JP2021529821A - イムノグロブリンを除去又は減少させるための遺伝子治療ベクターの形質導入を増加または増強するための組成物および方法 - Google Patents

イムノグロブリンを除去又は減少させるための遺伝子治療ベクターの形質導入を増加または増強するための組成物および方法 Download PDF

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JP2021529821A
JP2021529821A JP2021502435A JP2021502435A JP2021529821A JP 2021529821 A JP2021529821 A JP 2021529821A JP 2021502435 A JP2021502435 A JP 2021502435A JP 2021502435 A JP2021502435 A JP 2021502435A JP 2021529821 A JP2021529821 A JP 2021529821A
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disease
protein
vector
subject
viral vector
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JP2021529821A5 (https=
JPWO2020016318A5 (https=
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ラクロワ−デスマゼス，セバスチャン
ミンゴッツィ，フェデリコ
ディミトロフ，ヨルダン
レボルニュ，クリスティアン
アーマー，ショーン
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Sorbonne Universite
Universite Paris Cite
Spark Therapeutics Inc
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Sorbonne Universite
Universite de Paris
Spark Therapeutics Inc
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Application filed by Sorbonne Universite, Universite de Paris, Spark Therapeutics Inc filed Critical Sorbonne Universite
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JP2021502435A 2018-07-17 2019-07-17 イムノグロブリンを除去又は減少させるための遺伝子治療ベクターの形質導入を増加または増強するための組成物および方法 Pending JP2021529821A (ja)

Priority Applications (1)

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JP2024051529A JP2024075765A (ja) 2018-07-17 2024-03-27 イムノグロブリンを除去又は減少させるための遺伝子治療ベクターの形質導入を増加または増強するための組成物および方法

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Application Number Priority Date Filing Date Title
EP18305971 2018-07-17
EP18305971.6 2018-07-17
US201862768731P 2018-11-16 2018-11-16
US62/768,731 2018-11-16
PCT/EP2019/069280 WO2020016318A1 (en) 2018-07-17 2019-07-17 Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins

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JP2021529821A true JP2021529821A (ja) 2021-11-04
JP2021529821A5 JP2021529821A5 (https=) 2022-05-13
JPWO2020016318A5 JPWO2020016318A5 (https=) 2022-05-13

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JP2024051529A Pending JP2024075765A (ja) 2018-07-17 2024-03-27 イムノグロブリンを除去又は減少させるための遺伝子治療ベクターの形質導入を増加または増強するための組成物および方法

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US (3) US20210228738A1 (https=)
EP (2) EP4154904A1 (https=)
JP (2) JP2021529821A (https=)
KR (1) KR20210042308A (https=)
CN (2) CN112689515A (https=)
AU (2) AU2019306121B2 (https=)
BR (1) BR112021000807A8 (https=)
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UA (1) UA130604C2 (https=)
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Cited By (1)

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JP2022550930A (ja) * 2019-12-06 2022-12-06 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 抗ｖｅｇｆタンパク質組成物及びその製造方法

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4219547A3 (en) 2008-09-15 2023-10-18 uniQure biopharma B.V. Factor ix polypeptide mutant, its uses and a method for its production
GB201502305D0 (en) * 2015-02-12 2015-04-01 Hansa Medical Ab Protein
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
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
US20210363191A1 (en) 2018-04-03 2021-11-25 Stridebio, Inc. Antibody-evading virus vectors
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
US20200165608A1 (en) * 2018-11-23 2020-05-28 Florida State University Research Foundation, Inc. Inhibition of vascular endothelial cell-mediated phagocytic processes for treatment of demyelinating conditions
AU2020215682B2 (en) * 2019-01-28 2025-12-18 Duke University Compositions and methods for evading humoral immunity
CN114732822B (zh) * 2019-03-13 2023-06-30 中国人民解放军军事科学院军事医学研究院 氨基葡萄糖及其衍生物作为抗病毒药物的应用
BR112021018776A2 (pt) 2019-03-21 2021-11-30 Stridebio Inc Vetores de vírus adenoassociado recombinante
AR118928A1 (es) * 2019-05-14 2021-11-10 Biomarin Pharm Inc Métodos de redosificación de vectores de terapia génica
US12544437B2 (en) 2019-10-04 2026-02-10 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant AAV
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
KR20220133969A (ko) 2020-01-28 2022-10-05 프리라인 테라퓨틱스 리미티드 바이러스 벡터에 대한 중화 항체 역가를 결정하기 위한 개선된 분석법
MX2022009982A (es) 2020-02-14 2022-09-12 Ultragenyx Pharmaceutical Inc Terapia genica para el tratamiento de transtorno por deficiencia de cdkl5.
JP2023525034A (ja) 2020-05-08 2023-06-14 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Ｖｅｇｆトラップおよびミニトラップならびに眼障害およびがんの治療方法
CN111621506B (zh) * 2020-05-09 2021-07-30 华中农业大学 牛支原体分泌蛋白MbovP0145及其应用
GB202007431D0 (en) * 2020-05-19 2020-07-01 Hansa Biopharma AB Cysteine protease
EP4162951A4 (en) * 2020-06-05 2024-07-10 Shanghai Bao Pharmaceuticals Co., Ltd. PHARMACEUTICAL COMPOSITION OF ENZYMES AND VIRUSES AND ASSOCIATED APPLICATION
JP2023532219A (ja) * 2020-06-18 2023-07-27 上海宝済薬業有限公司 免疫グロブリン分解酵素ＩｄｅＥの突然変異体
RU2020122141A (ru) * 2020-07-03 2022-01-04 Максим Петрович Никитин Способ и композиция на основе генотерапевтического агента и антитела, связывающегося с клетками крови
WO2022026632A2 (en) * 2020-07-29 2022-02-03 University Of Florida Research Foundation, Incorporated Improved aav-mediated x-linked retinoschisis therapies
EP4200408A1 (en) 2020-08-19 2023-06-28 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of rett syndrome
AU2021350732A1 (en) * 2020-09-28 2023-05-11 Scout Bio, Inc. Composition and uses thereof
AU2021358957A1 (en) 2020-10-07 2023-06-08 AskBio Inc. Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy. disorders including limb girdle 21
EP4304662A4 (en) * 2021-03-08 2025-06-18 The Regents of the University of California Lentivirus protection via fc overexpression
WO2022192193A1 (en) * 2021-03-09 2022-09-15 The Board Of Regents Of The University Of Texas System Methods and compositions for mutagenesis screening in mammalian cells
JP2024515626A (ja) 2021-04-16 2024-04-10 アスクレピオス バイオファーマシューティカル， インコーポレイテッド 血液脳関門を横断し低減された液性応答を惹起する合理的ポリプロイドａａｖビリオン
WO2023280175A1 (en) * 2021-07-06 2023-01-12 National Institute Of Biological Sciences, Beijing Methods for treating complex i deficiencies or cancers by modulating gro3p biosynthesis
WO2023019168A1 (en) 2021-08-11 2023-02-16 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating a muscular dystrophy
EP4349365A1 (en) * 2022-10-07 2024-04-10 Hansa Biopharma AB Co-treatment for gene therapy
CN115558021B (zh) * 2022-12-01 2023-03-17 山东大学 一种转录因子、重组细胞及其在制备肿瘤治疗药物的应用
CN116425873B (zh) * 2023-06-02 2023-09-05 苏州百道医疗科技有限公司 一种抗ck6重组兔单克隆抗体及其应用
CN117126270B (zh) * 2023-10-25 2024-02-13 首都儿科研究所 一种2型人博卡病毒型别特异性抗体及其应用
CN118217443B (zh) * 2024-01-15 2026-04-03 国科温州研究院(温州生物材料与工程研究所) 一种人发角蛋白止血海绵及其制备方法与应用
WO2025155923A1 (en) 2024-01-17 2025-07-24 The Broad Institute, Inc. Aav capsid modifications that enable improved cns-wide gene delivery through interactions with the transferrin receptor
WO2025217174A1 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Aav capsid modifications that enable improved cns-wide gene delivery through interactions with carbonic anhydrase iv
WO2025217163A2 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Novel aav capsids binding to human cd59
WO2025256977A1 (en) 2024-06-13 2025-12-18 Bayer Aktiengesellschaft Novel igm and igg cleaving enzymes
WO2025256978A1 (en) 2024-06-13 2025-12-18 Bayer Aktiengesellschaft Variants of igm and igg cleaving enzymes
WO2026041758A1 (en) * 2024-08-22 2026-02-26 Genethon Modified aav capsid with enhanced transduction efficiency
GB202414654D0 (en) 2024-10-04 2024-11-20 Hansa Biopharma AB Regimen for vector-based therapies

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008542418A (ja) * 2005-06-09 2008-11-27 ハンサ メディカル アクチボラゲット 自己免疫疾患及び移植片拒絶反応を治療するためのＩｄｅＳプロテイナーゼ（化膿連鎖球菌由来）の使用
JP2016525347A (ja) * 2013-07-12 2016-08-25 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ 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
JP2018510622A (ja) * 2015-02-12 2018-04-19 ハンサ メディカル アクチエボラグ システインプロテアーゼ

Family Cites Families (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US414A (en) 1837-09-28 Moetise-latch foe
US941A (en) 1838-09-22 Machine for sawing shingles and staves
US5173A (en) 1847-06-26 Machinery for
US5139A (en) 1847-06-05 Cajsfkles
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
WO1990005183A1 (en) 1988-10-31 1990-05-17 Immunex Corporation Interleukin-4 receptors
AU651596B2 (en) 1990-06-05 1994-07-28 Immunex Corporation Type II interleukin-1 receptors
WO1992001070A1 (en) 1990-07-09 1992-01-23 The United States Of America, As Represented By The Secretary, U.S. Department Of Commerce High efficiency packaging of mutant adeno-associated virus using amber suppressions
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
JP3534749B2 (ja) 1991-08-20 2004-06-07 アメリカ合衆国 アデノウイルスが介在する胃腸管への遺伝子の輸送
AU7676894A (en) 1993-08-27 1995-03-21 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Convection-enhanced drug delivery
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US6074849A (en) 1995-07-19 2000-06-13 Genetics Institute, Inc. Polynucleotides encoding human CTLA-8 related proteins
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US6569645B2 (en) 1999-05-14 2003-05-27 Genentech, Inc. IL-17 homologous polypeptides and therapeutic uses thereof
US6579520B2 (en) 1998-05-15 2003-06-17 Genentech, Inc. IL-17 related mammalian cytokine polypeptides (IL-17E)
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
NZ592917A (en) 2003-09-15 2012-12-21 Protiva Biotherapeutics Inc Stable polyethyleneglycol (PEG) dialkyloxypropyl (DAA) lipid conjugates
US7745651B2 (en) 2004-06-07 2010-06-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
EP3192788A1 (en) 2006-10-03 2017-07-19 Arbutus Biopharma Corporation Lipid containing formulations
US7649243B2 (en) 2006-11-06 2010-01-19 International Business Machines Corporation Semiconductor structures incorporating multiple crystallographic planes and methods for fabrication thereof
GB0624874D0 (en) 2006-12-13 2007-01-24 Hansa Medical Ab Treatment
EP2219587A4 (en) 2007-11-14 2012-11-21 Univ California STEROL-MODIFIED AMPHIPHILE LIPIDES
CN104119242B (zh) 2008-10-09 2017-07-07 泰米拉制药公司 改善的氨基脂质和递送核酸的方法
HUE037082T2 (hu) 2008-11-10 2018-08-28 Arbutus Biopharma Corp Új lipidek és készítmények terápiás hatóanyagok szállítására
WO2010054384A1 (en) 2008-11-10 2010-05-14 Alnylam Pharmaceuticals, Inc. Lipids and compositions for the delivery of therapeutics
US9018187B2 (en) 2009-07-01 2015-04-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
WO2011141704A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
WO2011141705A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
US8466122B2 (en) 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
GB201103780D0 (en) 2011-03-04 2011-04-20 Hansa Medical Ab Treatment for ige-mediated disease
ES2857773T5 (es) 2011-08-24 2024-06-04 Univ Leland Stanford Junior Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos
US9126966B2 (en) 2011-08-31 2015-09-08 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use thereof
GB201115841D0 (en) 2011-09-13 2011-10-26 Genovis Ab Protein and method
GB201201314D0 (en) * 2012-01-26 2012-03-07 Isis Innovation Composition
SG11201405157PA (en) 2012-02-24 2014-10-30 Protiva Biotherapeutics Inc Trialkyl cationic lipids and methods of use thereof
AU2013249202B2 (en) 2012-04-18 2018-08-09 The Children's Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using AAV capsid variants
TWI775096B (zh) 2012-05-15 2022-08-21 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
EP3605090A1 (en) 2012-05-23 2020-02-05 F. Hoffmann-La Roche AG Selection method for therapeutic agents
KR20240090694A (ko) 2013-07-22 2024-06-21 더 칠드런스 호스피탈 오브 필라델피아 변종 ａａｖ 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도
GB201413240D0 (en) 2014-07-25 2014-09-10 Hansa Medical Ab Method
GB201502306D0 (en) 2015-02-12 2015-04-01 Hansa Medical Ab Protein
AU2017207736A1 (en) * 2016-01-11 2018-07-12 Rubius Therapeutics, Inc. Compositions and methods related to multimodal therapeutic cell systems for immune indications
ES2877754T3 (es) 2016-02-04 2021-11-17 Genovis Ab Nuevas proteasas estreptocócicas
WO2018093868A1 (en) 2016-11-16 2018-05-24 University Of Florida Research Foundation, Inc. Immunoglobulin proteases, compositions, and uses thereof
WO2018109207A1 (en) 2016-12-16 2018-06-21 Uniqure Ip B.V. Immunoadsorption
WO2018158397A1 (en) 2017-03-02 2018-09-07 Genethon Method for removing anti-aav antibodies from a blood-derived composition
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

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2008542418A (ja) * 2005-06-09 2008-11-27 ハンサ メディカル アクチボラゲット 自己免疫疾患及び移植片拒絶反応を治療するためのＩｄｅＳプロテイナーゼ（化膿連鎖球菌由来）の使用
JP2016525347A (ja) * 2013-07-12 2016-08-25 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ 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
JP2018510622A (ja) * 2015-02-12 2018-04-19 ハンサ メディカル アクチエボラグ システインプロテアーゼ

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022550930A (ja) * 2019-12-06 2022-12-06 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 抗ｖｅｇｆタンパク質組成物及びその製造方法

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