CN115957350A - 用于治疗家族性高胆固醇血症的基因疗法 - Google Patents

用于治疗家族性高胆固醇血症的基因疗法 Download PDF

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CN115957350A
CN115957350A CN202211286244.1A CN202211286244A CN115957350A CN 115957350 A CN115957350 A CN 115957350A CN 202211286244 A CN202211286244 A CN 202211286244A CN 115957350 A CN115957350 A CN 115957350A
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aav8
vector
raav
hldlr
tbg
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J.M.威尔逊
D.J.拉德
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University of Pennsylvania Penn
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CN202211286244.1A 2015-12-11 2016-12-09 用于治疗家族性高胆固醇血症的基因疗法 Pending CN115957350A (zh)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201562266383P 2015-12-11 2015-12-11
US62/266383 2015-12-11
US201562269440P 2015-12-18 2015-12-18
US62/269440 2015-12-18
CN201680081580.9A CN109121421B (zh) 2015-12-11 2016-12-09 用于治疗家族性高胆固醇血症的基因疗法
PCT/US2016/065984 WO2017100682A1 (en) 2015-12-11 2016-12-09 Gene therapy for treating familial hypercholesterolemia

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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=)
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Families Citing this family (13)

* Cited by examiner, † Cited by third party
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

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2014152540A1 (en) * 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
CN105026554A (zh) * 2013-03-15 2015-11-04 宾夕法尼亚大学托管会 用于治疗mps1的组合物和方法
CN106470736A (zh) * 2014-05-13 2017-03-01 宾夕法尼亚州大学信托人 包括表达双抗体构建体的aav的组合物及其用途

Family Cites Families (40)

* Cited by examiner, † Cited by third party
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
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
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
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
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

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2014152540A1 (en) * 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
CN105026554A (zh) * 2013-03-15 2015-11-04 宾夕法尼亚大学托管会 用于治疗mps1的组合物和方法
CN105308183A (zh) * 2013-03-15 2016-02-03 现代治疗公司 改变胆固醇水平的组合物和方法
CN106470736A (zh) * 2014-05-13 2017-03-01 宾夕法尼亚州大学信托人 包括表达双抗体构建体的aav的组合物及其用途

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
CORINNA LEBHERZ等: "Gene therapy with novel adeno-associated virus vectors substantially diminishes atherosclerosis in a murine model of familial hypercholesterolemia", J GENE MED., 30 June 2004 (2004-06-30), pages 663 - 672 *
FASS, D.等: "Homo sapiens low density lipoprotein receptor (familial hypercholesterolemia) (LDLR), mRNA", GENBANK DATABASE, 11 June 2002 (2002-06-11), pages 000527 *
SADIK H. KASSIM等: "Adeno-Associated Virus Serotype 8 Gene Therapy Leads to Significant Lowering of Plasma Cholesterol Levels in Humanized Mouse Models of Homozygous and Heterozygous Familial Hypercholesterolemia", HUMAN GENE THERAPY, 14 November 2012 (2012-11-14), pages 20 *

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IL259850B1 (en) 2023-07-01
WO2017100682A1 (en) 2017-06-15
CO2018007165A2 (es) 2018-07-19
AU2023201657A1 (en) 2023-06-15
EP3400304A1 (en) 2018-11-14
IL303850A (en) 2023-08-01
JP7328760B2 (ja) 2023-08-17
US20210285013A1 (en) 2021-09-16
IL259850B2 (en) 2023-11-01
CN109121421A (zh) 2019-01-01
CA3008142A1 (en) 2017-06-15
IL259850A (en) 2018-07-31
KR20180113990A (ko) 2018-10-17
MA43570A (fr) 2018-11-14
JP2022050575A (ja) 2022-03-30
EP3400304B1 (en) 2022-04-06
BR112018011687A2 (pt) 2018-12-04
JP2019504003A (ja) 2019-02-14
US20190002917A1 (en) 2019-01-03
ES2921450T3 (es) 2022-08-25
US10889832B2 (en) 2021-01-12
MX2018007080A (es) 2018-11-12
AU2016366560A1 (en) 2018-06-28
CN109121421B (zh) 2022-11-01
EP4085934A1 (en) 2022-11-09

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