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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Вэньчао СУН
Дамодар ГУЛЛИПАЛЛИ
Такаси МИВА
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Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания
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    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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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 (enExample) 2020-02-26
RU2727411C2 true RU2727411C2 (ru) 2020-07-21

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US (4) US10988519B2 (enExample)
EP (1) EP3359663A4 (enExample)
JP (4) JP7261583B2 (enExample)
KR (1) KR102773865B1 (enExample)
CN (2) CN108291216B (enExample)
AU (2) AU2016326627B2 (enExample)
CA (1) CA2999299A1 (enExample)
HK (1) HK1251482A1 (enExample)
IL (2) IL296929B2 (enExample)
RU (1) RU2727411C2 (enExample)
WO (1) WO2017053732A2 (enExample)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7261583B2 (ja) * 2015-09-24 2023-04-20 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 補体媒介性疾患を処置するための組成物及び方法
GB201706808D0 (en) * 2017-04-28 2017-06-14 Univ Newcastle Modified complement proteins and uses thereof
JP2021500922A (ja) * 2017-10-20 2021-01-14 ジェミニ・セラピューティクス・インコーポレイテッドGemini Therapeutics Inc. 加齢黄斑変性を処置するための組成物および方法
GB201800620D0 (en) * 2018-01-15 2018-02-28 Univ Manchester C3b Binding Polypeptide
CA3119531A1 (en) * 2018-12-12 2020-06-18 Flagship Pioneering Innovations V, Inc. Anellosomes for delivering secreted therapeutic modalities
GB201821082D0 (en) * 2018-12-21 2019-02-06 Gyroscope Therapeutics Ltd Combination of complement factors i and h, and vector encoding thereof
EP3966231A4 (en) * 2019-04-10 2022-12-21 University of Massachusetts Factor h vectors and uses thereof
AU2020371663A1 (en) * 2019-10-22 2022-05-19 Applied Genetic Technologies Corporation Adeno-associated virus (AAV)vectors for the treatment of age-related macular degeneration and other ocular diseases and disorders
WO2021081401A1 (en) * 2019-10-23 2021-04-29 Gemini Therapeutics Inc. Methods for treating patients having cfh mutations with cfh-encoding vectors
EP4165181A2 (en) 2020-06-14 2023-04-19 Vertex Pharmaceuticals Incorporated Complement factor i-related compositions and methods
GB202010009D0 (en) * 2020-06-30 2020-08-12 Syncona Investment Man Ltd Vector
AU2022214429A1 (en) 2021-02-01 2023-09-14 Tern Therapeutics, Llc Gene therapy for neuronal ceroid lipofuscinoses
KR20240112733A (ko) 2023-01-11 2024-07-19 포항공과대학교 산학협력단 조현병의 예방 또는 치료용 나노바디
WO2024240095A1 (zh) * 2023-05-19 2024-11-28 合肥星眸生物科技有限公司 用于治疗补体旁路相关疾病的aav载体

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
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
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
ES2224375T3 (es) 1997-04-14 2005-03-01 Cell Genesys, Inc. Metodos para aumentar la eficacia del producto de aav recombinante.
WO1999061643A1 (en) 1998-05-27 1999-12-02 University Of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixananol gradient
CA2348382C (en) 1998-11-10 2013-09-17 The University Of North Carolina At Chapel Hill Chimeric parvovirus vectors and methods of making and administering the same
EP2369002A1 (en) 1999-08-09 2011-09-28 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 intrastrand base pairs
ES2327609T3 (es) 2000-06-01 2009-11-02 University Of North Carolina At Chapel Hill Procedimientos y compuestos para controlar la liberacion de vectores de parvovirus reconbinantes.
NZ618298A (en) 2001-11-13 2015-04-24 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
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
WO2003104413A2 (en) 2002-06-05 2003-12-18 University Of Florida Production of pseudotyped recombinant aav virions
HUE035567T2 (en) 2003-09-30 2018-05-28 Univ Pennsylvania Adeno-associated virus (AAV) clusters, sequences, vectors and applications containing them
MX2007009565A (es) 2005-02-14 2008-03-10 Univ Iowa Res Found Metodos y reactivos para tratamiento y diagnostico de degeneracion macular relacionada con la edad.
EP1866422B1 (en) 2005-04-07 2016-04-06 The Trustees of The University of Pennsylvania Method of increasing the function of an aav vector
US7456683B2 (en) 2005-06-09 2008-11-25 Panasonic Corporation Amplitude error compensating device and quadrature skew error compensating 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
DK2044111T3 (en) 2006-06-21 2014-11-17 Musc Found For Res Dev OBJECTIVE TO 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
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
DK2954051T3 (da) 2013-02-08 2019-07-08 Univ Pennsylvania Modificeret kapsid til genoverførsel til behandling af nethinden
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
KR102745604B1 (ko) 2015-08-06 2024-12-23 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Glp-1 및 대사성 질환 치료용 조성물에서 이의 용도
JP7261583B2 (ja) * 2015-09-24 2023-04-20 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 補体媒介性疾患を処置するための組成物及び方法
GB201519086D0 (en) * 2015-10-28 2015-12-09 Syncona Partners Llp Gene Therapy
WO2018009814A1 (en) * 2016-07-08 2018-01-11 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of disorders and diseases involving rdh12
EP3966231A4 (en) 2019-04-10 2022-12-21 University of Massachusetts Factor h vectors and uses thereof
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
WO2024211416A2 (en) * 2023-04-07 2024-10-10 4D Molecular Therapeutics Inc. Aav variants for treatment of complement disorders

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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WO2017053732A3 (en) 2017-05-11
US20250051408A1 (en) 2025-02-13
US20190177381A1 (en) 2019-06-13
RU2018114907A (ru) 2019-10-24
JP2025029082A (ja) 2025-03-05
IL296929B2 (en) 2025-07-01
JP7261583B2 (ja) 2023-04-20
AU2023201582B2 (en) 2025-11-13
JP7324253B2 (ja) 2023-08-09
WO2017053732A2 (en) 2017-03-30
AU2023201582A1 (en) 2023-04-13
IL296929B1 (en) 2025-03-01
AU2016326627B2 (en) 2022-12-15
IL258024B2 (en) 2023-03-01
EP3359663A4 (en) 2019-08-14
IL258024A (en) 2018-05-31
JP2018527941A (ja) 2018-09-27
KR102773865B1 (ko) 2025-03-04
RU2018114907A3 (enExample) 2020-02-26
IL258024B (en) 2022-11-01
AU2016326627A1 (en) 2018-04-05
US12180254B1 (en) 2024-12-31
HK1251482A1 (zh) 2019-02-01
CN115976105A (zh) 2023-04-18
JP2023139256A (ja) 2023-10-03
US20210171592A1 (en) 2021-06-10
CA2999299A1 (en) 2017-03-30
US10988519B2 (en) 2021-04-27
JP2022000017A (ja) 2022-01-04
CN108291216A (zh) 2018-07-17
CN108291216B (zh) 2022-09-16
KR20180055872A (ko) 2018-05-25
IL296929A (en) 2022-12-01
EP3359663A2 (en) 2018-08-15

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