AU2018227483A1 - Modified AAV capsids and uses thereof - Google Patents

Modified AAV capsids and uses thereof Download PDF

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Publication number
AU2018227483A1
AU2018227483A1 AU2018227483A AU2018227483A AU2018227483A1 AU 2018227483 A1 AU2018227483 A1 AU 2018227483A1 AU 2018227483 A AU2018227483 A AU 2018227483A AU 2018227483 A AU2018227483 A AU 2018227483A AU 2018227483 A1 AU2018227483 A1 AU 2018227483A1
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AU
Australia
Prior art keywords
aav
capsid protein
amino acid
viral vector
vector
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.)
Abandoned
Application number
AU2018227483A
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English (en)
Inventor
Annahita KERAVALA
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Adverum Biotechnologies Inc
Original Assignee
Adverum Biotechnologies Inc
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Filing date
Publication date
Application filed by Adverum Biotechnologies Inc filed Critical Adverum Biotechnologies Inc
Publication of AU2018227483A1 publication Critical patent/AU2018227483A1/en
Abandoned legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81—Protease inhibitors
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/22—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against growth factors ; against growth regulators
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • 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
    • C12N2750/14145—Special targeting system for viral vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
AU2018227483A 2017-02-28 2018-02-28 Modified AAV capsids and uses thereof Abandoned AU2018227483A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762464878P 2017-02-28 2017-02-28
US62/464,878 2017-02-28
PCT/US2018/020215 WO2018160686A1 (en) 2017-02-28 2018-02-28 Modified aav capsids and uses thereof

Publications (1)

Publication Number Publication Date
AU2018227483A1 true AU2018227483A1 (en) 2019-09-12

Family

ID=63370228

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2018227483A Abandoned AU2018227483A1 (en) 2017-02-28 2018-02-28 Modified AAV capsids and uses thereof

Country Status (7)

Country Link
US (1) US20210130413A1 (enExample)
EP (1) EP3589277A4 (enExample)
JP (1) JP2020510424A (enExample)
CN (1) CN110650733A (enExample)
AU (1) AU2018227483A1 (enExample)
CA (1) CA3054687A1 (enExample)
WO (1) WO2018160686A1 (enExample)

Cited By (1)

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CN114686448A (zh) * 2022-03-31 2022-07-01 上海勉亦生物科技有限公司 具有肝脏特异靶向性的纯化的腺相关病毒及其应用

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JP6689249B2 (ja) 2014-03-17 2020-05-20 アドヴェラム バイオテクノロジーズ， インコーポレイテッド 錐体細胞における増強された遺伝子発現のための組成物および方法
AU2016226289B2 (en) 2015-03-02 2021-04-29 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
US12173305B2 (en) 2016-05-26 2024-12-24 University Of Iowa Research Foundation cis and trans requirements for terminal resolution of human bocavirus 1
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
CA3054942A1 (en) 2017-03-17 2018-09-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression
WO2019079494A1 (en) * 2017-10-18 2019-04-25 Regenxbio, Inc. TREATMENT OF OCULAR DISEASES AND METASTATIC COLON CANCER WITH A VEGF TRAP WITH HUMAN POST-TRANSLATIONAL MODIFICATION
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
CN113966399A (zh) * 2018-09-26 2022-01-21 加州理工学院 用于靶向基因疗法的腺相关病毒组合物
EP3934698A1 (en) * 2019-03-04 2022-01-12 Adverum Biotechnologies, Inc. Sequential intravitreal administration of aav gene therapy to contralateral eyes
TW202102526A (zh) * 2019-04-04 2021-01-16 美商銳進科斯生物股份有限公司 重組腺相關病毒及其用途
WO2020214672A1 (en) * 2019-04-15 2020-10-22 University Of Iowa Research Foundation Methods and compositions for transgene expression
SG11202111414RA (en) * 2019-04-24 2021-11-29 Regenxbio Inc Fully-human post-translationally modified antibody therapeutics
TW202532428A (zh) * 2019-04-26 2025-08-16 美商愛德維仁生物科技公司 用於玻璃體內遞送之變異體aav蛋白殼
EP4125977A4 (en) * 2020-05-05 2024-09-25 Duke University CROSS-SPECIES COMPATIBLE ADENO-ASSOCIATED VIRUS COMPOSITIONS AND METHODS OF USE THEREOF
CN115927398B (zh) * 2020-07-29 2024-11-19 北京三诺佳邑生物技术有限责任公司 一组肝靶向新型腺相关病毒的获得及其应用
JP2023540094A (ja) * 2020-09-03 2023-09-21 ユニバーシティ オブ マサチューセッツ Ｋｈ９０２（コンベルセプト）の送達のためのアデノ随伴ウイルスおよびその使用
AR123948A1 (es) * 2020-10-28 2023-01-25 Regenxbio Inc ANTICUERPOS ANTI-TNF-a VECTORIZADOS PARA INDICACIONES OCULARES
CN112961220B (zh) * 2021-04-19 2023-11-17 信念医药科技(上海)有限公司 腺相关病毒衣壳蛋白及包含其的腺相关病毒载体
CN113121652B (zh) * 2021-04-19 2022-10-11 上海信致医药科技有限公司 视网膜和肌肉高亲和性腺相关病毒衣壳蛋白及相关应用
CN117157309A (zh) 2021-05-28 2023-12-01 上海瑞宏迪医药有限公司 衣壳变异的重组腺相关病毒及其应用
CN113564187A (zh) * 2021-07-30 2021-10-29 上海信致医药科技有限公司 基于aav的抗血管生成基因递送系统及其用途
CN117230117A (zh) * 2023-08-04 2023-12-15 中国科学院深圳先进技术研究院 重组腺相关病毒包装质粒、突变体及其制备方法和应用
CN117143920A (zh) * 2023-08-30 2023-12-01 呈诺再生医学科技(北京)有限公司 一种治疗新生血管性眼部疾病的基因药物
CN117368466B (zh) * 2023-12-08 2024-03-12 安徽惠邦生物工程有限公司 用生化分析仪测定尿结合珠蛋白的试剂盒及其制备方法
WO2025214359A1 (en) * 2024-04-09 2025-10-16 Hangzhou Jiayin Biotech Ltd. A novel aav variant

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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
US8663624B2 (en) * 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
DK2699270T3 (en) * 2011-04-22 2017-10-02 Univ California ADENO-ASSOCIATED VIRUS VIRUSES WITH VARIANT CAPSID AND METHODS FOR USING IT
GB201403260D0 (en) * 2014-02-25 2014-04-09 Univ Manchester Treatment of retinal degeneration using gene therapy
EP3117002B1 (en) * 2014-03-11 2025-12-03 Wayne State University A modified mglur6 promoter and methods of use

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114686448A (zh) * 2022-03-31 2022-07-01 上海勉亦生物科技有限公司 具有肝脏特异靶向性的纯化的腺相关病毒及其应用

Also Published As

Publication number Publication date
US20210130413A1 (en) 2021-05-06
EP3589277A1 (en) 2020-01-08
CN110650733A (zh) 2020-01-03
JP2020510424A (ja) 2020-04-09
CA3054687A1 (en) 2018-09-07
EP3589277A4 (en) 2020-08-26
WO2018160686A1 (en) 2018-09-07

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MK4 Application lapsed section 142(2)(d) - no continuation fee paid for the application