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

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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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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • 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
    • A61K48/005Medicinal 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0085Brain, e.g. brain implants; Spinal cord
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14132Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Neurosurgery (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Engineering & Computer Science (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Mycology (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
BR112019009834B1 (pt) * 2016-11-17 2022-05-17 Ohio State Innovation Foundation Vetor viral adeno-associado recombinante (raav), vírus, composição farmacêutica e uso de um vetor raav
WO2020163300A1 (en) * 2019-02-04 2020-08-13 Research Institute At Nationwide Children's Hospital Adeno-associated virus delivery of cln3 polynucleotide
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
CA3216711A1 (en) 2021-04-13 2022-10-20 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

Family Cites Families (33)

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AU678867B2 (en) 1993-11-09 1997-06-12 Medical College Of Ohio, The Stable cell lines capable of expressing the adeno-associated virus replication gene
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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AU707866B2 (en) 1994-12-06 1999-07-22 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
EP0847442A1 (en) 1995-08-30 1998-06-17 Genzyme Corporation Chromatographic purification of adenovirus and aav
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BR112019009834B1 (pt) * 2016-11-17 2022-05-17 Ohio State Innovation Foundation Vetor viral adeno-associado recombinante (raav), vírus, composição farmacêutica e uso de um vetor raav
EA201992882A1 (ru) * 2017-06-06 2020-05-25 Юниверсити Оф Массачусеттс САМОРЕГУЛИРУЕМЫЕ ВЕКТОРЫ НА ОСНОВЕ AAV ДЛЯ БЕЗОПАСНОЙ ЭКСПРЕССИИ MeCP2 ПРИ СИНДРОМЕ PETTA

Also Published As

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

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B07E Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette]
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B09A Decision: intention to grant [chapter 9.1 patent gazette]
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Free format text: PRAZO DE VALIDADE: 20 (VINTE) ANOS CONTADOS A PARTIR DE 17/11/2017, OBSERVADAS AS CONDICOES LEGAIS