JP2019504003A5 - - Google Patents
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- 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
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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) | IL303850A (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 |
|---|---|---|---|---|
| ES2876409T3 (es) | 2014-04-25 | 2021-11-12 | Univ Pennsylvania | Variantes del RLBD y su uso en composiciones para reducir los niveles de colesterol |
| IL303850A (en) | 2015-12-11 | 2023-08-01 | Univ Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| KR102695908B1 (ko) | 2016-07-21 | 2024-08-14 | 스파크 테라퓨틱스, 인코포레이티드 | 재조합 아데노-관련 바이러스(rAAV) 벡터를 고수율로 생산하기 위한 확장 가능한 고회수 방법 및 이에 의해 생산된 재조합 아데노-관련 바이러스(rAAV) 벡터 |
| 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 |
| US11554147B2 (en) * | 2017-02-20 | 2023-01-17 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| KR20200104864A (ko) | 2017-11-30 | 2020-09-04 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 뮤코다당류증 iiib형에 대한 유전자 요법 |
| US20220040332A1 (en) * | 2018-12-20 | 2022-02-10 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating familial hypercholesterolemia |
| SG11202107614PA (en) | 2019-01-18 | 2021-08-30 | Astrazeneca Ab | Pcsk9 inhibitors and methods of use thereof |
| AU2020209216A1 (en) | 2019-01-18 | 2021-08-26 | Astrazeneca Ab | PCSK9 inhibitors and methods of use thereof |
| MX2022006427A (es) * | 2019-11-28 | 2022-09-07 | Regenxbio Inc | "construcciones para terapia génica con microdistrofina y uso de las mismas. |
| WO2021222653A1 (en) * | 2020-04-29 | 2021-11-04 | Saliogen Therapeutics, Inc. | Compositions and methods for treatment of familial hypercholesterolemia and elevated low-density lipoprotein cholesterol |
| 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 |
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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 |
| 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 |
| WO1998046728A1 (en) | 1997-04-14 | 1998-10-22 | 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 |
| ES2340230T3 (es) | 1998-11-10 | 2010-05-31 | University Of North Carolina At Chapel Hill | Vectores viricos y sus procedimientos de preparacion y administracion. |
| PT1916258E (pt) | 1999-08-09 | 2014-07-29 | Genzyme Corp | Aumento da expressão de uma sequência nucleotídica heteróloga de cadeia simples a partir de vectores virais recombinantes por concepção da sequência de maneira que esta forme pares de bases intracadeia |
| 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 |
| NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| DK2359869T3 (en) * | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| 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 |
| CN1856576B (zh) | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| WO2006110689A2 (en) | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| EP1777906A1 (en) | 2005-06-09 | 2007-04-25 | Matsushita Electric Industrial Co., Ltd. | Amplitude error compensating apparatus and orthogonality error compensating apparatus |
| EP1986737A1 (en) | 2006-02-24 | 2008-11-05 | 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 |
| BRPI1007155A2 (pt) * | 2009-01-29 | 2017-05-30 | Univ Of California San Francisco | métodos para tratar um distúrbio neurológico cortical, e para dispensar um agente terapêutico para o córtex em um primata |
| US20120252877A1 (en) * | 2009-08-14 | 2012-10-04 | Theramind Research, Llc | Methods and compositions for treatment of tuberous sclerosis complex |
| DK2529020T3 (en) * | 2010-01-28 | 2018-08-06 | Childrens Hospital Philadelphia | SCALABLE PREPARATION PLATF FOR CLEANING VIRAL VECTORS AND CLEANED VIRAL VECTORS FOR USE IN GENTHERAPY |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US20130023033A1 (en) | 2010-03-29 | 2013-01-24 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| EP2721063A4 (en) | 2011-06-20 | 2015-01-14 | Hoffmann La Roche | PCSK9 BINDING POLYPEPTIDES AND METHOD OF USE THEREOF |
| 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 |
| ES2992345T3 (es) | 2011-09-16 | 2024-12-11 | Regeneron Pharma | Métodos para reducir los niveles de lipoproteína(a) mediante la administración de un inhibidor de la proproteína convertasa subtilisina kexina-9 (PCSK9) |
| AU2013221212B2 (en) * | 2012-02-17 | 2018-08-09 | The Children's Hospital Of Philadelphia | AAV vector compositions and methods for gene transfer to cells, organs and tissues |
| US9567376B2 (en) | 2013-02-08 | 2017-02-14 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
| 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 |
| SI2984166T1 (sl) * | 2013-03-15 | 2020-09-30 | The Trustees Of The University Of Pennsylvania | Sestavki za zdravljenje MPSI |
| US8980864B2 (en) * | 2013-03-15 | 2015-03-17 | Moderna Therapeutics, Inc. | Compositions and methods of altering cholesterol levels |
| 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 |
| 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 |
| ES2876409T3 (es) * | 2014-04-25 | 2021-11-12 | Univ Pennsylvania | Variantes del RLBD y su uso en composiciones para reducir los niveles de colesterol |
| PE20170261A1 (es) * | 2014-05-13 | 2017-04-12 | Univ Pennsylvania | Composiciones que comprenden virus adenoasociado (aav) que expresa constructos de anticuerpos duales y sus usos |
| WO2017100676A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| IL303850A (en) | 2015-12-11 | 2023-08-01 | Univ 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 |
-
2016
- 2016-12-09 IL IL303850A patent/IL303850A/en unknown
- 2016-12-09 CN CN202211286244.1A patent/CN115957350A/zh active Pending
- 2016-12-09 BR BR112018011687A patent/BR112018011687A2/pt not_active IP Right Cessation
- 2016-12-09 MA MA043570A patent/MA43570A/fr unknown
- 2016-12-09 MX MX2018007080A patent/MX2018007080A/es unknown
- 2016-12-09 EP EP16825936.4A patent/EP3400304B1/en active Active
- 2016-12-09 ES ES16825936T patent/ES2921450T3/es active Active
- 2016-12-09 US US16/060,409 patent/US10889832B2/en active Active
- 2016-12-09 WO PCT/US2016/065984 patent/WO2017100682A1/en not_active Ceased
- 2016-12-09 KR KR1020187019657A patent/KR20180113990A/ko not_active Ceased
- 2016-12-09 CA CA3008142A patent/CA3008142A1/en active Pending
- 2016-12-09 CN CN201680081580.9A patent/CN109121421B/zh active Active
- 2016-12-09 AU AU2016366560A patent/AU2016366560A1/en not_active Abandoned
- 2016-12-09 IL IL259850A patent/IL259850B2/en unknown
- 2016-12-09 JP JP2018530566A patent/JP7328760B2/ja active Active
- 2016-12-09 EP EP22165782.8A patent/EP4085934A1/en not_active Withdrawn
-
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
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