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
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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 (cg-RX-API-DMAC7.html) | 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 (cg-RX-API-DMAC7.html) |
| EP (2) | EP3134431B1 (cg-RX-API-DMAC7.html) |
| JP (3) | JP6741590B2 (cg-RX-API-DMAC7.html) |
| BR (1) | BR112016024379A2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA2946392A1 (cg-RX-API-DMAC7.html) |
| ES (1) | ES2876409T3 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2015164778A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20160067219A (ko) | 2013-10-03 | 2016-06-13 | 모더나 세라퓨틱스, 인코포레이티드 | 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드 |
| 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 |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| RU2020109343A (ru) | 2014-11-05 | 2020-03-17 | Вояджер Терапьютикс, Инк. | Полинуклеотиды aadc для лечения болезни паркинсона |
| CN112375760A (zh) | 2014-11-14 | 2021-02-19 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| WO2016077687A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| 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 |
| IL259850B2 (en) * | 2015-12-11 | 2023-11-01 | Univ Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| IL322403A (en) * | 2016-04-15 | 2025-09-01 | Univ Pennsylvania | Preparations for the treatment of wet age-related macular degeneration |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| WO2017189959A1 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| SG11201809643UA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| IL302748B1 (en) | 2016-05-18 | 2025-09-01 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| 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 |
| JP2019531787A (ja) | 2016-08-30 | 2019-11-07 | 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 | 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム |
| WO2018126112A1 (en) * | 2016-12-30 | 2018-07-05 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating phenylketonuria |
| CA3051011A1 (en) * | 2017-02-20 | 2018-08-23 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| KR20190135000A (ko) | 2017-02-28 | 2019-12-05 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | Aav 벡터를 기반으로 하는 인플루엔자 백신 |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| CN118956781A (zh) | 2017-02-28 | 2024-11-15 | 宾夕法尼亚州大学信托人 | 腺相关病毒(aav)进化枝f载体及其用途 |
| ES2953153T3 (es) * | 2017-04-21 | 2023-11-08 | Prec Biosciences Inc | Meganucleasas diseñadas específicas para secuencias de reconocimiento en el gen pcsk9 |
| EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| US11752181B2 (en) | 2017-05-05 | 2023-09-12 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| EP3654860A1 (en) | 2017-07-17 | 2020-05-27 | Voyager Therapeutics, Inc. | Trajectory array guide system |
| CN111448308A (zh) | 2017-08-03 | 2020-07-24 | 沃雅戈治疗公司 | 递送aav的组合物和方法 |
| WO2019067840A1 (en) | 2017-09-29 | 2019-04-04 | Voyager Therapeutics, Inc. | RESTRICTING CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE FROM FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| 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 |
| CA3099306A1 (en) | 2018-05-15 | 2019-11-21 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| 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 |
| EP4121544A1 (en) * | 2020-03-19 | 2023-01-25 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav |
| KR20230002594A (ko) * | 2020-04-29 | 2023-01-05 | 살리오젠 테라퓨틱스 인코포레이티드 | 가족성 고콜레스테롤혈증 및 상승된 저밀도 지단백질 콜레스테롤의 치료를 위한 조성물 및 방법 |
| BR112022022212A2 (pt) | 2020-05-12 | 2022-12-13 | Univ Pennsylvania | Composições para redução específica de drg de expressão de transgene |
| IL300410A (en) | 2020-08-14 | 2023-04-01 | Univ Pennsylvania | Novel AAV capsids and compositions containing them |
| JP2023550581A (ja) | 2020-10-29 | 2023-12-04 | 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 | Ａａｖカプシド及びそれを含有する組成物 |
| MX2023006444A (es) | 2020-12-01 | 2023-08-10 | Univ Pennsylvania | Composiciones novedosas con motivos dirigidos a tejido específico y composiciones que los contienen. |
| WO2022226263A1 (en) | 2021-04-23 | 2022-10-27 | The Trustees Of The University Of Pennsylvania | Novel compositions with brain-specific targeting motifs and compositions containing same |
| 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 |
| EP4409010A1 (en) | 2021-10-02 | 2024-08-07 | 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 |
| WO2024015966A2 (en) | 2022-07-15 | 2024-01-18 | 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 |
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| 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 |
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| 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 |
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| MX346493B (es) | 2001-11-13 | 2017-03-21 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| JP4810062B2 (ja) | 2001-12-17 | 2011-11-09 | 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 | アデノ随伴ウイルス（ａａｖ）血清型８の配列 |
| US20070015238A1 (en) | 2002-06-05 | 2007-01-18 | Snyder Richard O | Production of pseudotyped recombinant AAV virions |
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| EP1777906A1 (en) | 2005-06-09 | 2007-04-25 | Matsushita Electric Industrial Co., Ltd. | Amplitude error compensating apparatus and orthogonality error compensating apparatus |
| 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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| 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 |
| WO2011020118A1 (en) | 2009-08-14 | 2011-02-17 | Theramind Research, Llc | Methods and compositions for treatment of tuberous sclerosis complex |
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| EP2553106A2 (en) | 2010-03-29 | 2013-02-06 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| JP2014519848A (ja) * | 2011-06-20 | 2014-08-21 | ジェネンテック， インコーポレイテッド | Ｐｃｓｋ９結合ポリペプチドと使用方法 |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| EP3795581A3 (en) | 2011-08-24 | 2021-06-09 | The Board of Trustees of the Leland Stanford Junior University | New avv capsid proteins for nucleic acid transfer |
| ES2773111T3 (es) | 2011-09-16 | 2020-07-09 | Regeneron Pharma | Inhibidor de la proproteína convertasa subtilisina/kexina 9 (PCSK9) para su uso en la reducción de los niveles de lipoproteína (a) |
| CN104428009A (zh) * | 2012-02-07 | 2015-03-18 | 全球生物疗法美国有限公司 | 核酸输送的区室化方法及其组合物和应用 |
| BR112014020325A2 (pt) | 2012-02-17 | 2017-08-08 | Childrens Hospital Philadelphia | composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos |
| EP2929017A4 (en) * | 2012-12-05 | 2016-09-28 | Sangamo Biosciences Inc | METHOD AND COMPOSITIONS FOR REGULATING METABOLISM DISEASES |
| DK2954051T3 (da) | 2013-02-08 | 2019-07-08 | Univ Pennsylvania | Modificeret kapsid til genoverførsel til behandling af nethinden |
| 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 |
| US9254332B2 (en) | 2013-03-15 | 2016-02-09 | Arecor Limited | Stable aqueous formulations of adenovirus vectors |
| 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 |
| 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 |
| KR20160067219A (ko) | 2013-10-03 | 2016-06-13 | 모더나 세라퓨틱스, 인코포레이티드 | 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드 |
| 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 |
| IL259850B2 (en) | 2015-12-11 | 2023-11-01 | Univ 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 |
| CA3051011A1 (en) | 2017-02-20 | 2018-08-23 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
-
2015
- 2015-04-24 US US15/306,419 patent/US11555059B2/en active Active
- 2015-04-24 EP EP15721097.2A patent/EP3134431B1/en active Active
- 2015-04-24 WO PCT/US2015/027572 patent/WO2015164778A1/en not_active Ceased
- 2015-04-24 JP JP2016564200A patent/JP6741590B2/ja active Active
- 2015-04-24 ES ES15721097T patent/ES2876409T3/es active Active
- 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 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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