JP2018527941A5 - - Google Patents
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- Publication number
- JP2018527941A5 JP2018527941A5 JP2018515539A JP2018515539A JP2018527941A5 JP 2018527941 A5 JP2018527941 A5 JP 2018527941A5 JP 2018515539 A JP2018515539 A JP 2018515539A JP 2018515539 A JP2018515539 A JP 2018515539A JP 2018527941 A5 JP2018527941 A5 JP 2018527941A5
- Authority
- JP
- Japan
- Prior art keywords
- hfh
- scr
- variant
- scr1
- vector
- 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
Links
- 239000013598 vector Substances 0.000 claims 25
- 230000000295 complement effect Effects 0.000 claims 9
- 239000013607 AAV vector Substances 0.000 claims 7
- 108090000623 proteins and genes Proteins 0.000 claims 7
- 210000000234 capsid Anatomy 0.000 claims 6
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 4
- 208000035475 disorder Diseases 0.000 claims 4
- 230000036470 plasma concentration Effects 0.000 claims 4
- 241000702421 Dependoparvovirus Species 0.000 claims 3
- 206010064930 age-related macular degeneration Diseases 0.000 claims 3
- 125000003275 alpha amino acid group Chemical group 0.000 claims 3
- 150000001413 amino acids Chemical class 0.000 claims 3
- 208000002780 macular degeneration Diseases 0.000 claims 3
- 239000008194 pharmaceutical composition Substances 0.000 claims 3
- 102000004169 proteins and genes Human genes 0.000 claims 3
- 208000035913 Atypical hemolytic uremic syndrome Diseases 0.000 claims 2
- 102000016550 Complement Factor H Human genes 0.000 claims 2
- 108010053085 Complement Factor H Proteins 0.000 claims 2
- 208000000733 Paroxysmal Hemoglobinuria Diseases 0.000 claims 2
- 102100036050 Phosphatidylinositol N-acetylglucosaminyltransferase subunit A Human genes 0.000 claims 2
- 210000003494 hepatocyte Anatomy 0.000 claims 2
- 208000017169 kidney disease Diseases 0.000 claims 2
- 230000001404 mediated effect Effects 0.000 claims 2
- 201000003045 paroxysmal nocturnal hemoglobinuria Diseases 0.000 claims 2
- 230000009711 regulatory function Effects 0.000 claims 2
- 102100034088 40S ribosomal protein S4, X isoform Human genes 0.000 claims 1
- NEEVCWPRIZJJRJ-LWRDCAMISA-N 5-(benzylideneamino)-6-[(e)-benzylideneamino]-2-sulfanylidene-1h-pyrimidin-4-one Chemical compound C=1C=CC=CC=1C=NC=1C(=O)NC(=S)NC=1\N=C\C1=CC=CC=C1 NEEVCWPRIZJJRJ-LWRDCAMISA-N 0.000 claims 1
- 208000009304 Acute Kidney Injury Diseases 0.000 claims 1
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 1
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 208000035143 Bacterial infection Diseases 0.000 claims 1
- 208000016323 C3 glomerulonephritis Diseases 0.000 claims 1
- 108091026890 Coding region Proteins 0.000 claims 1
- 206010051920 Glomerulonephropathy Diseases 0.000 claims 1
- 101000732165 Homo sapiens 40S ribosomal protein S4, X isoform Proteins 0.000 claims 1
- 101000737574 Homo sapiens Complement factor H Proteins 0.000 claims 1
- 208000032382 Ischaemic stroke Diseases 0.000 claims 1
- 241000713666 Lentivirus Species 0.000 claims 1
- 208000004451 Membranoproliferative Glomerulonephritis Diseases 0.000 claims 1
- 208000033626 Renal failure acute Diseases 0.000 claims 1
- 206010043561 Thrombocytopenic purpura Diseases 0.000 claims 1
- 201000007023 Thrombotic Thrombocytopenic Purpura Diseases 0.000 claims 1
- 241000700618 Vaccinia virus Species 0.000 claims 1
- 201000011040 acute kidney failure Diseases 0.000 claims 1
- 208000012998 acute renal failure Diseases 0.000 claims 1
- 208000022362 bacterial infectious disease Diseases 0.000 claims 1
- 244000052616 bacterial pathogen Species 0.000 claims 1
- 208000022401 dense deposit disease Diseases 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 241001493065 dsRNA viruses Species 0.000 claims 1
- 230000013595 glycosylation Effects 0.000 claims 1
- 238000006206 glycosylation reaction Methods 0.000 claims 1
- 208000007475 hemolytic anemia Diseases 0.000 claims 1
- 102000045512 human CFH Human genes 0.000 claims 1
- 238000007918 intramuscular administration Methods 0.000 claims 1
- 238000001990 intravenous administration Methods 0.000 claims 1
- 239000002502 liposome Substances 0.000 claims 1
- 206010062198 microangiopathy Diseases 0.000 claims 1
- 239000002539 nanocarrier Substances 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 201000000980 schizophrenia Diseases 0.000 claims 1
- 238000007920 subcutaneous administration Methods 0.000 claims 1
- 208000024891 symptom Diseases 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
Priority Applications (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| 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 |
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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021128847A Division 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018527941A JP2018527941A (ja) | 2018-09-27 |
| JP2018527941A5 true JP2018527941A5 (enExample) | 2019-11-07 |
| JP7261583B2 JP7261583B2 (ja) | 2023-04-20 |
Family
ID=58387476
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018515539A Active JP7261583B2 (ja) | 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 |
| JP2021128847A Active 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 Pending 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 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 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021128847A Active 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 Pending 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 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 |
Country Status (11)
| Country | Link |
|---|---|
| US (4) | US10988519B2 (enExample) |
| EP (1) | EP3359663A4 (enExample) |
| JP (4) | JP7261583B2 (enExample) |
| KR (1) | KR102773865B1 (enExample) |
| CN (2) | CN115976105B (enExample) |
| AU (3) | AU2016326627B2 (enExample) |
| CA (1) | CA2999299A1 (enExample) |
| HK (1) | HK1251482A1 (enExample) |
| IL (2) | IL258024B2 (enExample) |
| RU (1) | RU2727411C2 (enExample) |
| WO (1) | WO2017053732A2 (enExample) |
Families Citing this family (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3359663A4 (en) * | 2015-09-24 | 2019-08-14 | The Trustees of The University of Pennsylvania | COMPOSITION AND METHOD FOR THE TREATMENT OF A COMPLEMENT-MEDIATED DISEASE |
| 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 |
| CA3121528A1 (en) | 2018-12-12 | 2020-06-18 | Flagship Pioneering Innovations V, Inc. | Anellosomes and methods of use |
| US20230048858A1 (en) * | 2018-12-12 | 2023-02-16 | 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 |
| AU2020272875A1 (en) * | 2019-04-10 | 2021-10-14 | University Of Massachusetts | Factor h vectors and uses thereof |
| JP2022553309A (ja) * | 2019-10-22 | 2022-12-22 | アプライド ジェネティック テクノロジーズ コーポレイション | 加齢黄斑変性ならびに他の眼疾患および障害の治療のためのアデノ随伴ウイルス（ａａｖ）ベクター |
| EP4048319A1 (en) * | 2019-10-23 | 2022-08-31 | Gemini Therapeutics Sub, Inc. | Methods for treating patients having cfh mutations with cfh-encoding vectors |
| WO2021257480A2 (en) | 2020-06-14 | 2021-12-23 | Catalyst Biosciences, Inc. | Complement factor i-related compositions and methods |
| GB202010009D0 (en) * | 2020-06-30 | 2020-08-12 | Syncona Investment Man Ltd | Vector |
| MX2023008826A (es) | 2021-02-01 | 2023-09-15 | Regenxbio Inc | Terapia génica para lipofuscinosis neuronal ceroidea. |
| KR20240112733A (ko) | 2023-01-11 | 2024-07-19 | 포항공과대학교 산학협력단 | 조현병의 예방 또는 치료용 나노바디 |
| JP2026522196A (ja) * | 2023-05-19 | 2026-07-07 | 合肥星眸生物科技有限公司 | 補体第二経路関連疾患を治療するためのａａｖベクター |
Family Cites Families (48)
| 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 |
| 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 |
| EP1007637B1 (en) | 1997-04-14 | 2004-06-30 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant aav product |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| EP1135468B1 (en) | 1998-11-10 | 2010-01-06 | University Of North Carolina At Chapel Hill | Virus vectors and methods of making and administering the same |
| US6596535B1 (en) | 1999-08-09 | 2003-07-22 | Targeted Genetics Corporation | Metabolically activated recombinant viral vectors and methods for the preparation and use |
| AU6814901A (en) | 2000-06-01 | 2001-12-11 | Univ North Carolina | Methods and compounds for controlled release of recombinant parvovirus vectors |
| CN103589692B (zh) | 2001-11-13 | 2018-07-24 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| 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 |
| EP1668143B1 (en) | 2003-09-30 | 2013-03-20 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| EP1560025A3 (en) * | 2003-10-03 | 2011-09-07 | F. Hoffmann-La Roche AG | Specific markers for diabetes |
| US7745389B2 (en) | 2005-02-14 | 2010-06-29 | University Of Iowa Research Foundation | Methods for treatment of age-related macular degeneration |
| DE23193606T1 (de) | 2005-04-07 | 2024-02-15 | The Trustees Of The University Of Pennsylvania | Verfahren zur erhöhung der funktion eines aav-vektors |
| JP4495210B2 (ja) | 2005-06-09 | 2010-06-30 | パナソニック株式会社 | 振幅誤差補償装置及び直交度誤差補償装置 |
| DK2500030T4 (en) | 2005-11-04 | 2018-10-29 | Genentech Inc | Use of complement pathway inhibitors for the treatment of eye 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 |
| JP5250548B2 (ja) | 2006-06-21 | 2013-07-31 | エムユーエスシー ファウンデーション フォー リサーチ ディベロップメント | 疾患の治療のための補体ｈ因子の標的化 |
| 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 |
| AU2011222883B2 (en) * | 2010-03-05 | 2016-05-26 | Omeros Corporation | Chimeric inhibitor molecules of complement activation |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | 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 |
| BR112012033119A2 (pt) * | 2010-06-22 | 2016-10-25 | Univ Colorado Regents | anticorpos para o fragamento c3d do componente do complemento 3. |
| WO2013142362A1 (en) * | 2012-03-19 | 2013-09-26 | The Trustees Of The University Of Pennsylvania | Regulator of complement activation and uses thereof |
| WO2014124282A1 (en) | 2013-02-08 | 2014-08-14 | 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 |
| FR3015484A1 (fr) * | 2013-12-20 | 2015-06-26 | Lab Francais Du Fractionnement | Proteines recombinantes possedant une activite de facteur h |
| KR101591823B1 (ko) * | 2013-12-27 | 2016-02-04 | 재단법인 목암생명공학연구소 | 증가된 유전자 발현능을 갖는 발현벡터 |
| WO2017024198A1 (en) | 2015-08-06 | 2017-02-09 | The Trustees Of The University Of Pennsylvania | Glp-1 and use thereof in compositions for treating metabolic diseases |
| EP3359663A4 (en) * | 2015-09-24 | 2019-08-14 | The Trustees of The University of Pennsylvania | COMPOSITION AND METHOD FOR THE TREATMENT OF A COMPLEMENT-MEDIATED DISEASE |
| GB201519086D0 (en) * | 2015-10-28 | 2015-12-09 | Syncona Partners Llp | Gene Therapy |
| LT3394089T (lt) * | 2015-12-23 | 2021-11-25 | eleva GmbH | Polipeptidai, skirti komplemento aktyvacijos inhibicijai |
| 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 |
| US20240254177A1 (en) * | 2018-08-22 | 2024-08-01 | Alexion Pharmaceuticals, Inc. | COMPLEMENT FACTOR H AND Fc BINDING DOMAIN FUSION PROTEINS |
| WO2020157274A1 (en) * | 2019-01-31 | 2020-08-06 | Albert-Ludwigs-Universität Freiburg | Methods for optimizing heterologous gene expression |
| AU2020272875A1 (en) | 2019-04-10 | 2021-10-14 | 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 |
| KR20260003703A (ko) * | 2023-04-07 | 2026-01-07 | 4디 몰레큘러 테라퓨틱스 아이엔씨. | 보체 장애의 치료를 위한 aav 변이체 |
-
2016
- 2016-09-23 EP EP16849709.7A patent/EP3359663A4/en active Pending
- 2016-09-23 CN CN202211039079.XA patent/CN115976105B/zh active Active
- 2016-09-23 US US15/762,721 patent/US10988519B2/en active Active
- 2016-09-23 KR KR1020187011034A patent/KR102773865B1/ko active Active
- 2016-09-23 CA CA2999299A patent/CA2999299A1/en active Pending
- 2016-09-23 CN CN201680064219.5A patent/CN108291216B/zh active Active
- 2016-09-23 RU RU2018114907A patent/RU2727411C2/ru active
- 2016-09-23 HK HK18111116.6A patent/HK1251482A1/zh unknown
- 2016-09-23 JP JP2018515539A patent/JP7261583B2/ja active Active
- 2016-09-23 IL IL258024A patent/IL258024B2/en unknown
- 2016-09-23 IL IL296929A patent/IL296929B2/en unknown
- 2016-09-23 AU AU2016326627A patent/AU2016326627B2/en active Active
- 2016-09-23 WO PCT/US2016/053347 patent/WO2017053732A2/en not_active Ceased
-
2021
- 2021-01-22 US US17/156,023 patent/US20210171592A1/en not_active Abandoned
- 2021-08-05 JP JP2021128847A patent/JP7324253B2/ja active Active
-
2023
- 2023-03-14 AU AU2023201582A patent/AU2023201582B2/en active Active
- 2023-07-28 JP JP2023123167A patent/JP2023139256A/ja active Pending
- 2023-10-26 US US18/494,883 patent/US12180254B1/en active Active
-
2024
- 2024-10-23 US US18/924,426 patent/US20250051408A1/en active Pending
- 2024-12-02 JP JP2024209719A patent/JP2025029082A/ja active Pending
-
2026
- 2026-02-13 AU AU2026201080A patent/AU2026201080A1/en active Pending
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