JP2016525347A5 - - Google Patents
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- JP2016525347A5 JP2016525347A5 JP2016525827A JP2016525827A JP2016525347A5 JP 2016525347 A5 JP2016525347 A5 JP 2016525347A5 JP 2016525827 A JP2016525827 A JP 2016525827A JP 2016525827 A JP2016525827 A JP 2016525827A JP 2016525347 A5 JP2016525347 A5 JP 2016525347A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- reporter transgene
- biological sample
- cells
- disease
- 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.)
- Pending
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- 238000000034 method Methods 0.000 claims 71
- 241000702421 Dependoparvovirus Species 0.000 claims 50
- 108700019146 Transgenes Proteins 0.000 claims 25
- 210000004027 cell Anatomy 0.000 claims 19
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- 208000015181 infectious disease Diseases 0.000 claims 14
- 230000002458 infectious effect Effects 0.000 claims 14
- 239000002245 particle Substances 0.000 claims 14
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 9
- 239000013607 AAV vector Substances 0.000 claims 8
- 239000013598 vector Substances 0.000 claims 8
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 7
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 7
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 7
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 7
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 7
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 7
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 7
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 7
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 7
- 241000649047 Adeno-associated virus 12 Species 0.000 claims 7
- 239000000203 mixture Substances 0.000 claims 6
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- RYGMFSIKBFXOCR-UHFFFAOYSA-N Copper Chemical compound [Cu] RYGMFSIKBFXOCR-UHFFFAOYSA-N 0.000 claims 1
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- 101000729271 Homo sapiens Retinoid isomerohydrolase Proteins 0.000 claims 1
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- 206010056886 Mucopolysaccharidosis I Diseases 0.000 claims 1
- 208000031888 Mycoses Diseases 0.000 claims 1
- 206010028980 Neoplasm Diseases 0.000 claims 1
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 102000010292 Peptide Elongation Factor 1 Human genes 0.000 claims 1
- 108010077524 Peptide Elongation Factor 1 Proteins 0.000 claims 1
- 102100037401 Putative uncharacterized protein encoded by MIR7-3HG Human genes 0.000 claims 1
- 108010052090 Renilla Luciferases Proteins 0.000 claims 1
- 102100031176 Retinoid isomerohydrolase Human genes 0.000 claims 1
- 208000002903 Thalassemia Diseases 0.000 claims 1
- 108090000992 Transferases Proteins 0.000 claims 1
- 206010067584 Type 1 diabetes mellitus Diseases 0.000 claims 1
- 208000018839 Wilson disease Diseases 0.000 claims 1
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- 201000009628 adenosine deaminase deficiency Diseases 0.000 claims 1
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- 208000036556 autosomal recessive T cell-negative B cell-negative NK cell-negative due to adenosine deaminase deficiency severe combined immunodeficiency Diseases 0.000 claims 1
- 230000001580 bacterial effect Effects 0.000 claims 1
- 102000005936 beta-Galactosidase Human genes 0.000 claims 1
- 108010005774 beta-Galactosidase Proteins 0.000 claims 1
- 230000015572 biosynthetic process Effects 0.000 claims 1
- 210000004556 brain Anatomy 0.000 claims 1
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- WIIZWVCIJKGZOK-RKDXNWHRSA-N chloramphenicol Chemical compound ClC(Cl)C(=O)N[C@H](CO)[C@H](O)C1=CC=C([N+]([O-])=O)C=C1 WIIZWVCIJKGZOK-RKDXNWHRSA-N 0.000 claims 1
- 229960005091 chloramphenicol Drugs 0.000 claims 1
- 229910052802 copper Inorganic materials 0.000 claims 1
- 239000010949 copper Substances 0.000 claims 1
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- 238000000338 in vitro Methods 0.000 claims 1
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- 108010079892 phosphoglycerol kinase Proteins 0.000 claims 1
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- 108700026220 vif Genes Proteins 0.000 claims 1
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Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361845841P | 2013-07-12 | 2013-07-12 | |
| US61/845,841 | 2013-07-12 | ||
| PCT/US2014/046428 WO2015006743A1 (en) | 2013-07-12 | 2014-07-11 | Aav vector and assay for anti-aav (adeno-associated virus) neutralizing antibodies |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019072287A Division JP2019162109A (ja) | 2013-07-12 | 2019-04-04 | Ａａｖベクター、及び抗ａａｖ（アデノ関連ウイルス）中和抗体についてのアッセイ |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2016525347A JP2016525347A (ja) | 2016-08-25 |
| JP2016525347A5 true JP2016525347A5 (https=) | 2017-07-27 |
Family
ID=52280652
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016525827A Pending JP2016525347A (ja) | 2013-07-12 | 2014-07-11 | Ａａｖベクター、及び抗ａａｖ（アデノ関連ウイルス）中和抗体についてのアッセイ |
| JP2019072287A Pending JP2019162109A (ja) | 2013-07-12 | 2019-04-04 | Ａａｖベクター、及び抗ａａｖ（アデノ関連ウイルス）中和抗体についてのアッセイ |
| JP2021195840A Pending JP2022037027A (ja) | 2013-07-12 | 2021-12-02 | Ａａｖベクター、及び抗ａａｖ（アデノ関連ウイルス）中和抗体についてのアッセイ |
| JP2023204220A Pending JP2024026253A (ja) | 2013-07-12 | 2023-12-01 | Ａａｖベクター、及び抗ａａｖ（アデノ関連ウイルス）中和抗体についてのアッセイ |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019072287A Pending JP2019162109A (ja) | 2013-07-12 | 2019-04-04 | Ａａｖベクター、及び抗ａａｖ（アデノ関連ウイルス）中和抗体についてのアッセイ |
| JP2021195840A Pending JP2022037027A (ja) | 2013-07-12 | 2021-12-02 | Ａａｖベクター、及び抗ａａｖ（アデノ関連ウイルス）中和抗体についてのアッセイ |
| JP2023204220A Pending JP2024026253A (ja) | 2013-07-12 | 2023-12-01 | Ａａｖベクター、及び抗ａａｖ（アデノ関連ウイルス）中和抗体についてのアッセイ |
Country Status (12)
| Country | Link |
|---|---|
| US (3) | US11408899B2 (https=) |
| EP (2) | EP4039813A1 (https=) |
| JP (4) | JP2016525347A (https=) |
| KR (1) | KR102445330B1 (https=) |
| CN (2) | CN116004721A (https=) |
| AU (1) | AU2014287005C1 (https=) |
| CA (1) | CA2918038C (https=) |
| HK (1) | HK1222673A1 (https=) |
| IL (1) | IL243438B (https=) |
| MX (2) | MX2016000345A (https=) |
| RU (1) | RU2705249C2 (https=) |
| WO (1) | WO2015006743A1 (https=) |
Families Citing this family (38)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN107635584B (zh) * | 2015-04-09 | 2022-06-17 | 康奈尔大学 | 基因治疗以预防对过敏原的反应 |
| MX390848B (es) | 2015-06-23 | 2025-03-21 | Childrens Hospital Philadelphia | Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos. |
| SG11201802408RA (en) | 2015-11-05 | 2018-05-30 | Bamboo Therapeutics Inc | Modified friedreich ataxia genes and vectors for gene therapy |
| EP3384035A4 (en) * | 2015-12-02 | 2019-08-07 | Voyager Therapeutics, Inc. | ASSAYS FOR THE DETECTION OF AAV-NEUTRALIZING ANTIBODIES |
| EP3645021A4 (en) | 2017-06-30 | 2021-04-21 | Intima Bioscience, Inc. | ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY |
| US20210228738A1 (en) * | 2017-07-17 | 2021-07-29 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins |
| CN107828821A (zh) * | 2017-11-15 | 2018-03-23 | 四川大学 | 一种腺相关病毒感染敏感的细胞系的构建方法及腺相关病毒中和抗体含量测定的检测方法 |
| CA3086046C (en) | 2017-12-29 | 2023-02-21 | Helixmith Co., Ltd. | Adeno-associated virus (aav) vector having hybrid hgf gene introduced thereto |
| US20190256867A1 (en) | 2018-02-01 | 2019-08-22 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring pah gene function and methods of use thereof |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| JP7244547B2 (ja) | 2018-02-19 | 2023-03-22 | ホモロジー・メディシンズ・インコーポレイテッド | Ｆ８遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法 |
| MA51353B1 (fr) | 2018-04-05 | 2022-09-30 | Univ Sorbonne | Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit |
| CA3100946A1 (en) * | 2018-05-22 | 2019-11-28 | The Brigham And Women's Hospital, Inc. | Gene therapy for alzheimer's disease |
| CN110846392A (zh) * | 2018-08-20 | 2020-02-28 | 武汉纽福斯生物科技有限公司 | 一种重组腺相关病毒或含其的试剂盒及其应用 |
| CA3119349A1 (en) * | 2018-11-16 | 2020-05-22 | Spark Therapeutics, Inc. | In vitro assay for detecting enhancers and inhibitors of adeno associated virus (aav) vector transduction and/or detecting or quantitating anti-aav binding antibodies |
| EP3918308A4 (en) * | 2019-01-31 | 2023-09-27 | Precision Bioservices, Inc. | METHOD FOR DETECTING AND CHARACTERIZING ANTIVIRAL VECTOR ANTIBODIES |
| KR102917057B1 (ko) | 2019-04-24 | 2026-01-23 | 다카라 바이오 가부시키가이샤 | 뇌에 지향성을 가지는 aav 돌연변이체 |
| JP7686573B6 (ja) | 2019-05-24 | 2025-07-04 | ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッド | アルツハイマー病のための遺伝子治療 |
| BR112022010373A2 (pt) | 2019-11-29 | 2022-08-16 | Paros Bio Inc | Terapia genética para doenças neurodegenerativas |
| TW202140791A (zh) | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
| KR20220133969A (ko) | 2020-01-28 | 2022-10-05 | 프리라인 테라퓨틱스 리미티드 | 바이러스 벡터에 대한 중화 항체 역가를 결정하기 위한 개선된 분석법 |
| WO2021209275A1 (en) * | 2020-04-12 | 2021-10-21 | Novoscope Ip Limited | Particles, dna & rna |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
| EP4421179A3 (en) * | 2020-09-10 | 2024-12-18 | Genethon | Peptide-modified aav capsid |
| CN112143712B (zh) * | 2020-09-30 | 2022-11-01 | 中国科学院深圳先进技术研究院 | 一种重组腺相关病毒及其制备方法和在抗体检测的应用 |
| WO2023114816A1 (en) | 2021-12-14 | 2023-06-22 | Neurogene, Inc. | Recombinant optimized galc constructs and methods for treating galc-associated disorders |
| CN114277090A (zh) * | 2021-12-17 | 2022-04-05 | 宁波熙宁检测技术有限公司 | Aav8中和抗体检测方法和检测试剂盒 |
| GB202201242D0 (en) | 2022-01-31 | 2022-03-16 | Univ Edinburgh | Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders |
| EP4514984A4 (en) * | 2022-04-25 | 2026-04-22 | Beijing Hanmoluojie Tech Co Ltd | 3D MOLECULAR SURFACE FEATURES MAPPING OF AAV CAPSIDS |
| GB202206336D0 (en) | 2022-04-29 | 2022-06-15 | Univ Edinburgh | Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders |
| AU2023267671A1 (en) * | 2022-05-13 | 2024-12-05 | The Children's Hospital Of Philadelphia | Adeno-associated viral vectors for targeting brain microvasculature |
| EP4536862A1 (en) * | 2022-06-09 | 2025-04-16 | Wisconsin Alumni Research Foundation | Generation of next generation recombinant aav gene therapy vectors that adopt 3d conformation |
| CN115047188A (zh) * | 2022-06-10 | 2022-09-13 | 上海鼎新基因科技有限公司 | 一种抗腺相关病毒中和抗体的检测方法 |
| CN117802161A (zh) * | 2022-06-30 | 2024-04-02 | 苏州吉恒基因科技有限公司 | 精准重组腺相关病毒载体及其用途 |
| WO2024098054A1 (en) * | 2022-11-04 | 2024-05-10 | The Children's Hospital Of Philadelphia | Compositions and methods for b cell directed immunotherapies for anti-aav neutralizing alloantibodies |
| CN117285620B (zh) * | 2023-11-27 | 2024-02-13 | 恺佧生物科技(上海)有限公司 | 抗aav9抗体及aav9滴度测定elisa试剂盒 |
| WO2025119252A1 (en) * | 2023-12-06 | 2025-06-12 | Immvira Biopharmaceuticals Co., Limited | Methods for detecting an anti-hsv antibody in a sample |
| WO2026017965A1 (en) | 2024-07-19 | 2026-01-22 | The University Court Of The University Of Edinburgh | Recombinant therapeutic fxn constructs and methods of treating friedreich ataxia and related conditions |
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| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
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| JP4025400B2 (ja) * | 1997-04-16 | 2007-12-19 | 財団法人化学及血清療法研究所 | 新規免疫制御分子及びその製造方法 |
| WO1999061601A2 (en) * | 1998-05-28 | 1999-12-02 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Aav5 vector and uses thereof |
| ATE362542T1 (de) * | 1998-11-05 | 2007-06-15 | Univ Pennsylvania | Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten |
| US6759237B1 (en) | 1998-11-05 | 2004-07-06 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
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| CA2385823A1 (en) * | 1999-10-01 | 2001-04-12 | Haifeng Chen | Production of recombinant aav using adenovirus comprising aav rep/cap genes |
| US7241447B1 (en) * | 1999-10-07 | 2007-07-10 | University Of Iowa Research Foundation | Adeno-associated virus vectors and uses thereof |
| ATE318923T1 (de) * | 2000-06-01 | 2006-03-15 | Univ North Carolina | Doppelsträngige parvovirus-vektoren |
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| AU2003274397A1 (en) * | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| MXPA04012560A (es) * | 2002-06-13 | 2005-10-19 | Wyeth Corp | Inhibidores de la actividad genica inflamatoria y biosintesis de colesterol. |
| EP2335069A4 (en) * | 2008-09-25 | 2012-08-01 | Univ Duke | METHOD FOR DETECTING ANTIBODIES AND ANTIBODY HIV VIRON COMPLEXES |
| HRP20250897T1 (hr) | 2009-05-02 | 2025-09-26 | Genzyme Corporation | Genska terapija za neurodegenerativne poremećaje |
| KR101464902B1 (ko) * | 2010-08-20 | 2014-11-24 | 나이키 이노베이트 씨.브이. | 골프 클럽 및 골프 클럽 헤드 |
| CN102576029A (zh) | 2010-10-01 | 2012-07-11 | 松下电器产业株式会社 | 从脂质双层膜的外侧向内侧输送钾离子的方法 |
| US8202687B2 (en) | 2010-10-01 | 2012-06-19 | Panasonic Corporation | Method for transporting potassium ions from front side to back side of lipid bilayer membrane |
| BR112013010487B1 (pt) | 2010-11-02 | 2021-02-02 | Promega Corporation | compostos de coelenterazina, kit compreendendo ditos compostos e método para detectar luminescência em uma amostra in vitro |
| EP2675484B1 (en) | 2011-02-14 | 2018-05-30 | The Children's Hospital of Philadelphia | Improved aav8 vector with enhanced functional activity and methods of use thereof |
| CN103014064A (zh) * | 2011-09-22 | 2013-04-03 | 北京五加和分子医学研究所有限公司 | Itr同向排列的aav载体构建及其应用 |
| HK1202434A1 (en) * | 2011-11-22 | 2015-10-02 | The Children's Hospital Of Philadelphia | Virus vectors for highly efficient transgene delivery |
-
2014
- 2014-07-11 HK HK16110663.7A patent/HK1222673A1/zh unknown
- 2014-07-11 EP EP21209775.2A patent/EP4039813A1/en active Pending
- 2014-07-11 AU AU2014287005A patent/AU2014287005C1/en active Active
- 2014-07-11 JP JP2016525827A patent/JP2016525347A/ja active Pending
- 2014-07-11 RU RU2016104614A patent/RU2705249C2/ru not_active Application Discontinuation
- 2014-07-11 WO PCT/US2014/046428 patent/WO2015006743A1/en not_active Ceased
- 2014-07-11 MX MX2016000345A patent/MX2016000345A/es unknown
- 2014-07-11 US US14/904,205 patent/US11408899B2/en active Active
- 2014-07-11 CA CA2918038A patent/CA2918038C/en active Active
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