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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
Authority
CR
Costa Rica
Prior art keywords
aav
methods
variant
virions
proteins
Prior art date
Application number
CR20180589A
Other languages
English (en)
Inventor
David; Schaffer
Melissa Kotterman
David H ; Kirn
Original Assignee
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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    • 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
    • A61K48/0008Medicinal 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
    • A61K48/0016Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/12Viral antigens
    • A61K39/235Adenoviridae
    • 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
    • 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
    • A61K48/0008Medicinal 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
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K48/0008Medicinal 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
    • A61K48/0025Medicinal 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
    • 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
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    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • 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
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
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    • 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

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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

Country Status (27)

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US (8) US11364308B2 (es)
EP (3) EP3445773B1 (es)
JP (1) JP6592208B2 (es)
KR (1) KR102234930B1 (es)
CN (3) CN115925999A (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 (1) DK3445773T5 (es)
ES (1) ES2941502T3 (es)
FI (1) 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 (1) 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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