SA519401806B1 - توصيل داخل القراب لفيروس مرتبط غديا ناتج عودة الارتباط الجيني يرمز بروتين 2 ربط ميثيل -cpg - Google Patents

توصيل داخل القراب لفيروس مرتبط غديا ناتج عودة الارتباط الجيني يرمز بروتين 2 ربط ميثيل -cpg Download PDF

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SA519401806B1
SA519401806B1 SA519401806A SA519401806A SA519401806B1 SA 519401806 B1 SA519401806 B1 SA 519401806B1 SA 519401806 A SA519401806 A SA 519401806A SA 519401806 A SA519401806 A SA 519401806A SA 519401806 B1 SA519401806 B1 SA 519401806B1
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associated virus
mecp2
vector
aav
virus
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SA519401806A
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Arabic (ar)
English (en)
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كيه. كاسبر براين
فوست كيفين
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أوهايو ستيت إنوفيشين فاونديشين
ناشنوايد تشيلدرينز هوسبيتال إنك.
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Publication of SA519401806B1 publication Critical patent/SA519401806B1/ar

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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
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • 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)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
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  • Biomedical Technology (AREA)
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  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (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)
SA519401806A 2016-11-17 2019-05-17 توصيل داخل القراب لفيروس مرتبط غديا ناتج عودة الارتباط الجيني يرمز بروتين 2 ربط ميثيل -cpg SA519401806B1 (ar)

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US201662423618P 2016-11-17 2016-11-17

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SA519401806B1 true SA519401806B1 (ar) 2023-03-08

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US (3) US11583564B2 (enExample)
EP (1) EP3541429A4 (enExample)
JP (3) JP7106534B2 (enExample)
KR (1) KR102641846B1 (enExample)
CN (2) CN110325219B (enExample)
AU (2) AU2017362491B2 (enExample)
BR (1) BR112019009834B1 (enExample)
CA (1) CA3044291A1 (enExample)
CL (1) CL2019001354A1 (enExample)
IL (2) IL322146A (enExample)
MA (1) MA46863A (enExample)
MX (1) MX2019005874A (enExample)
SA (1) SA519401806B1 (enExample)
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CA3044291A1 (en) * 2016-11-17 2018-05-24 Nationwide Children's Hospital Inc. Intrathecal delivery of recombinant adeno-associated virus encoding methyl-cpg binding protein 2
CN120505370A (zh) 2017-11-08 2025-08-19 诺华股份有限公司 制备病毒载体的手段和方法及其用途
EP3801638A1 (en) 2018-06-08 2021-04-14 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
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
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
WO2022221424A1 (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
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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SG10202105090WA (en) 2021-06-29
CA3044291A1 (en) 2018-05-24
US20250312400A1 (en) 2025-10-09
JP2022137210A (ja) 2022-09-21
MX2019005874A (es) 2020-02-07
BR112019009834A2 (pt) 2019-10-08
AU2017362491A1 (en) 2019-06-06
CN118490845A (zh) 2024-08-16
EP3541429A1 (en) 2019-09-25
MA46863A (fr) 2019-09-25
RU2019118300A (ru) 2020-12-17
JP2024045289A (ja) 2024-04-02
IL266678B2 (en) 2025-12-01
CN110325219A (zh) 2019-10-11
IL266678B1 (en) 2025-08-01
US11583564B2 (en) 2023-02-21
CN110325219B (zh) 2024-03-26
IL266678A (en) 2019-07-31
US20200179467A1 (en) 2020-06-11
US12357663B2 (en) 2025-07-15
KR20190126047A (ko) 2019-11-08
AU2017362491B2 (en) 2023-02-02
JP2020503265A (ja) 2020-01-30
CL2019001354A1 (es) 2019-09-13
KR102641846B1 (ko) 2024-02-27
EP3541429A4 (en) 2020-08-12
RU2019118300A3 (enExample) 2021-01-11
US20230321164A1 (en) 2023-10-12
BR112019009834B1 (pt) 2022-05-17
JP7106534B2 (ja) 2022-07-26
WO2018094251A1 (en) 2018-05-24
AU2023202598A1 (en) 2023-07-20
IL322146A (en) 2025-09-01

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