2020-08-14 | 2022-06-01 | 賓州大學委員會 | 新穎aav衣殼及含有其之組成物 |
| CA3196499A1 (en) | 2020-10-29 | 2022-05-05 | James M. Wilson | Aav capsids and compositions containing same |
| AR124216A1 (es) | 2020-12-01 | 2023-03-01 | Univ Pennsylvania | Composiciones nuevas con motivos selectivos específicos del tejido y composiciones que las contienen |
| MX2023012513A (es) | 2021-04-23 | 2023-12-15 | Univ Pennsylvania | Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen. |
| AR125467A1 (es) | 2021-04-27 | 2023-07-19 | Univ Pennsylvania | Cápsides de virus adenoasociados derivados de porcinos y usos de los estos |
| CN113684215A (zh) * | 2021-09-07 | 2021-11-23 | 华中科技大学同济医学院附属协和医院 | 高胆固醇血症相关的新突变基因及其应用 |
| TW202325845A (zh) | 2021-10-02 | 2023-07-01 | 賓州大學委員會 | 新穎aav衣殼及含其之組成物 |
| WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| US20260021205A1 (en) | 2022-07-15 | 2026-01-22 | The Trustees Of The University Of Pennsylvania | Recombinant aav having aav clade d and clade e capsids and compositions containing same |
| AR133098A1 (es) | 2023-06-29 | 2025-08-27 | Univ Pennsylvania | Aav mutante con motivos dirigidos al sistema nervioso central y composiciones que lo contienen |
| WO2025231085A1 (en) * | 2024-04-30 | 2025-11-06 | Airna Corporation | Site-directed editing of rna |
Family Cites Families (49)
| 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 |
| US5817789A (en) * | 1995-06-06 | 1998-10-06 | Transkaryotic Therapies, Inc. | Chimeric proteins for use in transport of a selected substance into cells |
| 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 |
| AU728220B2 (en) | 1997-04-14 | 2001-01-04 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant AAV product |
| EP1080218A1 (en) | 1998-05-27 | 2001-03-07 | University of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient |
| ATE454445T1 (de) | 1998-11-10 | 2010-01-15 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
| 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 |
| WO2001027303A1 (en) * | 1999-10-12 | 2001-04-19 | The University Of North Carolina At Chapel Hill | Adeno-associated virus vectors encoding factor viii and methods of using the same |
| CA2410901C (en) | 2000-06-01 | 2012-03-20 | University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| 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 |
| US20070015238A1 (en) | 2002-06-05 | 2007-01-18 | Snyder Richard O | Production of pseudotyped recombinant AAV virions |
| HUE054805T2 (hu) | 2003-09-30 | 2021-09-28 | Univ Pennsylvania | Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik |
| CN101018858A (zh) | 2004-06-01 | 2007-08-15 | 建新公司 | 防止aav载体聚集的组合物和方法 |
| 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 |
| US7943374B2 (en) * | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
| WO2007100682A1 (en) | 2006-02-24 | 2007-09-07 | Kenergy, Inc. | Class-e radio frequency amplifier for use with an implantable medical device |
| 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 |
| KR101010352B1 (ko) | 2008-05-30 | 2011-01-25 | 삼성중공업 주식회사 | 전력 제어 장치 및 방법 |
| EP2391388A1 (en) | 2009-01-29 | 2011-12-07 | University of California-San Francisco | Methods for distributing high levels of therapeutic agent throughout the cortex to treat neurological disorders |
| WO2011020118A1 (en) | 2009-08-14 | 2011-02-17 | Theramind Research, Llc | Methods and compositions for treatment of tuberous sclerosis complex |
| ES2680915T3 (es) | 2010-01-28 | 2018-09-11 | The Children's Hospital Of Philadelphia | Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica |
| KR20130040844A (ko) | 2010-03-29 | 2013-04-24 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 약학적으로 유발된 전이유전자 제거 시스템 |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| KR20140041747A (ko) * | 2011-06-20 | 2014-04-04 | 제넨테크, 인크. | Pcsk9-결합 폴리펩티드 및 사용 방법 |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| EP3147295B2 (en) | 2011-08-24 | 2023-11-22 | The Board of Trustees of the Leland Stanford Junior University | New avv capsid proteins for nucleic acid transfer |
| HUE069234T2 (hu) | 2011-09-16 | 2025-02-28 | Regeneron Pharma | Eljárások lipoprotein(a)-szint csökkentésére proprotein-konvertáz szubtilizin/kexin 9 (PCSK9) inhibitorának beadásával |
| SG11201404741VA (en) * | 2012-02-07 | 2014-09-26 | Global Bio Therapeutics Usa Inc | Compartmentalized method of nucleic acid delivery and compositions and uses thereof |
| KR102057540B1 (ko) | 2012-02-17 | 2019-12-19 | 더 칠드런스 호스피탈 오브 필라델피아 | 세포, 기관 및 조직으로의 유전자 전이를 위한 ａａｖ 벡터 조성물 및 방법 |
| 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 |
| ES2741502T3 (es) | 2013-02-08 | 2020-02-11 | Univ Pennsylvania | Cápside de AAV8 modificada para la transferencia génica para terapias de retina |
| AU2014216160B2 (en) | 2013-02-15 | 2017-03-30 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | AAV8 retinoschisin expression vector for treating X-linked retinoschisis |
| 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 |
| 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 |
| US20150065556A1 (en) * | 2013-08-05 | 2015-03-05 | Whitehead Institute For Biomedical Research | Therapeutic targets for mitochondrial disorders |
| KR20160067219A (ko) * | 2013-10-03 | 2016-06-13 | 모더나 세라퓨틱스, 인코포레이티드 | 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드 |
| 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 |
| WO2017100676A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| KR20190118163A (ko) | 2017-02-20 | 2019-10-17 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법 |
-
2015
- 2015-04-24 CA CA2946392A patent/CA2946392A1/en active Pending
- 2015-04-24 EP EP15721097.2A patent/EP3134431B1/en active Active
- 2015-04-24 JP JP2016564200A patent/JP6741590B2/ja active Active
- 2015-04-24 BR BR112016024379A patent/BR112016024379A2/pt active Search and Examination
- 2015-04-24 WO PCT/US2015/027572 patent/WO2015164778A1/en not_active Ceased
- 2015-04-24 ES ES15721097T patent/ES2876409T3/es active Active
- 2015-04-24 US US15/306,419 patent/US11555059B2/en active Active
- 2015-04-24 EP EP21166865.2A patent/EP3919508A1/en not_active Withdrawn
-
2020
- 2020-04-17 JP JP2020074046A patent/JP7280853B2/ja active Active
-
2022
- 2022-11-16 US US18/055,928 patent/US20230272032A1/en not_active Abandoned
-
2023
- 2023-05-12 JP JP2023078988A patent/JP2023100960A/ja active Pending
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