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MX2018013463A - Capsides variantes de virus adenoasociados y metodos de uso de las mismas. - Google Patents

Capsides variantes de virus adenoasociados y metodos de uso de las mismas.

Info

Publication number
MX2018013463A
MX2018013463A MX2018013463A MX2018013463A MX2018013463A MX 2018013463 A MX2018013463 A MX 2018013463A MX 2018013463 A MX2018013463 A MX 2018013463A MX 2018013463 A MX2018013463 A MX 2018013463A MX 2018013463 A MX2018013463 A MX 2018013463A
Authority
MX
Mexico
Prior art keywords
aav
methods
virions
adeno
capsid protein
Prior art date
Application number
MX2018013463A
Other languages
English (en)
Inventor
H Kirn David
Kotterman Melissa
Schaffer David
Original Assignee
4D Molecular Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 4D Molecular Therapeutics Inc filed Critical 4D Molecular Therapeutics Inc
Publication of MX2018013463A publication Critical patent/MX2018013463A/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
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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
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
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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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Abstract

En el presente documento se proveen proteínas de la cápside del virus adenoasociado (AAV) variantes que tienen una o más modificaciones en la secuencia de aminoácidos en relación con una proteína de la cápside de AAV progenitora, que, cuando está presente en un virión de AAV, confiere un aumento de la infectividad de uno o más tipos de células de la retina en comparación con la infectividad de las células de la retina por un virión de AAV que comprende la proteína de la cápside de AAV progenitora no modificada. También se proveen viriones de AAV recombinantes y composiciones farmacéuticas de los mismos que comprenden una proteína de la cápside de AAV variante como se describe en el presente documento, los métodos para hacer estas proteínas de la cápside y viriones de de rAAV, y métodos para usar estas proteínas de la cápside y viriones de VAA en la investigación y en la práctica clínica, por ejemplo en el suministro de secuencias de ácido nucleico a una o más células de la retina para el tratamiento de trastornos y enfermedades de la retina.
MX2018013463A 2016-05-13 2017-05-12 Capsides variantes de virus adenoasociados y metodos de uso de las mismas. MX2018013463A (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
MX2018013463A true MX2018013463A (es) 2019-07-04

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MX2018013463A MX2018013463A (es) 2016-05-13 2017-05-12 Capsides variantes de virus adenoasociados y metodos de uso de las mismas.
MX2023009627A MX2023009627A (es) 2016-05-13 2018-11-05 Capsides variantes de virus adenoasociados y metodos de uso de las mismas.

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US (8) US11364308B2 (es)
EP (3) EP4206216B1 (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 (2) DK4206216T3 (es)
ES (1) ES2941502T3 (es)
FI (2) FI4206216T3 (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) PT4206216T (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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DK3445773T3 (da) 2023-03-20
US11364308B2 (en) 2022-06-21
EP4206216B1 (en) 2025-10-08
UA124343C2 (uk) 2021-09-01
DK3445773T5 (da) 2024-09-09
EP3445773A4 (en) 2019-10-02
PT4206216T (pt) 2025-12-03
JP2019518458A (ja) 2019-07-04
CN115925999A (zh) 2023-04-07
FI3445773T3 (fi) 2023-03-30
US11419949B2 (en) 2022-08-23
CN109476707B (zh) 2022-12-02
NZ748395A (en) 2019-12-20
US11766487B2 (en) 2023-09-26
US20210162071A1 (en) 2021-06-03
MX2023009627A (es) 2023-08-28
US20190255192A1 (en) 2019-08-22
CN109476707A (zh) 2019-03-15
JP6592208B2 (ja) 2019-10-16
WO2017197355A2 (en) 2017-11-16
SG11201809684YA (en) 2018-11-29
KR102234930B1 (ko) 2021-04-02
RU2018142768A (ru) 2020-06-16
AU2017261812B2 (en) 2019-04-18
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PH12018502387A1 (en) 2019-03-25
US11167041B2 (en) 2021-11-09
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US20210069348A1 (en) 2021-03-11
EP3445773A2 (en) 2019-02-27
PE20190129A1 (es) 2019-01-17
CA3023592A1 (en) 2017-11-16
CL2018003196A1 (es) 2019-02-15
KR20190005223A (ko) 2019-01-15
DK4206216T3 (da) 2025-12-01
EP3445773B1 (en) 2022-12-28
EP4206216A1 (en) 2023-07-05
US11179477B2 (en) 2021-11-23
PT3445773T (pt) 2023-03-13
CO2018013255A2 (es) 2018-12-14
MA44740A (fr) 2019-02-27
US20230001018A1 (en) 2023-01-05
EP4209501A1 (en) 2023-07-12
BR112018072849B1 (pt) 2020-12-22
RU2725286C2 (ru) 2020-06-30
BR112018072849A2 (pt) 2019-03-06
US20230211013A1 (en) 2023-07-06

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