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) | CN108291216B (enExample) |
| AU (3) | 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)
| 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 |
| WO2019079718A1 (en) * | 2017-10-20 | 2019-04-25 | 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 |
| EP3894568A2 (en) * | 2018-12-12 | 2021-10-20 | 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 |
| CN113924313A (zh) * | 2019-04-10 | 2022-01-11 | 马萨诸塞大学 | 因子h载体及其用途 |
| IL292379A (en) * | 2019-10-22 | 2022-06-01 | Applied Genetic Tech 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 |
| IL299048A (en) | 2020-06-14 | 2023-02-01 | Vertex Pharma | Variants of complement factor - 1 fusion structures and preparations containing them and their uses |
| GB202010009D0 (en) * | 2020-06-30 | 2020-08-12 | Syncona Investment Man Ltd | Vector |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | 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 |
Family Cites Families (48)
| 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 |
| 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 |
| ATE454445T1 (de) | 1998-11-10 | 2010-01-15 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
| 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 |
| CA2410901C (en) | 2000-06-01 | 2012-03-20 | University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| SG10202108118RA (en) | 2001-11-13 | 2021-08-30 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| ES2975413T3 (es) | 2001-12-17 | 2024-07-05 | Univ Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas |
| US20070015238A1 (en) | 2002-06-05 | 2007-01-18 | Snyder Richard O | Production of pseudotyped recombinant AAV virions |
| HUE054805T2 (hu) | 2003-09-30 | 2021-09-28 | Univ Pennsylvania | Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik |
| EP1560025A3 (en) * | 2003-10-03 | 2011-09-07 | F. Hoffmann-La Roche AG | Specific markers for diabetes |
| US8088579B2 (en) | 2005-02-14 | 2012-01-03 | University Of Iowa Research Foundation | Complement factor H for diagnosis of age-related macular degeneration |
| EP3085389A1 (en) | 2005-04-07 | 2016-10-26 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| WO2006132118A1 (ja) | 2005-06-09 | 2006-12-14 | Matsushita Electric Industrial Co., Ltd. | 振幅誤差補償装置及び直交度誤差補償装置 |
| PT2500030E (pt) * | 2005-11-04 | 2015-09-24 | Genentech Inc | Uso de inibidores da via do complemento para tratar doenças oculares |
| WO2007120542A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid library and aav capsid proteins |
| PT2044111E (pt) * | 2006-06-21 | 2014-11-12 | Univ Colorado Regents | Abordagem seletiva do fator h do complemento para o tratamento de doenças |
| 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 |
| US9815876B2 (en) * | 2010-03-05 | 2017-11-14 | Omeros Corporation | Chimeric inhibitor molecules of complement activation |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| KR20130040844A (ko) | 2010-03-29 | 2013-04-24 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 약학적으로 유발된 전이유전자 제거 시스템 |
| MX2012014975A (es) * | 2010-06-22 | 2013-03-12 | Univ Colorado Regents | Anticuerpos al fragmento c3d de componente 3 de complemento. |
| WO2013142362A1 (en) | 2012-03-19 | 2013-09-26 | The Trustees Of The University Of Pennsylvania | Regulator of complement activation and uses thereof |
| ES2741502T3 (es) | 2013-02-08 | 2020-02-11 | Univ Pennsylvania | Cápside de AAV8 modificada para la transferencia génica para terapias de retina |
| WO2015012924A2 (en) | 2013-04-29 | 2015-01-29 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and use 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 |
| 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 |
| 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 |
| AU2016375183B2 (en) * | 2015-12-23 | 2021-01-21 | eleva GmbH | Polypeptides for inhibiting complement activation |
| KR102799528B1 (ko) * | 2016-07-08 | 2025-04-23 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Rdh12가 연루된 장애 및 질환의 치료를 위한 방법 및 조성물 |
| 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 |
| CN113924313A (zh) | 2019-04-10 | 2022-01-11 | 马萨诸塞大学 | 因子h载体及其用途 |
| 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 |
-
2016
- 2016-09-23 EP EP16849709.7A patent/EP3359663A4/en active Pending
- 2016-09-23 US US15/762,721 patent/US10988519B2/en active Active
- 2016-09-23 WO PCT/US2016/053347 patent/WO2017053732A2/en not_active Ceased
- 2016-09-23 JP JP2018515539A patent/JP7261583B2/ja active Active
- 2016-09-23 AU AU2016326627A patent/AU2016326627B2/en active Active
- 2016-09-23 CA CA2999299A patent/CA2999299A1/en active Pending
- 2016-09-23 KR KR1020187011034A patent/KR102773865B1/ko active Active
- 2016-09-23 CN CN201680064219.5A patent/CN108291216B/zh active Active
- 2016-09-23 CN CN202211039079.XA patent/CN115976105A/zh active Pending
- 2016-09-23 HK HK18111116.6A patent/HK1251482A1/zh unknown
- 2016-09-23 IL IL296929A patent/IL296929B2/en unknown
- 2016-09-23 IL IL258024A patent/IL258024B2/en unknown
- 2016-09-23 RU RU2018114907A patent/RU2727411C2/ru active
-
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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