JP7261583B2 - 補体媒介性疾患を処置するための組成物及び方法 - Google Patents

補体媒介性疾患を処置するための組成物及び方法 Download PDF

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JP7261583B2
JP7261583B2 JP2018515539A JP2018515539A JP7261583B2 JP 7261583 B2 JP7261583 B2 JP 7261583B2 JP 2018515539 A JP2018515539 A JP 2018515539A JP 2018515539 A JP2018515539 A JP 2018515539A JP 7261583 B2 JP7261583 B2 JP 7261583B2
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宋文超
ガリパリ,ダモダル
三輪隆史
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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
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JP2018515539A 2015-09-24 2016-09-23 補体媒介性疾患を処置するための組成物及び方法 Active JP7261583B2 (ja)

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JP2021128847A JP7324253B2 (ja) 2015-09-24 2021-08-05 補体媒介性疾患を処置するための組成物及び方法
JP2023123167A JP2023139256A (ja) 2015-09-24 2023-07-28 補体媒介性疾患を処置するための組成物及び方法
JP2024209719A JP2025029082A (ja) 2015-09-24 2024-12-02 補体媒介性疾患を処置するための組成物及び方法

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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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JP2024209719A Pending JP2025029082A (ja) 2015-09-24 2024-12-02 補体媒介性疾患を処置するための組成物及び方法

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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023139256A (ja) * 2015-09-24 2023-10-03 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

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201706808D0 (en) * 2017-04-28 2017-06-14 Univ Newcastle Modified complement proteins and uses thereof
AU2018351491A1 (en) * 2017-10-20 2020-05-07 Gemini Therapeutics Inc. Compositions and methods for treating age-related macular degeneration
GB201800620D0 (en) * 2018-01-15 2018-02-28 Univ Manchester C3b Binding Polypeptide
WO2020123773A2 (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
MX2022004770A (es) * 2019-10-22 2022-10-07 Applied Genetic Tech Corporation Vectores de virus adeno-asociados (aav) para el tratamiento de la degeneracion macular relacionada con la edad y otras enfermedades y trastornos oculares.
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
JP2024505257A (ja) 2021-02-01 2024-02-05 レジェンクスバイオ インコーポレーテッド 神経セロイドリポフスチン症の遺伝子治療
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 (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013515474A (ja) 2009-12-24 2013-05-09 ザ ユニヴァーシティ コート オブ ザ ユニヴァーシティ オブ エディンバラ 組換え体h因子ならびにそのバリアントおよびコンジュゲート
US20150110766A1 (en) 2012-03-19 2015-04-23 University Ulm 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 (41)

* 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
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
AU728220B2 (en) 1997-04-14 2001-01-04 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant AAV product
EP1080218A1 (en) 1998-05-27 2001-03-07 University of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient
WO2000028004A1 (en) 1998-11-10 2000-05-18 The University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
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
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
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
CN102199626B (zh) 2003-09-30 2015-06-24 宾夕法尼亚大学托管会 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
WO2006088950A2 (en) * 2005-02-14 2006-08-24 University Of Iowa Research Foundation Methods and reagents for treatment and diagnosis of age-related macular degeneration
EP2383346B1 (en) 2005-04-07 2014-10-08 The Trustees of the University of Pennsylvania Modified AAVrh.64 capsids, compositions containing same and uses thereof
EP1777906A1 (en) 2005-06-09 2007-04-25 Matsushita Electric Industrial Co., Ltd. Amplitude error compensating apparatus and orthogonality error compensating apparatus
NZ567483A (en) * 2005-11-04 2012-04-27 Genentech Inc Use of complement pathway inhibitors to treat ocular diseases
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
BRPI0712987A2 (pt) * 2006-06-21 2012-04-10 Musc Found For Res Dev tratamento de doenças direcionado ao fator h do complemento
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
EP3815708A1 (en) * 2010-03-05 2021-05-05 Omeros Corporation Chimeric inhibitor molecules of complement activation
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
CA2803588A1 (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
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
US12221615B2 (en) 2015-08-06 2025-02-11 The Trustees Of The University Of Pennsylvania GLP-1 and use thereof in compositions for treating metabolic diseases
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
BR112019000171A2 (pt) * 2016-07-08 2019-10-01 Univ Pennsylvania métodos e composições para tratamento de distúrbios e doenças envolvendo 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 (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013515474A (ja) 2009-12-24 2013-05-09 ザ ユニヴァーシティ コート オブ ザ ユニヴァーシティ オブ エディンバラ 組換え体h因子ならびにそのバリアントおよびコンジュゲート
US20150110766A1 (en) 2012-03-19 2015-04-23 University Ulm 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 (2)

* Cited by examiner, † Cited by third party
Title
Drug Discovery System (2007) Vol.22-6, pp.643-650
坂田洋一,血友病遺伝子治療の最新情報と展望,Hemophilia Topics (2007)Vol.13, pp.1-4, バイエル薬品株式会社 [online], [検索日 2020.7.14],URL,<https://pharma-navi.bayer.jp/hemophilia/static/pdf/disease_info/hemophilia_topics/vol13.pdf>

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023139256A (ja) * 2015-09-24 2023-10-03 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

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