CR20180589A - Cápsulas variantes de virus adenoasociados y métodos de uso de estas - Google Patents

Cápsulas variantes de virus adenoasociados y métodos de uso de estas

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Publication number
CR20180589A
CR20180589A CR20180589A CR20180589A CR20180589A CR 20180589 A CR20180589 A CR 20180589A CR 20180589 A CR20180589 A CR 20180589A CR 20180589 A CR20180589 A CR 20180589A CR 20180589 A CR20180589 A CR 20180589A
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Costa Rica
Prior art keywords
aav
methods
variant
virions
proteins
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Application number
CR20180589A
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English (en)
Inventor
David; Schaffer
Melissa Kotterman
David H ; Kirn
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4D Molecular Therapeutics Inc
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Publication date
Application filed by 4D Molecular Therapeutics Inc filed Critical 4D Molecular Therapeutics Inc
Publication of CR20180589A publication Critical patent/CR20180589A/es

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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
    • 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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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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    • A—HUMAN NECESSITIES
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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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    • C12N15/00—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
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    • C07—ORGANIC CHEMISTRY
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    • C07K2319/00—Fusion polypeptide
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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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    • 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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    • C12N2750/00011—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)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
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Abstract

En la presente se proporcionan proteínas de la cápside del virus adenoasociado (AAV) variante que tienen una o más modificaciones en la secuencia de aminoácidos en relación con una proteína de la cápside de AAV parental, que, cuando está presente en un virión de AAV, confiere un aumento de la infectividad de uno o más tipos de células retinianas en comparación con la infectividad de las células retinianas por un virión de AAV que comprende la proteína de cápside de AAV parental no modificada. También se proporcionan viriones de AAV recombinantes y composiciones farmacéuticas de estos que comprenden una proteína de la cápside de AAV variante como se describe en la presente, métodos para producir estos viriones y proteínas de la cápside de rAAV, y métodos para usar estos viriones y proteínas de la cápside de rAAV en la investigación y en la práctica clínica, por ejemplo, en la administración de secuencias de ácido nucleico a una o más células de la retina para el tratamiento de trastornos y enfermedades de la retina
CR20180589A 2016-05-13 2017-05-12 Cápsulas variantes de virus adenoasociados y métodos de uso de estas CR20180589A (es)

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 (1)

Publication Number Publication Date
CR20180589A true CR20180589A (es) 2019-02-27

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CR20180589A CR20180589A (es) 2016-05-13 2017-05-12 Cápsulas variantes de virus adenoasociados y métodos de uso de estas

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

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KR20190005223A (ko) 2019-01-15
MA44740A (fr) 2019-02-27
EP3445773B1 (en) 2022-12-28
DK3445773T3 (da) 2023-03-20
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 (es) 2020-06-16
US20240066145A1 (en) 2024-02-29
US11576983B2 (en) 2023-02-14
ZA201905485B (en) 2021-03-31
DK3445773T5 (da) 2024-09-09
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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