RU2727411C2 - Композиция и способ для лечения заболевания, опосредованного комплементом - Google Patents

Композиция и способ для лечения заболевания, опосредованного комплементом Download PDF

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RU2727411C2
RU2727411C2 RU2018114907A RU2018114907A RU2727411C2 RU 2727411 C2 RU2727411 C2 RU 2727411C2 RU 2018114907 A RU2018114907 A RU 2018114907A RU 2018114907 A RU2018114907 A RU 2018114907A RU 2727411 C2 RU2727411 C2 RU 2727411C2
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scr1
complement
hfh
vector
variant
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RU2018114907A
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Вэньчао СУН
Дамодар ГУЛЛИПАЛЛИ
Такаси МИВА
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Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания
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    • C07K14/472—Complement proteins, e.g. anaphylatoxin, C3a, C5a
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

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RU2018114907A 2015-09-24 2016-09-23 Композиция и способ для лечения заболевания, опосредованного комплементом RU2727411C2 (ru)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562232008P 2015-09-24 2015-09-24
US62/232,008 2015-09-24
PCT/US2016/053347 WO2017053732A2 (en) 2015-09-24 2016-09-23 Composition and method for treating complement-mediated disease

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RU2018114907A RU2018114907A (ru) 2019-10-24
RU2018114907A3 RU2018114907A3 (https=) 2020-02-26
RU2727411C2 true RU2727411C2 (ru) 2020-07-21

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US (4) US10988519B2 (https=)
EP (1) EP3359663A4 (https=)
JP (4) JP7261583B2 (https=)
KR (1) KR102773865B1 (https=)
CN (2) CN108291216B (https=)
AU (3) AU2016326627B2 (https=)
CA (1) CA2999299A1 (https=)
HK (1) HK1251482A1 (https=)
IL (2) IL258024B2 (https=)
RU (1) RU2727411C2 (https=)
WO (1) WO2017053732A2 (https=)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108291216B (zh) * 2015-09-24 2022-09-16 宾夕法尼亚州大学信托人 用于治疗补体介导的疾病的组合物和方法
GB201706808D0 (en) * 2017-04-28 2017-06-14 Univ Newcastle Modified complement proteins and uses thereof
CN111788311A (zh) * 2017-10-20 2020-10-16 双子治疗公司 用于治疗年龄相关性黄斑变性的组合物和方法
GB201800620D0 (en) 2018-01-15 2018-02-28 Univ Manchester C3b Binding Polypeptide
KR20210131309A (ko) * 2018-12-12 2021-11-02 플래그쉽 파이어니어링 이노베이션스 브이, 인크. 분비형 치료 양식을 운반하기 위한 아넬로좀
GB201821082D0 (en) * 2018-12-21 2019-02-06 Gyroscope Therapeutics Ltd Combination of complement factors i and h, and vector encoding thereof
KR20210149803A (ko) * 2019-04-10 2021-12-09 유니버시티 오브 매사추세츠 인자 h 벡터 및 그의 용도
CA3158517A1 (en) * 2019-10-22 2021-04-29 Applied Genetic Technologies Corporation Adeno-associated virus (aav)vectors for the treatment of age-related macular degeneration and other ocular diseases and disorders
EP4048319A1 (en) * 2019-10-23 2022-08-31 Gemini Therapeutics Sub, Inc. Methods for treating patients having cfh mutations with cfh-encoding vectors
AU2021293174A1 (en) 2020-06-14 2023-01-19 Vertex Pharmaceuticals Incorporated Complement Factor I-related compositions and methods
GB202010009D0 (en) * 2020-06-30 2020-08-12 Syncona Investment Man Ltd Vector
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
KR20240112733A (ko) 2023-01-11 2024-07-19 포항공과대학교 산학협력단 조현병의 예방 또는 치료용 나노바디
EP4714980A1 (en) * 2023-05-19 2026-03-25 Starrygene Therapeutics Co., Ltd. Aav vector for treating alternative complement pathway-related diseases

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2417099C2 (ru) * 2005-11-04 2011-04-27 Дженентек, Инк. Применение ингибиторов пути комплемента для лечения глазных болезней
WO2011107591A1 (en) * 2010-03-05 2011-09-09 Rigshospitalet Chimeric inhibitor molecules of complement activation
WO2011163412A1 (en) * 2010-06-22 2011-12-29 The Regents Of The University Of Colorado, A Body Corporate Antibodies to the c3d fragment of complement component 3
WO2013142362A1 (en) * 2012-03-19 2013-09-26 The Trustees Of The University Of Pennsylvania Regulator of complement activation and uses thereof
WO2015092335A2 (fr) * 2013-12-20 2015-06-25 Laboratoire Francais Du Fractionnement Et Des Biotechnologies Proteines recombinantes possedant une activite de facteur h

Family Cites Families (39)

* 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
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
US5679546A (en) 1993-09-24 1997-10-21 Cytomed, Inc. Chimeric proteins which block complement activation
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
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
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
EP2357254A1 (en) 2005-02-14 2011-08-17 University of Iowa Research Foundation Methods and reagents for treatment and diagnosis of age-related macular degeneration
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
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
WO2007149567A2 (en) 2006-06-21 2007-12-27 Musc Foundation For Research Development Targeting complement factor h for treatment of diseases
GB0701213D0 (en) * 2007-01-23 2007-02-28 Univ Cardiff Factor H polymorphisms in the diagnosis and therapy of inflammatory diseases such as age-related macular degeneration
KR101010352B1 (ko) 2008-05-30 2011-01-25 삼성중공업 주식회사 전력 제어 장치 및 방법
GB0922659D0 (en) * 2009-12-24 2010-02-10 Univ Edinburgh Factor H
US20130023033A1 (en) 2010-03-29 2013-01-24 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
US9567376B2 (en) 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
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
ES2523016B1 (es) * 2013-05-20 2015-09-09 3P Biopharmaceuticals Vectores alfavirales y líneas celulares para la producción de proteínas recombinantes
KR101591823B1 (ko) * 2013-12-27 2016-02-04 재단법인 목암생명공학연구소 증가된 유전자 발현능을 갖는 발현벡터
EP3332012A4 (en) 2015-08-06 2019-01-16 The Trustees of The University of Pennsylvania GLP-1 AND USE THEREOF IN COMPOSITIONS FOR THE TREATMENT OF METABOLISM DISEASES
CN108291216B (zh) * 2015-09-24 2022-09-16 宾夕法尼亚州大学信托人 用于治疗补体介导的疾病的组合物和方法
GB201519086D0 (en) 2015-10-28 2015-12-09 Syncona Partners Llp Gene Therapy
US11197936B2 (en) * 2016-07-08 2021-12-14 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of disorders and diseases involving RDH12
KR20210149803A (ko) 2019-04-10 2021-12-09 유니버시티 오브 매사추세츠 인자 h 벡터 및 그의 용도
CN117947041A (zh) * 2022-10-28 2024-04-30 上海瑞宏迪医药有限公司 编码重组补体因子h多肽的核酸分子及用途
CN121311596A (zh) * 2023-04-07 2026-01-09 4D分子治疗有限公司 用于治疗补体病症的aav变体

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2417099C2 (ru) * 2005-11-04 2011-04-27 Дженентек, Инк. Применение ингибиторов пути комплемента для лечения глазных болезней
WO2011107591A1 (en) * 2010-03-05 2011-09-09 Rigshospitalet Chimeric inhibitor molecules of complement activation
WO2011163412A1 (en) * 2010-06-22 2011-12-29 The Regents Of The University Of Colorado, A Body Corporate Antibodies to the c3d fragment of complement component 3
WO2013142362A1 (en) * 2012-03-19 2013-09-26 The Trustees Of The University Of Pennsylvania Regulator of complement activation and uses thereof
WO2015092335A2 (fr) * 2013-12-20 2015-06-25 Laboratoire Francais Du Fractionnement Et Des Biotechnologies Proteines recombinantes possedant une activite de facteur h

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
NICHOLS E.-M.ET AL., An extended mini-complement factor H molecule ameliorates experimental C3 glomerulopathy, KIDNEY INTERNATIONAL, 29.07.2015, v. 88, n. 6, p.1314 - 1322. *

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KR102773865B1 (ko) 2025-03-04
JP2025029082A (ja) 2025-03-05
US20210171592A1 (en) 2021-06-10
JP2023139256A (ja) 2023-10-03
JP7324253B2 (ja) 2023-08-09
JP7261583B2 (ja) 2023-04-20
EP3359663A2 (en) 2018-08-15
US10988519B2 (en) 2021-04-27
RU2018114907A (ru) 2019-10-24
AU2023201582A1 (en) 2023-04-13
JP2022000017A (ja) 2022-01-04
IL258024B2 (en) 2023-03-01
IL258024B (en) 2022-11-01
KR20180055872A (ko) 2018-05-25
AU2016326627A1 (en) 2018-04-05
CN115976105A (zh) 2023-04-18
CA2999299A1 (en) 2017-03-30
US20190177381A1 (en) 2019-06-13
AU2026201080A1 (en) 2026-03-05
CN108291216B (zh) 2022-09-16
CN108291216A (zh) 2018-07-17
WO2017053732A3 (en) 2017-05-11
US12180254B1 (en) 2024-12-31
AU2023201582B2 (en) 2025-11-13
IL296929B1 (en) 2025-03-01
RU2018114907A3 (https=) 2020-02-26
WO2017053732A2 (en) 2017-03-30
EP3359663A4 (en) 2019-08-14
IL296929B2 (en) 2025-07-01
AU2016326627B2 (en) 2022-12-15
HK1251482A1 (zh) 2019-02-01
IL258024A (en) 2018-05-31
IL296929A (en) 2022-12-01
JP2018527941A (ja) 2018-09-27

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