JP2017513504A5 - - Google Patents
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- 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) | EP3919508A1 (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 (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2016538829A (ja) | 2013-10-03 | 2016-12-15 | モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 低密度リポタンパク質受容体をコードするポリヌクレオチド |
| BR112016024379A2 (pt) | 2014-04-25 | 2017-10-10 | Univ Pennsylvania | variantes ldlr e seu uso em composições para reduzir os níveis de colesterol |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| SG10202007103TA (en) | 2014-11-05 | 2020-09-29 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| IL284949B2 (en) | 2014-11-14 | 2024-06-01 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| MX2017006216A (es) | 2014-11-14 | 2018-08-29 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela). |
| 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 |
| EP3307310A2 (en) | 2015-05-13 | 2018-04-18 | The Trustees Of The University Of Pennsylvania | Aav-mediated expression of anti-influenza antibodies and methods of use thereof |
| EP3400304B1 (en) * | 2015-12-11 | 2022-04-06 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| JP7046828B2 (ja) * | 2016-04-15 | 2022-04-04 | 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 | 滲出型加齢性黄斑変性の治療のための組成物 |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| AU2017268382B2 (en) | 2016-05-18 | 2023-09-28 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
| EP3458588B1 (en) | 2016-05-18 | 2025-10-29 | 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 |
| US11298041B2 (en) | 2016-08-30 | 2022-04-12 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| WO2018126112A1 (en) * | 2016-12-30 | 2018-07-05 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating phenylketonuria |
| US11554147B2 (en) | 2017-02-20 | 2023-01-17 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| CN110573523A (zh) | 2017-02-28 | 2019-12-13 | 宾夕法尼亚州大学信托人 | 基于aav载体的流感疫苗 |
| EP3589730B1 (en) | 2017-02-28 | 2023-12-27 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clade f vector and uses therefor |
| WO2018195449A1 (en) * | 2017-04-21 | 2018-10-25 | Precision Biosciences, Inc. | Engineered meganucleases specific for recognition sequences in the pcsk9 gene |
| CN111108198A (zh) | 2017-05-05 | 2020-05-05 | 沃雅戈治疗公司 | 治疗亨廷顿病的组合物和方法 |
| EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | 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 |
| AU2018302016A1 (en) | 2017-07-17 | 2020-02-06 | The Regents Of The University Of California | Trajectory array guide system |
| 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 |
| EP3687582A4 (en) | 2017-09-29 | 2021-07-14 | Voyager Therapeutics, Inc. | RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION |
| CN111479924B (zh) | 2017-10-16 | 2024-06-14 | 沃雅戈治疗公司 | 肌萎缩性侧索硬化症(als)的治疗 |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| MA52631A (fr) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics Inc | Compositions et méthodes pour le traitement de la maladie de parkinson |
| JP7528066B2 (ja) | 2018-09-28 | 2024-08-05 | ボイジャー セラピューティクス インコーポレイテッド | 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法 |
| WO2020132155A1 (en) * | 2018-12-20 | 2020-06-25 | 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 |
| EP4142877A4 (en) * | 2020-04-29 | 2024-07-03 | Saliogen Therapeutics, Inc. | Compositions and methods for treatment of familial hypercholesterolemia and elevated low-density lipoprotein cholesterol |
| BR112022022212A2 (pt) | 2020-05-12 | 2022-12-13 | Univ Pennsylvania | Composições para redução específica de drg de expressão de transgene |
| 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 |
| CN117042787A (zh) | 2020-10-29 | 2023-11-10 | 宾夕法尼亚州大学信托人 | Aav衣壳和含有aav衣壳的组合物 |
| AU2021390480A1 (en) | 2020-12-01 | 2023-06-22 | The Trustees Of The University Of Pennsylvania | Novel compositions with tissue-specific targeting motifs and compositions containing same |
| TW202305124A (zh) | 2021-04-23 | 2023-02-01 | 賓州大學委員會 | 具有腦特異性靶向模體的新穎構成物及含有其之組成物 |
| TW202309066A (zh) | 2021-04-27 | 2023-03-01 | 賓州大學委員會 | 衍生自豬的腺相關病毒衣殼及其用途 |
| 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 |
| WO2023056399A1 (en) | 2021-10-02 | 2023-04-06 | The Trustees Of The University Of Pennsylvania | Novel aav capsids and compositions containing same |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | 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 |
| TW202516019A (zh) | 2023-06-29 | 2025-04-16 | 賓州大學委員會 | 具中樞神經系統靶向模體的突變aav及含有其之組成物 |
| 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 |
| 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 |
| 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 |
| EP1007637B1 (en) | 1997-04-14 | 2004-06-30 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant aav product |
| WO1999061643A1 (en) | 1998-05-27 | 1999-12-02 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixananol gradient |
| 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 |
| ES2308989T3 (es) | 1999-08-09 | 2008-12-16 | Targeted Genetics Corporation | Aumento de la expresion de una secuencia nucleotidica heterologa a partir de vectores viricos recombinantes que contienen una secuencia que forman pares de bases intracatenarios. |
| AU7879000A (en) * | 1999-10-12 | 2001-04-23 | University Of North Carolina At Chapel Hill, The | Adeno-associated virus vectors encoding factor viii and methods of using the same |
| AU6814901A (en) | 2000-06-01 | 2001-12-11 | Univ North Carolina | Methods and compounds for controlled release of recombinant parvovirus vectors |
| IL161827A0 (en) | 2001-11-13 | 2005-11-20 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novelsequences identified thereby |
| CA2469785C (en) | 2001-12-17 | 2014-04-22 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor |
| WO2003104413A2 (en) | 2002-06-05 | 2003-12-18 | University Of Florida | Production of pseudotyped recombinant aav virions |
| EP3211085B1 (en) | 2003-09-30 | 2021-03-31 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| 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 |
| CN104293835B (zh) | 2005-04-07 | 2017-07-04 | 宾夕法尼亚大学托管会 | 增强腺相关病毒载体功能的方法 |
| 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 |
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| 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 |
| WO2010088560A1 (en) | 2009-01-29 | 2010-08-05 | 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 |
| MX342858B (es) | 2010-03-29 | 2016-10-13 | The Trustees Of The Univ Of Pennsylvania * | Sistema de ablacion transgenica inducida farmacologicamente. |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| CN103717615A (zh) * | 2011-06-20 | 2014-04-09 | 霍夫曼-拉罗奇有限公司 | 结合pcsk9的多肽及使用方法 |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| EP2748185A1 (en) | 2011-08-24 | 2014-07-02 | The Board of Trustees of The Leland Stanford Junior University | New aav capsid proteins for nucleic acid transfer |
| MY170089A (en) | 2011-09-16 | 2019-07-04 | Regeneron Pharma | Methods for reducing lipoproten(a) levels by administering an inhibitor of proprotein convertase subtilisin kexin-9 (pcsk9) |
| BR112014019431A8 (pt) * | 2012-02-07 | 2017-07-11 | Global Bio Therapeutics Usa Inc | Método compartimentalizado de distribuição de ácido nucleico e composições e usos desses |
| 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 |
| EP2929017A4 (en) * | 2012-12-05 | 2016-09-28 | Sangamo Biosciences Inc | METHOD AND COMPOSITIONS FOR REGULATING METABOLISM DISEASES |
| US9567376B2 (en) | 2013-02-08 | 2017-02-14 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| US9873893B2 (en) | 2013-02-15 | 2018-01-23 | The United States Of America As Represented By The Secretary, Dept. Of Health And Human Services | Methods and compositions for treating genetically linked diseases of the eye |
| US9254332B2 (en) | 2013-03-15 | 2016-02-09 | Arecor Limited | Stable aqueous formulations of adenovirus vectors |
| PL2984166T3 (pl) * | 2013-03-15 | 2021-01-11 | The Trustees Of The University Of Pennsylvania | Kompozycje do leczenia mpsi |
| US9719106B2 (en) | 2013-04-29 | 2017-08-01 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof |
| US20150065556A1 (en) * | 2013-08-05 | 2015-03-05 | Whitehead Institute For Biomedical Research | Therapeutic targets for mitochondrial disorders |
| JP2016538829A (ja) * | 2013-10-03 | 2016-12-15 | モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 低密度リポタンパク質受容体をコードするポリヌクレオチド |
| BR112016024379A2 (pt) | 2014-04-25 | 2017-10-10 | Univ Pennsylvania | variantes ldlr e seu uso em composições para reduzir os níveis de colesterol |
| EP3387117B1 (en) | 2015-12-11 | 2022-11-23 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| EP3400304B1 (en) | 2015-12-11 | 2022-04-06 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| US11554147B2 (en) | 2017-02-20 | 2023-01-17 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
-
2015
- 2015-04-24 BR BR112016024379A patent/BR112016024379A2/pt active Search and Examination
- 2015-04-24 CA CA2946392A patent/CA2946392A1/en active Pending
- 2015-04-24 JP JP2016564200A patent/JP6741590B2/ja active Active
- 2015-04-24 WO PCT/US2015/027572 patent/WO2015164778A1/en not_active Ceased
- 2015-04-24 EP EP21166865.2A patent/EP3919508A1/en not_active Withdrawn
- 2015-04-24 EP EP15721097.2A patent/EP3134431B1/en active Active
- 2015-04-24 ES ES15721097T patent/ES2876409T3/es active Active
- 2015-04-24 US US15/306,419 patent/US11555059B2/en active Active
-
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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