BR112019009834A2 - liberação intratecal de vírus adeno-associado recombinante que codifica a proteína de ligação metil-cpg 2 - Google Patents

liberação intratecal de vírus adeno-associado recombinante que codifica a proteína de ligação metil-cpg 2

Info

Publication number
BR112019009834A2
BR112019009834A2 BR112019009834A BR112019009834A BR112019009834A2 BR 112019009834 A2 BR112019009834 A2 BR 112019009834A2 BR 112019009834 A BR112019009834 A BR 112019009834A BR 112019009834 A BR112019009834 A BR 112019009834A BR 112019009834 A2 BR112019009834 A2 BR 112019009834A2
Authority
BR
Brazil
Prior art keywords
binding protein
associated virus
recombinant adeno
virus encoding
cpg binding
Prior art date
Application number
BR112019009834A
Other languages
English (en)
Other versions
BR112019009834B1 (pt
Inventor
k kaspar Brian
Foust Kevin
Original Assignee
Nationwide Childrens Hospital Inc
Ohio State Innovation Foundation
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, Ohio State Innovation Foundation filed Critical Nationwide Childrens Hospital Inc
Publication of BR112019009834A2 publication Critical patent/BR112019009834A2/pt
Publication of BR112019009834B1 publication Critical patent/BR112019009834B1/pt

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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
    • 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
    • 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
    • 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
    • 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
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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
    • C12N2750/14141—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
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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
    • C12N2750/14141—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)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Toxicology (AREA)
  • General Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Mycology (AREA)
  • Psychology (AREA)
  • Dermatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

métodos e materiais para a liberação intratecal de vírus adeno-associado recombinante 9 (raav9) que codifica a proteína de ligação metil-cpg 2 (mecp2) são fornecidos. o uso dos métodos e materiais é contemplado, por exemplo, para o tratamento da síndrome de rett.
BR112019009834-8A 2016-11-17 2017-11-17 Vetor viral adeno-associado recombinante (raav), vírus, composição farmacêutica e uso de um vetor raav BR112019009834B1 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662423618P 2016-11-17 2016-11-17
US62/423,618 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 (2)

Publication Number Publication Date
BR112019009834A2 true BR112019009834A2 (pt) 2019-10-08
BR112019009834B1 BR112019009834B1 (pt) 2022-05-17

Family

ID=62145776

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112019009834-8A BR112019009834B1 (pt) 2016-11-17 2017-11-17 Vetor viral adeno-associado recombinante (raav), vírus, composição farmacêutica e uso de um vetor raav

Country Status (15)

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

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.
CA3129077A1 (en) * 2019-02-04 2020-08-13 Research Institute At Nationwide Children's Hospital Adeno-associated virus delivery of cln3 polynucleotide
US20220389453A1 (en) * 2019-10-18 2022-12-08 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of disorders associated with 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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ATE272123T1 (de) 1993-11-09 2004-08-15 Ohio Med College Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
RU2273645C9 (ru) 1994-08-17 2006-11-27 Дзе Рокефеллер Юниверсити Полипептид ожирения (ов)(варианты), его аналог (варианты) и слитый белок (варианты), изолированная молекула нуклеиновой кислоты, молекула днк, рекомбинантный вектор клонирования, рекомбинантный вектор экспрессии, фармацевтическая композиция, моноклональное и поликлональное антитело
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KR20200015701A (ko) * 2017-06-06 2020-02-12 유니버시티 오브 매사추세츠 레트 증후군에서의 MeCP2의 안전한 발현을 위한 자가-조절 AAV 벡터

Also Published As

Publication number Publication date
AU2017362491A1 (en) 2019-06-06
MX2019005874A (es) 2020-02-07
AU2017362491B2 (en) 2023-02-02
KR20190126047A (ko) 2019-11-08
AU2023202598A1 (en) 2023-07-20
RU2019118300A3 (pt) 2021-01-11
CA3044291A1 (en) 2018-05-24
MA46863A (fr) 2019-09-25
US11583564B2 (en) 2023-02-21
JP2020503265A (ja) 2020-01-30
EP3541429A1 (en) 2019-09-25
JP2024045289A (ja) 2024-04-02
BR112019009834B1 (pt) 2022-05-17
SA519401806B1 (ar) 2023-03-08
US20230321164A1 (en) 2023-10-12
KR102641846B1 (ko) 2024-02-27
JP7106534B2 (ja) 2022-07-26
EP3541429A4 (en) 2020-08-12
US20200179467A1 (en) 2020-06-11
WO2018094251A1 (en) 2018-05-24
IL266678A (en) 2019-07-31
SG10202105090WA (en) 2021-06-29
CN110325219B (zh) 2024-03-26
JP2022137210A (ja) 2022-09-21
CL2019001354A1 (es) 2019-09-13
RU2019118300A (ru) 2020-12-17
CN110325219A (zh) 2019-10-11

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