AU2018228881B2 - Gene therapy for ocular disorders - Google Patents

Gene therapy for ocular disorders Download PDF

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Publication number
AU2018228881B2
AU2018228881B2 AU2018228881A AU2018228881A AU2018228881B2 AU 2018228881 B2 AU2018228881 B2 AU 2018228881B2 AU 2018228881 A AU2018228881 A AU 2018228881A AU 2018228881 A AU2018228881 A AU 2018228881A AU 2018228881 B2 AU2018228881 B2 AU 2018228881B2
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AU
Australia
Prior art keywords
lca5
aav
raav
sequence
capsid
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AU2018228881A
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AU2018228881A1 (en
Inventor
Jean Bennett
Jeannette BENNICELLI
Sergei NIKONOV
Ji-yun SONG
Junwei Sun
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University of Pennsylvania Penn
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University of Pennsylvania Penn
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Priority to AU2024202693A priority Critical patent/AU2024202693A1/en
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special targeting system for viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/22Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Ophthalmology & Optometry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
AU2018228881A 2017-03-01 2018-03-01 Gene therapy for ocular disorders Active AU2018228881B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2024202693A AU2024202693A1 (en) 2017-03-01 2024-04-24 Gene Therapy For Ocular Disorders

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762465649P 2017-03-01 2017-03-01
US62/465,649 2017-03-01
US201762469642P 2017-03-10 2017-03-10
US62/469,642 2017-03-10
PCT/US2018/020470 WO2018160849A1 (en) 2017-03-01 2018-03-01 Gene therapy for ocular disorders

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2024202693A Division AU2024202693A1 (en) 2017-03-01 2024-04-24 Gene Therapy For Ocular Disorders

Publications (2)

Publication Number Publication Date
AU2018228881A1 AU2018228881A1 (en) 2019-09-12
AU2018228881B2 true AU2018228881B2 (en) 2024-01-25

Family

ID=63370488

Family Applications (2)

Application Number Title Priority Date Filing Date
AU2018228881A Active AU2018228881B2 (en) 2017-03-01 2018-03-01 Gene therapy for ocular disorders
AU2024202693A Pending AU2024202693A1 (en) 2017-03-01 2024-04-24 Gene Therapy For Ocular Disorders

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2024202693A Pending AU2024202693A1 (en) 2017-03-01 2024-04-24 Gene Therapy For Ocular Disorders

Country Status (11)

Country Link
US (2) US11564996B2 (enExample)
EP (2) EP4656244A2 (enExample)
JP (3) JP7211960B2 (enExample)
KR (3) KR20240104196A (enExample)
CN (2) CN119752819A (enExample)
AU (2) AU2018228881B2 (enExample)
BR (1) BR112019017327A2 (enExample)
CA (1) CA3054136A1 (enExample)
IL (2) IL314284A (enExample)
RU (1) RU2019130004A (enExample)
WO (1) WO2018160849A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
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AU2019296451B2 (en) * 2018-06-29 2021-04-29 Wuhan Neurophth Biotechnology Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
JP7640549B2 (ja) * 2019-12-12 2025-03-05 ノバルティス アーゲー 注射デバイス及び注射溶液移し替えシステム
EP4284335A1 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
US20220387627A1 (en) * 2021-06-03 2022-12-08 Hope for Hasti Vectors and gene therapy for treating cornelia de lange syndrome

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WO2016019364A1 (en) * 2014-08-01 2016-02-04 The Trustees Of The University Of Pennsylvania Compositions and methods for self-regulated inducible gene expression

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US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
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Also Published As

Publication number Publication date
BR112019017327A2 (pt) 2020-04-14
AU2018228881A1 (en) 2019-09-12
JP7671790B2 (ja) 2025-05-02
EP4656244A2 (en) 2025-12-03
CN110582572A (zh) 2019-12-17
EP3589738A1 (en) 2020-01-08
KR20240104196A (ko) 2024-07-04
US11564996B2 (en) 2023-01-31
US20230233709A1 (en) 2023-07-27
RU2019130004A3 (enExample) 2021-07-05
IL314284A (en) 2024-09-01
KR20190125357A (ko) 2019-11-06
CA3054136A1 (en) 2018-09-07
JP2020510433A (ja) 2020-04-09
WO2018160849A8 (en) 2019-10-03
JP2023040219A (ja) 2023-03-22
US20190388561A1 (en) 2019-12-26
JP7211960B2 (ja) 2023-01-24
KR102545070B1 (ko) 2023-06-19
EP3589738A4 (en) 2021-01-06
JP2025114604A (ja) 2025-08-05
IL268946A (en) 2019-10-31
RU2019130004A (ru) 2021-04-01
KR20230093072A (ko) 2023-06-26
CN119752819A (zh) 2025-04-04
WO2018160849A1 (en) 2018-09-07
AU2024202693A1 (en) 2024-05-16

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