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DK3472183T3 - Variant adeno-associerede virus og fremgangsmåder til anvendelse - Google Patents

Variant adeno-associerede virus og fremgangsmåder til anvendelse Download PDF

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Publication number
DK3472183T3
DK3472183T3 DK17734575.8T DK17734575T DK3472183T3 DK 3472183 T3 DK3472183 T3 DK 3472183T3 DK 17734575 T DK17734575 T DK 17734575T DK 3472183 T3 DK3472183 T3 DK 3472183T3
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Denmark
Prior art keywords
methods
associated viruses
variant adeno
adeno
variant
Prior art date
Application number
DK17734575.8T
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English (en)
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DK3472183T5 (da
Inventor
Joshua Dudman
Adam Hantman
Loren Looger
Kimberly Ritola
David Schaffer
Sarada Viswanathan
Dougal Gowanlock Robinson Tervo
Bum-Yeol Hwang
Alla Karpova
Original Assignee
Hughes Howard Med Inst
Univ California
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Publication of DK3472183T3 publication Critical patent/DK3472183T3/da
Publication of DK3472183T5 publication Critical patent/DK3472183T5/da

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    • 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
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
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    • 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
    • C40—COMBINATORIAL TECHNOLOGY
    • C40B—COMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B30/00—Methods of screening libraries
    • C40B30/06—Methods of screening libraries by measuring effects on living organisms, tissues or cells
    • C—CHEMISTRY; METALLURGY
    • C40—COMBINATORIAL TECHNOLOGY
    • C40B—COMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B40/00—Libraries per se, e.g. arrays, mixtures
    • C40B40/04—Libraries containing only organic compounds
    • C40B40/06—Libraries containing nucleotides or polynucleotides, or derivatives thereof
    • C40B40/08—Libraries containing RNA or DNA which encodes proteins, e.g. gene libraries
    • 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
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    • 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
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Neurology (AREA)
  • Molecular Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychiatry (AREA)
  • Psychology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pain & Pain Management (AREA)
DK17734575.8T 2016-06-15 2017-06-15 Variant adeno-associerede virus og fremgangsmåder til anvendelse DK3472183T5 (da)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662350361P 2016-06-15 2016-06-15
US201662404585P 2016-10-05 2016-10-05
PCT/US2017/037781 WO2017218842A1 (en) 2016-06-15 2017-06-15 Variant adeno-associated viruses and methods of using

Publications (2)

Publication Number Publication Date
DK3472183T3 true DK3472183T3 (da) 2023-11-06
DK3472183T5 DK3472183T5 (da) 2024-09-09

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US (2) US10961282B2 (da)
EP (2) EP3472183B1 (da)
JP (2) JP7094277B2 (da)
KR (2) KR20230054754A (da)
CN (2) CN109641939B (da)
AU (2) AU2017286652B2 (da)
BR (1) BR112018076090A2 (da)
CA (1) CA3028113A1 (da)
DK (1) DK3472183T5 (da)
EA (1) EA201990033A1 (da)
ES (1) ES2962611T3 (da)
FI (1) FI3472183T3 (da)
HR (1) HRP20231403T1 (da)
HU (1) HUE063755T2 (da)
IL (2) IL263719B2 (da)
LT (1) LT3472183T (da)
MA (1) MA44546B1 (da)
MX (2) MX2018015770A (da)
MY (1) MY190221A (da)
PH (1) PH12018502664A1 (da)
PL (1) PL3472183T3 (da)
PT (1) PT3472183T (da)
RS (1) RS64769B1 (da)
SG (1) SG11201811189RA (da)
SI (1) SI3472183T1 (da)
WO (1) WO2017218842A1 (da)
ZA (1) ZA201900278B (da)

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MA44546B1 (fr) 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
WO2018022905A2 (en) 2016-07-29 2018-02-01 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
AU2018324477A1 (en) 2017-08-28 2019-10-17 The Regents Of The University Of California Adeno-associated virus capsid variants and methods of use thereof
CN111601884A (zh) * 2017-10-16 2020-08-28 维格内罗有限责任公司 Aav载体
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WO2023192450A1 (en) * 2022-03-30 2023-10-05 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Adeno-associated virus vectors for nucleic acid delivery to retinal ganglion cells and/or retinal pigment epithelium cells
WO2024059667A2 (en) * 2022-09-13 2024-03-21 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
WO2024056902A2 (en) 2022-09-16 2024-03-21 Christopher Shaw Compositions and methods for treating neurological diseases
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WO2024079530A2 (en) 2022-10-11 2024-04-18 Meiragtx Uk Ii Limited Nucleic acid regulatory elements for gene expression in the muscle and methods of use
WO2024079661A1 (en) 2022-10-11 2024-04-18 Meiragtx Uk Ii Limited Atp7b gene therapy
WO2024079657A1 (en) 2022-10-11 2024-04-18 Meiragtx Uk Ii Limited Formulations for aav gene therapy
WO2024079662A1 (en) 2022-10-11 2024-04-18 Meiragtx Uk Ii Limited Upf1 expression constructs
WO2024126696A1 (en) 2022-12-14 2024-06-20 King's College London Compositions and methods for treating neurological diseases
WO2024148189A2 (en) * 2023-01-05 2024-07-11 Emugen Therapeutics Llc Neuronal promoters and uses thereof
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WO2024161022A2 (en) 2023-02-03 2024-08-08 King's College London Compositions and methods for treating neurological diseases
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WO2018022905A2 (en) * 2016-07-29 2018-02-01 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof

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PL3472183T3 (pl) 2024-02-12
MA44546B1 (fr) 2021-03-31
US20210230231A1 (en) 2021-07-29
HRP20231403T1 (hr) 2024-02-16
PT3472183T (pt) 2023-11-16
KR102522661B1 (ko) 2023-04-18
EP4268852A3 (en) 2024-02-07
LT3472183T (lt) 2023-09-25
FI3472183T3 (fi) 2023-11-03
PH12018502664A1 (en) 2019-10-07
IL263719A (en) 2019-01-31
MX2018015770A (es) 2019-08-29
SI3472183T1 (sl) 2023-11-30
EP4268852A2 (en) 2023-11-01
KR20190039930A (ko) 2019-04-16
JP2019518793A (ja) 2019-07-04
CN109641939B (zh) 2022-11-08
MY190221A (en) 2022-04-06
JP7579301B2 (ja) 2024-11-07
ES2962611T3 (es) 2024-03-20
SG11201811189RA (en) 2019-01-30
IL263719B2 (en) 2023-09-01
EP3472183B1 (en) 2023-08-09
AU2017286652A1 (en) 2019-01-24
CN116003532A (zh) 2023-04-25
US20190300579A1 (en) 2019-10-03
MX2024000505A (es) 2024-03-19
JP7094277B2 (ja) 2022-07-01
EA201990033A1 (ru) 2019-06-28
US10961282B2 (en) 2021-03-30
HUE063755T2 (hu) 2024-01-28
WO2017218842A1 (en) 2017-12-21
BR112018076090A2 (pt) 2019-03-26
IL300981A (en) 2023-04-01
IL263719B1 (en) 2023-05-01
KR20230054754A (ko) 2023-04-25
JP2022126759A (ja) 2022-08-30
DK3472183T5 (da) 2024-09-09
AU2017286652B2 (en) 2021-10-07
AU2021225247A1 (en) 2021-09-30
CA3028113A1 (en) 2017-12-21
ZA201900278B (en) 2022-04-28
MA44546A1 (fr) 2019-07-31
EP3472183A1 (en) 2019-04-24
AU2021225247B2 (en) 2023-02-16
US11939355B2 (en) 2024-03-26
CN109641939A (zh) 2019-04-16
RS64769B1 (sr) 2023-11-30

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