JP2019162109A5 - - Google Patents
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- Publication number
- JP2019162109A5 JP2019162109A5 JP2019072287A JP2019072287A JP2019162109A5 JP 2019162109 A5 JP2019162109 A5 JP 2019162109A5 JP 2019072287 A JP2019072287 A JP 2019072287A JP 2019072287 A JP2019072287 A JP 2019072287A JP 2019162109 A5 JP2019162109 A5 JP 2019162109A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- reporter transgene
- cells
- expression
- antibody
- 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
Links
- 238000000034 method Methods 0.000 claims 41
- 241000702421 Dependoparvovirus Species 0.000 claims 32
- 108700019146 Transgenes Proteins 0.000 claims 19
- 210000004027 cell Anatomy 0.000 claims 13
- 239000002245 particle Substances 0.000 claims 9
- 239000012472 biological sample Substances 0.000 claims 8
- 208000015181 infectious disease Diseases 0.000 claims 8
- 230000002458 infectious effect Effects 0.000 claims 8
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 6
- 239000013598 vector Substances 0.000 claims 6
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 5
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 5
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 5
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 5
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 5
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 5
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 5
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 5
- 241000649045 Adeno-associated virus 10 Species 0.000 claims 5
- 241000649046 Adeno-associated virus 11 Species 0.000 claims 5
- 241000649047 Adeno-associated virus 12 Species 0.000 claims 5
- 108090000623 proteins and genes Proteins 0.000 claims 5
- 108060001084 Luciferase Proteins 0.000 claims 4
- 210000004962 mammalian cell Anatomy 0.000 claims 4
- 230000001105 regulatory effect Effects 0.000 claims 4
- 239000013607 AAV vector Substances 0.000 claims 3
- 102000004169 proteins and genes Human genes 0.000 claims 3
- 108010043121 Green Fluorescent Proteins Proteins 0.000 claims 2
- 102000004144 Green Fluorescent Proteins Human genes 0.000 claims 2
- 241000254158 Lampyridae Species 0.000 claims 2
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 230000000694 effects Effects 0.000 claims 2
- 239000005090 green fluorescent protein Substances 0.000 claims 2
- 239000000203 mixture Substances 0.000 claims 2
- 150000007523 nucleic acids Chemical group 0.000 claims 2
- 239000013641 positive control Substances 0.000 claims 2
- 230000001124 posttranscriptional effect Effects 0.000 claims 2
- 108010054624 red fluorescent protein Proteins 0.000 claims 2
- 102000002260 Alkaline Phosphatase Human genes 0.000 claims 1
- 108020004774 Alkaline Phosphatase Proteins 0.000 claims 1
- 108090000565 Capsid Proteins Proteins 0.000 claims 1
- 102100023321 Ceruloplasmin Human genes 0.000 claims 1
- 108090000790 Enzymes Proteins 0.000 claims 1
- 102000004190 Enzymes Human genes 0.000 claims 1
- 108700042658 GAP-43 Proteins 0.000 claims 1
- 208000028782 Hereditary disease Diseases 0.000 claims 1
- 101000600434 Homo sapiens Putative uncharacterized protein encoded by MIR7-3HG Proteins 0.000 claims 1
- 208000026350 Inborn Genetic disease Diseases 0.000 claims 1
- 241000282560 Macaca mulatta Species 0.000 claims 1
- 241000124008 Mammalia Species 0.000 claims 1
- 208000024556 Mendelian disease Diseases 0.000 claims 1
- 241000288906 Primates Species 0.000 claims 1
- 102100037401 Putative uncharacterized protein encoded by MIR7-3HG Human genes 0.000 claims 1
- 241000242739 Renilla Species 0.000 claims 1
- 241000242743 Renilla reniformis Species 0.000 claims 1
- 108090000992 Transferases Proteins 0.000 claims 1
- 230000005856 abnormality Effects 0.000 claims 1
- 102000005936 beta-Galactosidase Human genes 0.000 claims 1
- 108010005774 beta-Galactosidase Proteins 0.000 claims 1
- 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
- 230000007547 defect Effects 0.000 claims 1
- 239000012895 dilution Substances 0.000 claims 1
- 238000010790 dilution Methods 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 238000003780 insertion Methods 0.000 claims 1
- 230000037431 insertion Effects 0.000 claims 1
- 230000010354 integration Effects 0.000 claims 1
- 238000004020 luminiscence type Methods 0.000 claims 1
- 230000008488 polyadenylation Effects 0.000 claims 1
- 230000010076 replication Effects 0.000 claims 1
- 210000002966 serum Anatomy 0.000 claims 1
- 238000011144 upstream manufacturing Methods 0.000 claims 1
- 108700026220 vif Genes Proteins 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021195840A 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 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 |
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361845841P | 2013-07-12 | 2013-07-12 | |
| US61/845,841 | 2013-07-12 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016525827A Division 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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021195840A Division 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 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019162109A JP2019162109A (ja) | 2019-09-26 |
| JP2019162109A5 true JP2019162109A5 (https=) | 2020-09-17 |
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 Before (1)
| 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 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
Family Cites Families (23)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| EP1005376B1 (en) * | 1997-03-14 | 2010-11-03 | The Children's Hospital of Philadelphia | Compositions for use in gene therapy for treatment of hemophilia |
| 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 |
| DK1035133T3 (da) * | 1999-02-17 | 2005-05-02 | Pfizer Prod Inc | Fusionsproteiner, der omfatter bærere, som kan inducere et dobbelt immunrespons |
| 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 |
| US20050233457A1 (en) * | 2002-04-15 | 2005-10-20 | Andreas Block | Recombining viral vectors for the tetracycline-regulated expression of genes |
| EP1369491A1 (en) | 2002-06-05 | 2003-12-10 | Aladar A. Szalay | Light emitting microorganisms and cells for diagnosis and therapy of diseases associated with wounded or inflamed tissue |
| 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
- 2014-07-11 CN CN202310061410.6A patent/CN116004721A/zh active Pending
- 2014-07-11 EP EP14823769.6A patent/EP3019620A4/en not_active Withdrawn
- 2014-07-11 CN CN201480039270.1A patent/CN105518145A/zh active Pending
- 2014-07-11 KR KR1020167003572A patent/KR102445330B1/ko active Active
-
2015
- 2015-12-31 IL IL243438A patent/IL243438B/en active IP Right Grant
-
2016
- 2016-01-11 MX MX2021000675A patent/MX2021000675A/es unknown
-
2019
- 2019-04-04 JP JP2019072287A patent/JP2019162109A/ja active Pending
-
2021
- 2021-12-02 JP JP2021195840A patent/JP2022037027A/ja active Pending
-
2022
- 2022-06-30 US US17/855,571 patent/US20220334126A1/en not_active Abandoned
-
2023
- 2023-12-01 JP JP2023204220A patent/JP2024026253A/ja active Pending
-
2025
- 2025-10-24 US US19/368,502 patent/US20260043810A1/en active Pending
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