JP2023548145A - 眼の適応症に対するベクター化ｔｎｆ－アルファアンタゴニスト - Google Patents

眼の適応症に対するベクター化ｔｎｆ－アルファアンタゴニスト Download PDF

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JP2023548145A
JP2023548145A JP2023526231A JP2023526231A JP2023548145A JP 2023548145 A JP2023548145 A JP 2023548145A JP 2023526231 A JP2023526231 A JP 2023526231A JP 2023526231 A JP2023526231 A JP 2023526231A JP 2023548145 A JP2023548145 A JP 2023548145A
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serotype
aav
tnfα
cells
seq
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JP2023548145A5 (enExample
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ワン，シュー
マクドゥーガルド，デヴィン
ブルーダー，ジョセフ
リウ，イェ
ダノス，オリビエ
チャオ，チャンピング
リー，ウェイ－ファ
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リジェネックスバイオ インコーポレイテッド
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    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/1793—Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/715—Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons
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    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/24—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
    • C07K16/241—Tumor Necrosis Factors
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/62—DNA sequences coding for fusion proteins
    • C12N15/625—DNA sequences coding for fusion proteins containing a sequence coding for a signal sequence
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07K2317/00—Immunoglobulins specific features
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    • C07K2317/21—Immunoglobulins specific features characterized by taxonomic origin from primates, e.g. man
    • C—CHEMISTRY; METALLURGY
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    • C07K2317/55—Fab or Fab'
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    • C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
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    • C07K2319/32—Fusion polypeptide fusions with soluble part of a cell surface receptor, "decoy receptors"
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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JP2023526231A 2020-10-29 2021-10-29 眼の適応症に対するベクター化ｔｎｆ－アルファアンタゴニスト Pending JP2023548145A (ja)

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US202063107415P 2020-10-29 2020-10-29
US63/107,415 2020-10-29
PCT/US2021/057380 WO2022094295A1 (en) 2020-10-29 2021-10-29 Vectorized tnf-alpha antagonists for ocular indications

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JP2023548145A true JP2023548145A (ja) 2023-11-15
JP2023548145A5 JP2023548145A5 (enExample) 2024-11-07

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US (1) US20240384292A1 (enExample)
EP (1) EP4237453A1 (enExample)
JP (1) JP2023548145A (enExample)
KR (1) KR20230098176A (enExample)
CN (1) CN116457373A (enExample)
AU (1) AU2021369833A1 (enExample)
CA (1) CA3196964A1 (enExample)
IL (1) IL302282A (enExample)
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Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024238853A1 (en) * 2023-05-16 2024-11-21 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR3031112A1 (fr) * 2014-12-24 2016-07-01 Eyevensys Construction d'adn pour le traitement de pathologies oculaires
WO2019079496A2 (en) * 2017-10-18 2019-04-25 Regenxbio, Inc. FULLY HUMAN ANTIBODY-BASED THERAPEUTIC AGENTS HAVING POST-TRANSLATION MODIFICATION
JP2019518427A (ja) * 2016-04-15 2019-07-04 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 滲出型加齢性黄斑変性の治療のための組成物

Family Cites Families (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0804561B1 (en) 1993-02-12 2009-12-30 The Board Of Trustees Of The Leland Stanford Junior University Regulated transcription of targeted genes and other biological events
AU719001B2 (en) 1994-12-29 2000-05-04 Massachusetts Institute Of Technology Chimeric DNA-binding proteins
WO1996041865A1 (en) 1995-06-07 1996-12-27 Ariad Gene Therapeutics, Inc. Rapamcycin-based regulation of biological events
JP2003524368A (ja) 1997-08-26 2003-08-19 アリアド ジーン セラピューティクス インコーポレイテッド ニ量化ドメイン、三量化ドメインまたは四量化ドメインおよび補足的非相同転写活性化ドメイン、転写抑制ドメイン、ｄｎａ結合ドメインまたはリガンド結合ドメインを含む融合蛋白
JP2001514007A (ja) 1997-08-27 2001-09-11 アリアド ジーン セラピューティクス インコーポレイテッド キメラ転写アクチベーター、ならびにそれに関連する組成物および使用
JP2002508971A (ja) 1998-01-15 2002-03-26 アリアド・ジーン・セラピューティクス・インコーポレーテッド 多量体キメラ蛋白質を使用する生物学的イベントの調節
JP2002503667A (ja) 1998-02-13 2002-02-05 プレジデント・アンド・フェローズ・オブ・ハーバード・カレッジ 新規な二量体化剤、その製造および使用
CA2379166C (en) 1999-08-09 2013-03-26 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 instrastrand base pairs
US7067526B1 (en) 1999-08-24 2006-06-27 Ariad Gene Therapeutics, Inc. 28-epirapalogs
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
US20090010920A1 (en) 2003-03-03 2009-01-08 Xencor, Inc. Fc Variants Having Decreased Affinity for FcyRIIb
US8399618B2 (en) 2004-10-21 2013-03-19 Xencor, Inc. Immunoglobulin insertions, deletions, and substitutions
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
US8367805B2 (en) 2004-11-12 2013-02-05 Xencor, Inc. Fc variants with altered binding to FcRn
US20070135620A1 (en) 2004-11-12 2007-06-14 Xencor, Inc. Fc variants with altered binding to FcRn
US7183969B2 (en) 2004-12-22 2007-02-27 Raytheon Company System and technique for calibrating radar arrays
AU2006230419A1 (en) * 2005-03-31 2006-10-05 Targeted Genetics Corporation Methods for lowering the level of tumor necrosis factor (TNF) in TNF-associated disorders
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
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
US7846724B2 (en) 2006-04-11 2010-12-07 Hoffmann-La Roche Inc. Method for selecting CHO cell for production of glycosylated antibodies
US20090098136A1 (en) * 2007-10-15 2009-04-16 Alcon Research, Ltd. Use of tnf receptor antagonists for treating dry eye
JP2011512156A (ja) 2008-02-19 2011-04-21 アムステルダム モレキュラー セラピューティクス ビー．ブイ． 昆虫細胞におけるパルボウイルスｒｅｐ及びｃａｐタンパク質の発現の最適化
EP2425000B1 (en) 2009-04-30 2019-02-20 The Trustees Of The University Of Pennsylvania Compositions for targeting conducting airway cells comprising adeno-associated virus constructs
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
US10053513B2 (en) 2009-11-30 2018-08-21 Janssen Biotech, Inc. Antibody Fc mutants with ablated effector functions
US8628966B2 (en) 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
WO2012057363A1 (ja) 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
WO2012109570A1 (en) 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
CN105755044A (zh) 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
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
US9382319B2 (en) 2011-09-26 2016-07-05 Jn Biosciences Llc Hybrid constant regions
WO2013170078A1 (en) 2012-05-09 2013-11-14 Oregon Health & Science University Adeno associated virus plasmids and vectors
GB2510803B (en) 2012-11-13 2015-07-22 Cardiff Metropolitan University Trocar assembly
CN103045646B (zh) * 2012-12-27 2015-02-25 中国人民解放军军事医学科学院基础医学研究所 共表达两个独立的抗关节炎分子TNFR-Fc和CTLA4-FasL的重组腺相关病毒载体及其构建方法与应用
CN103131676A (zh) * 2013-01-04 2013-06-05 天津耀宇生物技术有限公司 表达重组人肿瘤坏死因子受体-Fc融合蛋白基因的家蚕重组杆状病毒及其制备方法和应用
TWI635098B (zh) 2013-02-01 2018-09-11 再生元醫藥公司 含嵌合恆定區之抗體
CA2905952A1 (en) 2013-03-13 2014-10-02 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
MX388221B (es) 2013-04-20 2025-03-19 Res Institute At Nationwide Children´S Hospital ADMINISTRACIÓN DE VIRUS ADENO-ASOCIADO RECOMBINANTE DE CONSTRUCCIONES DE POLINUCLÉOTIDOS U7snRNA DIRIGIDA AL EXÓN 2.
IL297919A (en) 2013-07-22 2023-01-01 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
ES2739288T3 (es) 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
CN106232618A (zh) 2013-10-11 2016-12-14 马萨诸塞眼科耳科诊所 预测祖先病毒序列的方法及其用途
WO2015164757A1 (en) 2014-04-25 2015-10-29 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
US10000551B2 (en) * 2014-09-11 2018-06-19 Protalix Ltd. Chimeric polypeptides, polynucleotides encoding same, cells expressing same and methods of producing same
AU2015320694B2 (en) 2014-09-24 2021-11-11 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
WO2016161010A2 (en) 2015-03-30 2016-10-06 Regeneron Pharmaceuticals, Inc. Heavy chain constant regions with reduced binding to fc gamma receptors
JP6665466B2 (ja) 2015-09-26 2020-03-13 日亜化学工業株式会社 半導体発光素子及びその製造方法
WO2017070491A1 (en) 2015-10-23 2017-04-27 Applied Genetic Technologies Corporation Ophthalmic formulations
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR3031112A1 (fr) * 2014-12-24 2016-07-01 Eyevensys Construction d'adn pour le traitement de pathologies oculaires
JP2019518427A (ja) * 2016-04-15 2019-07-04 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 滲出型加齢性黄斑変性の治療のための組成物
WO2019079496A2 (en) * 2017-10-18 2019-04-25 Regenxbio, Inc. FULLY HUMAN ANTIBODY-BASED THERAPEUTIC AGENTS HAVING POST-TRANSLATION MODIFICATION

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
INT. J. MOL. SCI., vol. vol.21, issue 12, JPN6025031328, June 2020 (2020-06-01), pages 1 - 52, ISSN: 0005781593 *
MOL. THER., vol. 26, no. 6, JPN6025031330, 2018, pages 1568 - 1580, ISSN: 0005781594 *
ウイルス, vol. 57, no. 1, JPN6025031327, 2007, pages 47 - 56, ISSN: 0005781592 *

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