AU2020229871B2 - Adeno-associated virus vectors for the delivery of therapeutics - Google Patents

Adeno-associated virus vectors for the delivery of therapeutics Download PDF

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Publication number
AU2020229871B2
AU2020229871B2 AU2020229871A AU2020229871A AU2020229871B2 AU 2020229871 B2 AU2020229871 B2 AU 2020229871B2 AU 2020229871 A AU2020229871 A AU 2020229871A AU 2020229871 A AU2020229871 A AU 2020229871A AU 2020229871 B2 AU2020229871 B2 AU 2020229871B2
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Australia
Prior art keywords
chr1
chrl
aav
dystrophy
gene
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AU2020229871A
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AU2020229871A1 (en
Inventor
Ann J. LIGOCKI
Carmelo Romano
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Regeneron Pharmaceuticals Inc
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Regeneron Pharmaceuticals Inc
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Publication of AU2020229871B2 publication Critical patent/AU2020229871B2/en
Priority to AU2023216849A priority Critical patent/AU2023216849A1/en
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K9/0051—Ocular inserts or implants
    • 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
    • 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
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5176—Compounds of unknown constitution, e.g. material from plants or animals
    • A61K9/5184—Virus capsids or envelopes enclosing drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Optics & Photonics (AREA)
  • Nanotechnology (AREA)
  • Botany (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2020229871A 2019-02-28 2020-02-27 Adeno-associated virus vectors for the delivery of therapeutics Active AU2020229871B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2023216849A AU2023216849A1 (en) 2019-02-28 2023-08-17 Adeno-associated virus vectors for the delivery of therapeutics

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962812017P 2019-02-28 2019-02-28
US62/812,017 2019-02-28
US201962831838P 2019-04-10 2019-04-10
US62/831,838 2019-04-10
US201962878865P 2019-07-26 2019-07-26
US62/878,865 2019-07-26
PCT/US2020/020134 WO2020176747A1 (en) 2019-02-28 2020-02-27 Adeno-associated virus vectors for the delivery of therapeutics

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2023216849A Division AU2023216849A1 (en) 2019-02-28 2023-08-17 Adeno-associated virus vectors for the delivery of therapeutics

Publications (2)

Publication Number Publication Date
AU2020229871A1 AU2020229871A1 (en) 2021-10-21
AU2020229871B2 true AU2020229871B2 (en) 2023-05-18

Family

ID=70155318

Family Applications (2)

Application Number Title Priority Date Filing Date
AU2020229871A Active AU2020229871B2 (en) 2019-02-28 2020-02-27 Adeno-associated virus vectors for the delivery of therapeutics
AU2023216849A Abandoned AU2023216849A1 (en) 2019-02-28 2023-08-17 Adeno-associated virus vectors for the delivery of therapeutics

Family Applications After (1)

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AU2023216849A Abandoned AU2023216849A1 (en) 2019-02-28 2023-08-17 Adeno-associated virus vectors for the delivery of therapeutics

Country Status (8)

Country Link
US (1) US20220047618A1 (enExample)
EP (1) EP3930694A1 (enExample)
JP (2) JP2022522756A (enExample)
CN (1) CN113557010A (enExample)
AU (2) AU2020229871B2 (enExample)
CA (1) CA3131919A1 (enExample)
IL (1) IL285639A (enExample)
WO (1) WO2020176747A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2022216614B2 (en) 2021-02-05 2026-03-05 Christiana Care Gene Editing Institute, Inc. Methods of and compositions for reducing gene expression and/or activity
CN115505593B (zh) * 2021-06-23 2025-10-10 武汉纽福斯生物科技有限公司 寡核苷酸、病毒载体及其应用和RNAi药物制剂
US20250319202A1 (en) * 2021-11-09 2025-10-16 Prime Medicine, Inc. Genome editing compositions and methods for treatment of fuchs endothelial corneal dystrophy
WO2023111351A1 (en) * 2021-12-17 2023-06-22 Christian Kupatt Aav vectors for gene therapy in endothelial cells

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017185054A1 (en) * 2016-04-22 2017-10-26 Intellia Therapeutics, Inc. Compositions and methods for treatment of diseases associated with trinucleotide repeats in transcription factor four
WO2018165541A1 (en) * 2017-03-10 2018-09-13 The Board Of Regents Of The University Of Texas System Treatment of fuchs' endothelial corneal dystrophy
WO2019025984A1 (en) * 2017-07-31 2019-02-07 Reflection Biotechnologies Limited CELLULAR MODELS AND THERAPIES FOR OCULAR DISEASES

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103589692B (zh) 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
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
CN103502458B (zh) 2011-02-17 2016-11-16 宾夕法尼亚大学托管会 用于改变组织特异性和改善aav9-介导的基因转移的组合物和方法
DK3693025T3 (da) 2011-04-22 2022-01-10 Univ California Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf
CN105899658B (zh) * 2013-12-12 2020-02-18 布罗德研究所有限公司 针对hbv和病毒性疾病以及障碍的crispr-cas系统和组合物的递送、用途和治疗应用
CN111206032B (zh) * 2013-12-12 2024-07-19 布罗德研究所有限公司 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用
IL251178B (en) 2014-09-16 2022-07-01 Genzyme Corp Adeno-associated viral vector for the treatment of glaucoma myocilin (myoc)
PL3298134T3 (pl) * 2015-05-16 2023-09-18 Genzyme Corporation Edycja genów mutacji głęboko intronowych
AU2016335032B2 (en) * 2015-10-05 2022-04-14 Proqr Therapeutics Ii B.V. Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion
WO2017178590A1 (en) * 2016-04-14 2017-10-19 Université de Lausanne Treatment and/or prevention of dna-triplet repeat diseases or disorders
EP3342868B1 (en) * 2016-12-30 2019-12-25 Systasy Bioscience GmbH Constructs and screening methods
EP3570888A1 (en) * 2017-03-03 2019-11-27 MacRegen, Inc. Treatment of age-related macular degeneration and other eye diseases with one or more therapeutic agents

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017185054A1 (en) * 2016-04-22 2017-10-26 Intellia Therapeutics, Inc. Compositions and methods for treatment of diseases associated with trinucleotide repeats in transcription factor four
WO2018165541A1 (en) * 2017-03-10 2018-09-13 The Board Of Regents Of The University Of Texas System Treatment of fuchs' endothelial corneal dystrophy
WO2019025984A1 (en) * 2017-07-31 2019-02-07 Reflection Biotechnologies Limited CELLULAR MODELS AND THERAPIES FOR OCULAR DISEASES

Also Published As

Publication number Publication date
WO2020176747A1 (en) 2020-09-03
CN113557010A (zh) 2021-10-26
EP3930694A1 (en) 2022-01-05
JP2025061656A (ja) 2025-04-11
IL285639A (en) 2021-09-30
US20220047618A1 (en) 2022-02-17
JP2022522756A (ja) 2022-04-20
AU2020229871A1 (en) 2021-10-21
CA3131919A1 (en) 2020-09-03
AU2023216849A1 (en) 2023-09-28

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