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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
cpg
adeno
recombinant virus
administration
associated recombinant
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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    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K48/005—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
    • A61K48/0075—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
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Neurology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Dermatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Mycology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)

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 (3) US11583564B2 (es)
EP (1) EP3541429A4 (es)
JP (3) JP7106534B2 (es)
KR (1) KR102641846B1 (es)
CN (2) CN118490845A (es)
AU (3) AU2017362491B2 (es)
BR (1) BR112019009834B1 (es)
CA (1) CA3044291A1 (es)
CL (1) CL2019001354A1 (es)
IL (2) IL322146A (es)
MA (1) MA46863A (es)
MX (1) MX2019005874A (es)
SA (1) SA519401806B1 (es)
SG (1) SG10202105090WA (es)
WO (1) WO2018094251A1 (es)

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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.
CN111566220A (zh) 2017-11-08 2020-08-21 阿维克斯公司 制备病毒载体的手段和方法及其用途
JP7478675B2 (ja) 2018-06-08 2024-05-07 ノバルティス アーゲー 薬物製品の効力を測定するための細胞ベースアッセイ
AU2020217708B2 (en) * 2019-02-04 2026-01-22 Ohio State Innovation Foundation Adeno-associated virus delivery of CLN3 polynucleotide
JP7781057B2 (ja) * 2019-10-18 2025-12-05 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 Ｉｒｆ２ｂｐｌ遺伝子の変異に関連する障害の治療のための材料および方法
AU2020385387A1 (en) * 2019-11-22 2022-06-02 Fondazione Irccs Ca' Granda Ospedale Maggiore Foliclinico Materials and methods for treatment of disorders associated with the IGHMBP2 gene
US20240189452A1 (en) 2021-04-13 2024-06-13 Research Institute At Nationwide Children's Hospital Recombinant Adeno-Associated Virus Encoding Methyl-CPG Binding Protein 2 for Treating PITT Hopkins Syndrome VIA Intrathecal Delivery
WO2024235164A1 (zh) * 2023-05-15 2024-11-21 上海金珂博生物技术有限公司 一种Rett综合症的基因治疗

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Also Published As

Publication number Publication date
US20250312400A1 (en) 2025-10-09
KR102641846B1 (ko) 2024-02-27
AU2023202598A1 (en) 2023-07-20
IL322146A (en) 2025-09-01
JP7106534B2 (ja) 2022-07-26
EP3541429A1 (en) 2019-09-25
MA46863A (fr) 2019-09-25
CN110325219B (zh) 2024-03-26
JP2022137210A (ja) 2022-09-21
CL2019001354A1 (es) 2019-09-13
RU2019118300A3 (es) 2021-01-11
CN118490845A (zh) 2024-08-16
WO2018094251A1 (en) 2018-05-24
KR20190126047A (ko) 2019-11-08
AU2026200191A1 (en) 2026-01-29
CN110325219A (zh) 2019-10-11
IL266678B1 (en) 2025-08-01
US12357663B2 (en) 2025-07-15
RU2019118300A (ru) 2020-12-17
AU2017362491A1 (en) 2019-06-06
BR112019009834B1 (pt) 2022-05-17
US11583564B2 (en) 2023-02-21
CA3044291A1 (en) 2018-05-24
IL266678A (en) 2019-07-31
IL266678B2 (en) 2025-12-01
JP2020503265A (ja) 2020-01-30
BR112019009834A2 (pt) 2019-10-08
AU2017362491B2 (en) 2023-02-02
AU2023202598B2 (en) 2026-02-05
US20230321164A1 (en) 2023-10-12
EP3541429A4 (en) 2020-08-12
SA519401806B1 (ar) 2023-03-08
SG10202105090WA (en) 2021-06-29
JP2024045289A (ja) 2024-04-02
US20200179467A1 (en) 2020-06-11

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