MX2019005874A - Administracion intratecal de virus recombinante adeno-asociado que codifica la proteina 2 que une metilo-cpg. - Google Patents

Administracion intratecal de virus recombinante adeno-asociado que codifica la proteina 2 que une metilo-cpg.

Info

Publication number
MX2019005874A
MX2019005874A MX2019005874A MX2019005874A MX2019005874A MX 2019005874 A MX2019005874 A MX 2019005874A MX 2019005874 A MX2019005874 A MX 2019005874A MX 2019005874 A MX2019005874 A MX 2019005874A MX 2019005874 A MX2019005874 A MX 2019005874A
Authority
MX
Mexico
Prior art keywords
binding protein
associated virus
recombinant adeno
intrathecal delivery
cpg binding
Prior art date
Application number
MX2019005874A
Other languages
English (en)
Inventor
k kaspar Brian
Foust Kevin
Original Assignee
Nationwide Childrens Hospital 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 Nationwide Childrens Hospital Inc filed Critical Nationwide Childrens Hospital Inc
Publication of MX2019005874A publication Critical patent/MX2019005874A/es

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • 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
    • 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
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • 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
    • 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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Abstract

Se proporcionan métodos y materiales para la administración intratecal del virus recombinante adeno-asociado 9 (rAAV9) que codifica la proteína 2 de unión a metil-CpG (MECP2). Se contempla el uso de los métodos y materiales, por ejemplo, para el tratamiento del síndrome de Rett.
MX2019005874A 2016-11-17 2017-11-17 Administracion intratecal de virus recombinante adeno-asociado que codifica la proteina 2 que une metilo-cpg. MX2019005874A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662423618P 2016-11-17 2016-11-17
PCT/US2017/062371 WO2018094251A1 (en) 2016-11-17 2017-11-17 Intrathecal delivery of recombinant adeno-associated virus encoding methyl-cpg binding protein 2

Publications (1)

Publication Number Publication Date
MX2019005874A true MX2019005874A (es) 2020-02-07

Family

ID=62145776

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2019005874A MX2019005874A (es) 2016-11-17 2017-11-17 Administracion intratecal de virus recombinante adeno-asociado que codifica la proteina 2 que une metilo-cpg.

Country Status (15)

Country Link
US (2) US11583564B2 (es)
EP (1) EP3541429A4 (es)
JP (3) JP7106534B2 (es)
KR (1) KR102641846B1 (es)
CN (1) CN110325219B (es)
AU (2) AU2017362491B2 (es)
BR (1) BR112019009834B1 (es)
CA (1) CA3044291A1 (es)
CL (1) CL2019001354A1 (es)
IL (1) IL266678A (es)
MA (1) MA46863A (es)
MX (1) MX2019005874A (es)
SA (1) SA519401806B1 (es)
SG (1) SG10202105090WA (es)
WO (1) WO2018094251A1 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2019005874A (es) * 2016-11-17 2020-02-07 Nationwide Childrens Hospital Inc Administracion intratecal de virus recombinante adeno-asociado que codifica la proteina 2 que une metilo-cpg.
EA202192160A1 (ru) * 2019-02-04 2021-11-17 Рисерч Инститьют Эт Нэшнуайд Чилдрен'С Хоспитал Доставка полинуклеотида cln3 с помощью аденоассоциированного вируса
AU2020366242A1 (en) * 2019-10-18 2022-05-26 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of disorders associated mutations in the IRF2BPL gene
JP2024515623A (ja) 2021-04-13 2024-04-10 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 髄腔内送達によってピット・ホプキンス症候群を治療するためのメチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルス

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Also Published As

Publication number Publication date
CN110325219A (zh) 2019-10-11
CN110325219B (zh) 2024-03-26
MA46863A (fr) 2019-09-25
EP3541429A4 (en) 2020-08-12
CA3044291A1 (en) 2018-05-24
US11583564B2 (en) 2023-02-21
EP3541429A1 (en) 2019-09-25
WO2018094251A1 (en) 2018-05-24
CL2019001354A1 (es) 2019-09-13
BR112019009834A2 (pt) 2019-10-08
KR20190126047A (ko) 2019-11-08
US20230321164A1 (en) 2023-10-12
RU2019118300A3 (es) 2021-01-11
KR102641846B1 (ko) 2024-02-27
JP2024045289A (ja) 2024-04-02
SA519401806B1 (ar) 2023-03-08
AU2017362491B2 (en) 2023-02-02
BR112019009834B1 (pt) 2022-05-17
AU2017362491A1 (en) 2019-06-06
IL266678A (en) 2019-07-31
AU2023202598A1 (en) 2023-07-20
JP2020503265A (ja) 2020-01-30
JP2022137210A (ja) 2022-09-21
US20200179467A1 (en) 2020-06-11
RU2019118300A (ru) 2020-12-17
JP7106534B2 (ja) 2022-07-26
SG10202105090WA (en) 2021-06-29

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