AU2017362491B2 - Intrathecal delivery of recombinant Adeno-associated virus encoding Methyl-CpG binding protein 2 - Google Patents

Intrathecal delivery of recombinant Adeno-associated virus encoding Methyl-CpG binding protein 2 Download PDF

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AU2017362491B2
AU2017362491B2 AU2017362491A AU2017362491A AU2017362491B2 AU 2017362491 B2 AU2017362491 B2 AU 2017362491B2 AU 2017362491 A AU2017362491 A AU 2017362491A AU 2017362491 A AU2017362491 A AU 2017362491A AU 2017362491 B2 AU2017362491 B2 AU 2017362491B2
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mecp2
vector
raav
avxs
virus
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AU2017362491A1 (en
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Kevin Foust
Brian K. Kaspar
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Nationwide Childrens Hospital Inc
Ohio State Innovation Foundation
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Nationwide Childrens Hospital Inc
Ohio State Innovation Foundation
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Assigned to NATIONWIDE CHILDREN'S HOSPITAL INC., OHIO STATE INNOVATION FOUNDATION reassignment NATIONWIDE CHILDREN'S HOSPITAL INC. Request for Assignment Assignors: FOUST, KEVIN, NATIONWIDE CHILDREN'S HOSPITAL INC.
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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
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    • 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
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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
    • C12N2750/14132—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
    • 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

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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)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • 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)
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  • Marine Sciences & Fisheries (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
AU2017362491A 2016-11-17 2017-11-17 Intrathecal delivery of recombinant Adeno-associated virus encoding Methyl-CpG binding protein 2 Active AU2017362491B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2023202598A AU2023202598A1 (en) 2016-11-17 2023-04-28 Intrathecal delivery of recombinant Adeno-associated virus encoding Methyl-CpG binding protein 2

Applications Claiming Priority (3)

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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

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AU2017362491A1 AU2017362491A1 (en) 2019-06-06
AU2017362491B2 true AU2017362491B2 (en) 2023-02-02

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US (3) US11583564B2 (OSRAM)
EP (1) EP3541429A4 (OSRAM)
JP (3) JP7106534B2 (OSRAM)
KR (1) KR102641846B1 (OSRAM)
CN (2) CN110325219B (OSRAM)
AU (2) AU2017362491B2 (OSRAM)
BR (1) BR112019009834B1 (OSRAM)
CA (1) CA3044291A1 (OSRAM)
CL (1) CL2019001354A1 (OSRAM)
IL (2) IL322146A (OSRAM)
MA (1) MA46863A (OSRAM)
MX (1) MX2019005874A (OSRAM)
SA (1) SA519401806B1 (OSRAM)
SG (1) SG10202105090WA (OSRAM)
WO (1) WO2018094251A1 (OSRAM)

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EP3707264A1 (en) 2017-11-08 2020-09-16 Avexis Inc. Means and method for preparing viral vectors and uses of same
US12228571B2 (en) 2018-06-08 2025-02-18 Novartis Ag Cell-based assay for measuring drug product potency
TW202045530A (zh) * 2019-02-04 2020-12-16 美國全美兒童醫院之研究學會 腺相關病毒對ｃｌｎ３聚核苷酸的遞送
CA3158131A1 (en) * 2019-10-18 2021-04-22 Nicolas Sebastien Wein Materials and methods for the treatment of disorders associated mutations in the irf2bpl gene
EP4061831A1 (en) * 2019-11-22 2022-09-28 Research Institute at Nationwide Children's Hospital Materials and methods for treatment of disorders associated with the ighmbp2 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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SG10202105090WA (en) 2021-06-29
US20230321164A1 (en) 2023-10-12
BR112019009834B1 (pt) 2022-05-17
JP7106534B2 (ja) 2022-07-26
EP3541429A1 (en) 2019-09-25
JP2022137210A (ja) 2022-09-21
IL266678B2 (en) 2025-12-01
RU2019118300A3 (OSRAM) 2021-01-11
WO2018094251A1 (en) 2018-05-24
US20250312400A1 (en) 2025-10-09
MA46863A (fr) 2019-09-25
EP3541429A4 (en) 2020-08-12
US20200179467A1 (en) 2020-06-11
CL2019001354A1 (es) 2019-09-13
US12357663B2 (en) 2025-07-15
CN110325219B (zh) 2024-03-26
US11583564B2 (en) 2023-02-21
IL266678B1 (en) 2025-08-01
CN110325219A (zh) 2019-10-11
CA3044291A1 (en) 2018-05-24
KR102641846B1 (ko) 2024-02-27
IL322146A (en) 2025-09-01
JP2020503265A (ja) 2020-01-30
SA519401806B1 (ar) 2023-03-08
MX2019005874A (es) 2020-02-07
JP2024045289A (ja) 2024-04-02
RU2019118300A (ru) 2020-12-17
IL266678A (en) 2019-07-31
KR20190126047A (ko) 2019-11-08
AU2023202598A1 (en) 2023-07-20
BR112019009834A2 (pt) 2019-10-08
AU2017362491A1 (en) 2019-06-06
CN118490845A (zh) 2024-08-16

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