JPWO2021009805A1 - - Google Patents

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Publication number
JPWO2021009805A1
JPWO2021009805A1 JP2021532564A JP2021532564A JPWO2021009805A1 JP WO2021009805 A1 JPWO2021009805 A1 JP WO2021009805A1 JP 2021532564 A JP2021532564 A JP 2021532564A JP 2021532564 A JP2021532564 A JP 2021532564A JP WO2021009805 A1 JPWO2021009805 A1 JP WO2021009805A1
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JP
Japan
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Application number
JP2021532564A
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JP7371954B2 (ja
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Publication of JPWO2021009805A1 publication Critical patent/JPWO2021009805A1/ja
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Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
JP2021532564A 2019-07-12 2019-07-12 ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン Active JP7371954B2 (ja)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
PCT/JP2019/027717 WO2021009805A1 (ja) 2019-07-12 2019-07-12 ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン

Publications (2)

Publication Number Publication Date
JPWO2021009805A1 true JPWO2021009805A1 (ja) 2021-01-21
JP7371954B2 JP7371954B2 (ja) 2023-10-31

Family

ID=74210253

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021532564A Active JP7371954B2 (ja) 2019-07-12 2019-07-12 ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン

Country Status (4)

Country Link
US (1) US20220265853A1 (ja)
EP (1) EP4006160A4 (ja)
JP (1) JP7371954B2 (ja)
WO (1) WO2021009805A1 (ja)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018139634A1 (ja) * 2017-01-30 2018-08-02 学校法人日本医科大学 アデノ随伴ウイルス（ａａｖ）キャプシドタンパク質の変異体
WO2022224372A1 (ja) * 2021-04-21 2022-10-27 学校法人自治医科大学 オルニチントランスカルバミラーゼ欠損症の治療用アデノ随伴ウイルスビリオン
WO2023106256A1 (ja) * 2021-12-06 2023-06-15 学校法人順天堂 改変型アデノ随伴ウイルスベクター

Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016526045A (ja) * 2013-05-31 2016-09-01 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア アデノ随伴ウイルス変異体及びその使用方法
JP2017506521A (ja) * 2014-02-17 2017-03-09 キングス カレッジ ロンドンＫｉｎｇｓ Ｃｏｌｌｅｇｅ Ｌｏｎｄｏｎ アデノ随伴ウイルスベクター
WO2018131551A1 (ja) * 2017-01-13 2018-07-19 学校法人自治医科大学 肝臓ゲノム上の凝固関連因子遺伝子を破壊するためのａａｖベクター
WO2018139634A1 (ja) * 2017-01-30 2018-08-02 学校法人日本医科大学 アデノ随伴ウイルス（ａａｖ）キャプシドタンパク質の変異体
JP2018520997A (ja) * 2015-05-11 2018-08-02 ユーシーエル ビジネス パブリック リミテッド カンパニー キャプシド
JP2018528253A (ja) * 2015-09-28 2018-09-27 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 抗体回避性ウイルスベクターのための方法および組成物
WO2018200419A1 (en) * 2017-04-23 2018-11-01 The Trustees Of The University Of Pennsylvania Viral vectors comprising engineered aav capsids and compositions containing the same
US20190142975A1 (en) * 2016-04-29 2019-05-16 Adverum Biotechnologies, Inc. Evasion of neutralizing antibodies by a recombinant adeno-associated virus
JP2019513401A (ja) * 2016-04-15 2019-05-30 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 新規なａａｖ８変異カプシド及びそれを含有する組成物

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
JP5500750B2 (ja) 2000-03-30 2014-05-21 ホワイトヘッド インスチチュート フォアー バイオメディカル リサーチ Ｒｎａ干渉のｒｎａ配列特異的メディエータ
US20030050273A1 (en) 2001-08-29 2003-03-13 Keiya Ozawa Compositions and methods for treating neurodegenerative diseases
US7112321B2 (en) 2001-12-19 2006-09-26 Genzyme Corporation Adeno-associated virus-mediated delivery of GDNF to skeletal muscles
JPWO2007001010A1 (ja) 2005-06-29 2009-01-22 国立大学法人金沢大学 パーキンソン病の治療のための医薬
PT2191001T (pt) 2007-04-09 2016-09-23 Univ Florida Composições com vetores raav possuindo proteínas da cápside modificadas com tirosina e métodos para o seu uso
CN103189507A (zh) 2010-10-27 2013-07-03 学校法人自治医科大学 用于向神经***细胞导入基因的腺相关病毒粒子
CN110606874B (zh) 2013-07-22 2024-04-19 费城儿童医院 用于基因转移到细胞、器官和组织中的变异aav和组合物、方法及用途
CN115093464A (zh) * 2013-10-11 2022-09-23 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
JP6592208B2 (ja) * 2016-05-13 2019-10-16 ４ディー モレキュラー セラピューティクス インコーポレイテッド アデノ関連ウイルス変異キャプシドおよびその使用方法

Patent Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016526045A (ja) * 2013-05-31 2016-09-01 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア アデノ随伴ウイルス変異体及びその使用方法
JP2017506521A (ja) * 2014-02-17 2017-03-09 キングス カレッジ ロンドンＫｉｎｇｓ Ｃｏｌｌｅｇｅ Ｌｏｎｄｏｎ アデノ随伴ウイルスベクター
JP2018520997A (ja) * 2015-05-11 2018-08-02 ユーシーエル ビジネス パブリック リミテッド カンパニー キャプシド
JP2018528253A (ja) * 2015-09-28 2018-09-27 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 抗体回避性ウイルスベクターのための方法および組成物
JP2019513401A (ja) * 2016-04-15 2019-05-30 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 新規なａａｖ８変異カプシド及びそれを含有する組成物
US20190142975A1 (en) * 2016-04-29 2019-05-16 Adverum Biotechnologies, Inc. Evasion of neutralizing antibodies by a recombinant adeno-associated virus
WO2018131551A1 (ja) * 2017-01-13 2018-07-19 学校法人自治医科大学 肝臓ゲノム上の凝固関連因子遺伝子を破壊するためのａａｖベクター
WO2018139634A1 (ja) * 2017-01-30 2018-08-02 学校法人日本医科大学 アデノ随伴ウイルス（ａａｖ）キャプシドタンパク質の変異体
WO2018200419A1 (en) * 2017-04-23 2018-11-01 The Trustees Of The University Of Pennsylvania Viral vectors comprising engineered aav capsids and compositions containing the same

Also Published As

Publication number Publication date
US20220265853A1 (en) 2022-08-25
EP4006160A1 (en) 2022-06-01
EP4006160A4 (en) 2023-06-28
JP7371954B2 (ja) 2023-10-31
WO2021009805A1 (ja) 2021-01-21

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