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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Publication number
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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Prior art keywords
mecp2
vector
raav
avxs
virus
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AU2017362491A
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AU2017362491A1 (en
Inventor
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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Publication of AU2017362491B2 publication Critical patent/AU2017362491B2/en
Priority to AU2023202598A priority Critical patent/AU2023202598B2/en
Priority to AU2026200191A priority patent/AU2026200191A1/en
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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

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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)
  • Public Health (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Wood Science & Technology (AREA)
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  • 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 (2)

Application Number Priority Date Filing Date Title
AU2023202598A AU2023202598B2 (en) 2016-11-17 2023-04-28 Intrathecal delivery of recombinant Adeno-associated virus encoding Methyl-CpG binding protein 2
AU2026200191A AU2026200191A1 (en) 2016-11-17 2026-01-13 Intrathecal delivery of recombinant Adeno-associated virus encoding Methyl-CpG binding protein 2

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

Related Child Applications (1)

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AU2023202598A Division AU2023202598B2 (en) 2016-11-17 2023-04-28 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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AU2017362491A Active AU2017362491B2 (en) 2016-11-17 2017-11-17 Intrathecal delivery of recombinant Adeno-associated virus encoding Methyl-CpG binding protein 2
AU2023202598A Active AU2023202598B2 (en) 2016-11-17 2023-04-28 Intrathecal delivery of recombinant Adeno-associated virus encoding Methyl-CpG binding protein 2
AU2026200191A Pending AU2026200191A1 (en) 2016-11-17 2026-01-13 Intrathecal delivery of recombinant Adeno-associated virus encoding Methyl-CpG binding protein 2

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AU2023202598A Active AU2023202598B2 (en) 2016-11-17 2023-04-28 Intrathecal delivery of recombinant Adeno-associated virus encoding Methyl-CpG binding protein 2
AU2026200191A Pending AU2026200191A1 (en) 2016-11-17 2026-01-13 Intrathecal delivery of recombinant Adeno-associated virus encoding Methyl-CpG binding protein 2

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US (3) US11583564B2 (https=)
EP (1) EP3541429A4 (https=)
JP (3) JP7106534B2 (https=)
KR (1) KR102641846B1 (https=)
CN (2) CN118490845A (https=)
AU (3) AU2017362491B2 (https=)
BR (1) BR112019009834B1 (https=)
CA (1) CA3044291A1 (https=)
CL (1) CL2019001354A1 (https=)
IL (2) IL266678B2 (https=)
MA (1) MA46863A (https=)
MX (1) MX2019005874A (https=)
SA (1) SA519401806B1 (https=)
SG (1) SG10202105090WA (https=)
WO (1) WO2018094251A1 (https=)

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MX2020004830A (es) 2017-11-08 2020-11-11 Avexis Inc Medios y metodo para preparar vectores virales y usos de los mismos.
CN112601557B (zh) 2018-06-08 2025-06-06 诺华股份有限公司 用于测量药物产品效力的基于细胞的测定
KR20210124300A (ko) * 2019-02-04 2021-10-14 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 Cln3 폴리뉴클레오티드의 아데노-관련 바이러스 전달
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
JP7684296B2 (ja) * 2019-11-22 2025-05-27 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 Ｉｇｈｍｂｐ２遺伝子に関連する障害の治療のための材料および方法
EP4323010A1 (en) * 2021-04-13 2024-02-21 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
EP4715058A1 (en) * 2023-05-15 2026-03-25 Genecombio Ltd. Gene therapy for rett syndrome

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IL266678B1 (en) 2025-08-01
AU2017362491A1 (en) 2019-06-06
BR112019009834B1 (pt) 2022-05-17
KR102641846B1 (ko) 2024-02-27
JP2020503265A (ja) 2020-01-30
US20250312400A1 (en) 2025-10-09
CN110325219A (zh) 2019-10-11
EP3541429A4 (en) 2020-08-12
WO2018094251A1 (en) 2018-05-24
AU2023202598B2 (en) 2026-02-05
CL2019001354A1 (es) 2019-09-13
US11583564B2 (en) 2023-02-21
SA519401806B1 (ar) 2023-03-08
SG10202105090WA (en) 2021-06-29
EP3541429A1 (en) 2019-09-25
JP2022137210A (ja) 2022-09-21
US12357663B2 (en) 2025-07-15
IL266678A (en) 2019-07-31
RU2019118300A (ru) 2020-12-17
IL266678B2 (en) 2025-12-01
BR112019009834A2 (pt) 2019-10-08
MX2019005874A (es) 2020-02-07
JP2024045289A (ja) 2024-04-02
CN118490845A (zh) 2024-08-16
MA46863A (fr) 2019-09-25
CN110325219B (zh) 2024-03-26
CA3044291A1 (en) 2018-05-24
AU2026200191A1 (en) 2026-01-29
AU2023202598A1 (en) 2023-07-20
US20200179467A1 (en) 2020-06-11
IL322146A (en) 2025-09-01
US20230321164A1 (en) 2023-10-12
KR20190126047A (ko) 2019-11-08
JP7106534B2 (ja) 2022-07-26
RU2019118300A3 (https=) 2021-01-11

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