DK3445773T5 - Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf - Google Patents

Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf Download PDF

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Publication number
DK3445773T5
DK3445773T5 DK17797002.7T DK17797002T DK3445773T5 DK 3445773 T5 DK3445773 T5 DK 3445773T5 DK 17797002 T DK17797002 T DK 17797002T DK 3445773 T5 DK3445773 T5 DK 3445773T5
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adeno
methods
same
associated virus
virus variant
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DK17797002.7T
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DK3445773T3 (da
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David H Kirn
Melissa Kotterman
David Schaffer
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4D Molecular Therapeutics Inc
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    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0016—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • A61K39/235—Adenoviridae
    • 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
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
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    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
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    • A61K9/00—Medicinal preparations characterised by special physical form
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    • 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
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
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    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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

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  • Health & Medical Sciences (AREA)
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  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Ophthalmology & Optometry (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
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DK17797002.7T 2016-05-13 2017-05-12 Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf DK3445773T5 (da)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662336441P 2016-05-13 2016-05-13
US201662378106P 2016-08-22 2016-08-22
US201662384590P 2016-09-07 2016-09-07
US201762454612P 2017-02-03 2017-02-03
PCT/US2017/032542 WO2017197355A2 (en) 2016-05-13 2017-05-12 Adeno-associated virus variant capsids and methods of use thereof

Publications (2)

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DK3445773T3 DK3445773T3 (da) 2023-03-20
DK3445773T5 true DK3445773T5 (da) 2024-09-09

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DK22208172.1T DK4206216T3 (da) 2016-05-13 2017-05-12 Adeno-associerede virusvarianter og fremgangsmåder til brug deraf

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US (9) US11364308B2 (da)
EP (3) EP3445773B1 (da)
JP (1) JP6592208B2 (da)
KR (1) KR102234930B1 (da)
CN (3) CN109476707B (da)
AU (1) AU2017261812B2 (da)
BR (1) BR112018072849B1 (da)
CA (1) CA3023592C (da)
CL (1) CL2018003196A1 (da)
CO (1) CO2018013255A2 (da)
CR (1) CR20180589A (da)
DK (2) DK3445773T5 (da)
ES (2) ES2941502T3 (da)
FI (2) FI3445773T3 (da)
IL (1) IL262922B (da)
MA (1) MA44740A (da)
MX (2) MX2018013463A (da)
NZ (1) NZ748395A (da)
PE (1) PE20190129A1 (da)
PH (1) PH12018502387A1 (da)
PT (2) PT3445773T (da)
RU (1) RU2725286C2 (da)
SA (1) SA518400418B1 (da)
SG (1) SG11201809684YA (da)
UA (1) UA124343C2 (da)
WO (1) WO2017197355A2 (da)
ZA (2) ZA201807625B (da)

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AU2019227910B2 (en) 2018-02-28 2025-07-17 The Regents Of The University Of California A modular system for gene and protein delivery based on AAV
KR20250111393A (ko) 2018-03-16 2025-07-22 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 캡시드 변형에 의한 증가하는 조직 특정 유전자 전달
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SG11201809684YA (en) 2018-11-29
CN109476707B (zh) 2022-12-02
US20210069349A1 (en) 2021-03-11
AU2017261812B2 (en) 2019-04-18
IL262922A (en) 2018-12-31
EP4206216B1 (en) 2025-10-08
EP4209501A1 (en) 2023-07-12
RU2725286C2 (ru) 2020-06-30
CA3023592C (en) 2020-12-08
RU2018142768A3 (da) 2020-06-16
US20240066145A1 (en) 2024-02-29
US11576983B2 (en) 2023-02-14
ZA201905485B (en) 2021-03-31
US11179477B2 (en) 2021-11-23
FI3445773T3 (fi) 2023-03-30
US11167041B2 (en) 2021-11-09
ZA201807625B (en) 2020-02-26
BR112018072849A2 (pt) 2019-03-06
BR112018072849B1 (pt) 2020-12-22
WO2017197355A2 (en) 2017-11-16
CN116333057A (zh) 2023-06-27
NZ748395A (en) 2019-12-20
PT3445773T (pt) 2023-03-13
JP6592208B2 (ja) 2019-10-16
FI4206216T3 (fi) 2025-12-17
PT4206216T (pt) 2025-12-03
US20210177990A1 (en) 2021-06-17
CA3023592A1 (en) 2017-11-16
US11419949B2 (en) 2022-08-23
US20230211013A1 (en) 2023-07-06
KR102234930B1 (ko) 2021-04-02
CN109476707A (zh) 2019-03-15
US11364308B2 (en) 2022-06-21
PE20190129A1 (es) 2019-01-17
JP2019518458A (ja) 2019-07-04
US20230001018A1 (en) 2023-01-05
US20210069348A1 (en) 2021-03-11
UA124343C2 (uk) 2021-09-01
RU2018142768A (ru) 2020-06-16
CL2018003196A1 (es) 2019-02-15
MX2018013463A (es) 2019-07-04
EP4206216A1 (en) 2023-07-05
US11766487B2 (en) 2023-09-26
US20210162071A1 (en) 2021-06-03
DK4206216T3 (da) 2025-12-01
IL262922B (en) 2019-09-26
AU2017261812A1 (en) 2018-11-15
ES2941502T3 (es) 2023-05-23
CN115925999A (zh) 2023-04-07
EP3445773A2 (en) 2019-02-27
ES3059853T3 (en) 2026-03-24
US20260053947A1 (en) 2026-02-26
MX2023009627A (es) 2023-08-28
US20190255192A1 (en) 2019-08-22

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