HUE063458T2 - Az Otoferlin gént helyreállító, AAV által közvetített génterápia - Google Patents

Az Otoferlin gént helyreállító, AAV által közvetített génterápia

Info

Publication number
HUE063458T2
HUE063458T2 HUE20701951A HUE20701951A HUE063458T2 HU E063458 T2 HUE063458 T2 HU E063458T2 HU E20701951 A HUE20701951 A HU E20701951A HU E20701951 A HUE20701951 A HU E20701951A HU E063458 T2 HUE063458 T2 HU E063458T2
Authority
HU
Hungary
Prior art keywords
aav
gene
otoferlin
gene therapy
mediated
Prior art date
Application number
HUE20701951A
Other languages
English (en)
Hungarian (hu)
Inventor
Saaid Safieddine
Christine Petit
Original Assignee
Pasteur Institut
Centre National De La Rech
Inst Nat Sante Rech Med
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=65351986&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=HUE063458(T2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Pasteur Institut, Centre National De La Rech, Inst Nat Sante Rech Med filed Critical Pasteur Institut
Publication of HUE063458T2 publication Critical patent/HUE063458T2/hu

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276Knock-out vertebrates
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/16Otologicals
    • 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
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/05Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/054Animals comprising random inserted nucleic acids (transgenic) inducing loss of function
    • A01K2217/056Animals comprising random inserted nucleic acids (transgenic) inducing loss of function due to mutation of coding region of the transgene (dominant negative)
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/075Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/20Animal model comprising regulated expression system
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0306Animal model for genetic diseases
    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N2750/14144Chimeric viral vector comprising heterologous viral elements for production of another viral vector
    • 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
    • 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
    • C12N2750/14171Demonstrated in vivo effect

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Environmental Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Cell Biology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
HUE20701951A 2019-01-18 2020-01-20 Az Otoferlin gént helyreállító, AAV által közvetített génterápia HUE063458T2 (hu)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP19305071 2019-01-18

Publications (1)

Publication Number Publication Date
HUE063458T2 true HUE063458T2 (hu) 2024-01-28

Family

ID=65351986

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE20701951A HUE063458T2 (hu) 2019-01-18 2020-01-20 Az Otoferlin gént helyreállító, AAV által közvetített génterápia

Country Status (22)

Country Link
US (1) US20220125875A1 (enExample)
EP (2) EP3911354B8 (enExample)
JP (2) JP7595013B2 (enExample)
KR (1) KR20210118112A (enExample)
CN (1) CN113924109A (enExample)
AU (1) AU2020208933B2 (enExample)
BR (1) BR112021013996A2 (enExample)
CA (1) CA3126884A1 (enExample)
DK (1) DK3911354T5 (enExample)
ES (1) ES2960880T3 (enExample)
FI (1) FI3911354T3 (enExample)
HR (1) HRP20231296T1 (enExample)
HU (1) HUE063458T2 (enExample)
IL (1) IL284742B2 (enExample)
LT (1) LT3911354T (enExample)
MX (1) MX2021008622A (enExample)
PL (1) PL3911354T3 (enExample)
PT (1) PT3911354T (enExample)
RS (1) RS64794B1 (enExample)
SI (1) SI3911354T1 (enExample)
SM (1) SMT202300359T1 (enExample)
WO (1) WO2020148458A1 (enExample)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2018261769B2 (en) 2017-05-05 2024-03-14 The Regents Of The University Of California Compositions and methods for expressing Otoferlin
CN119410636A (zh) 2018-04-27 2025-02-11 分贝治疗公司 肌球蛋白15启动子及其用途
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
EP3921032A4 (en) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
WO2020163761A1 (en) 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
BR112022016596A2 (pt) 2020-02-21 2022-11-16 Akouos Inc Composições e métodos para o tratamento de debilitação auditiva não associada à idade em um indivíduo humano
CN115666658A (zh) 2020-04-01 2023-01-31 佛罗里达州大学研究基金会 具有提高的治疗ush1b的安全性的双aav-myo7a载体
CA3197592A1 (en) * 2020-11-06 2022-05-12 Steven Pennock Variant adeno-associated virus (aav)capsid polypeptides and gene therapeutics thereof for treatment of hearing loss
MX2023009714A (es) * 2021-02-19 2023-10-03 Decibel Therapeutics Inc Metodos para el tratamiento de la perdida auditiva neurosensorial mediante el uso de sistemas de vectores duales de otoferlina.
MX2024003052A (es) * 2021-09-10 2024-05-14 Pasteur Institut Sistema de vectores aav8 recombinante dual que codifica la isoforma 5 de otoferlina y sus usos.
CN117106824B (zh) * 2022-05-17 2025-09-02 上海佑音医药生物科技有限公司 一种治疗听力损伤的双载体系统及其应用
EP4527933A4 (en) * 2022-05-17 2025-12-24 Eye & Ent Hospital Of Fudan Univ DUAL-SUPPORT SYSTEM FOR THE TREATMENT OF HEARING LOSS, AND ITS USE
CN116925239B (zh) * 2023-07-17 2024-10-18 苏州星奥拓维生物技术有限公司 双载体系统表达Otof基因的组合物和方法
CN117305367A (zh) * 2023-08-21 2023-12-29 复旦大学附属眼耳鼻喉科医院 一种表达全长耳畸蛋白的双aav载体系统及其应用

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1453547B1 (en) 2001-12-17 2016-09-21 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
AU2003209226A1 (en) 2002-01-11 2003-07-30 Michael Tennant Adiponectin gene therapy
WO2006036465A2 (en) 2004-09-03 2006-04-06 University Of Florida Compositions and methods for treating cystic fibrosis
US8298818B2 (en) 2006-04-28 2012-10-30 University Of Florida Research Foundation, Inc. Self-complementary adeno-associated virus having a truncated CMV-chicken β-actin promoter
WO2011075838A1 (en) 2009-12-21 2011-06-30 Audigen Inc. Method of treating or preventing hearing loss
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
KR102649543B1 (ko) * 2013-05-21 2024-03-21 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도
EP3386537B1 (en) 2015-12-11 2021-10-20 Massachusetts Eye and Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
EP4219724A3 (en) * 2016-08-23 2023-09-27 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
AU2018261769B2 (en) 2017-05-05 2024-03-14 The Regents Of The University Of California Compositions and methods for expressing Otoferlin
EA202091995A1 (ru) * 2018-02-22 2021-03-30 Акуос, Инк. Композиции и способы лечения не связанного с возрастом нарушения слуха у субъекта-человека
US11660353B2 (en) * 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
MX2022005236A (es) 2019-10-30 2022-06-08 Decibel Therapeutics Inc Composiciones y metodos para el tratamiento de la perdida auditiva neurosensorial mediante el uso de sistemas de vectores duales de otoferlina.

Also Published As

Publication number Publication date
CN113924109A (zh) 2022-01-11
BR112021013996A2 (pt) 2021-10-26
LT3911354T (lt) 2023-11-27
AU2020208933B2 (en) 2025-08-28
HRP20231296T1 (hr) 2024-02-16
EP4295913A3 (en) 2024-03-06
AU2020208933A8 (en) 2021-08-26
RS64794B1 (sr) 2023-11-30
ES2960880T3 (es) 2024-03-07
PL3911354T3 (pl) 2024-03-11
EP3911354B8 (en) 2024-04-03
DK3911354T5 (da) 2024-08-05
FI3911354T3 (fi) 2023-10-17
IL284742B1 (en) 2024-02-01
EP3911354B1 (en) 2023-07-19
CA3126884A1 (en) 2020-07-23
KR20210118112A (ko) 2021-09-29
PT3911354T (pt) 2023-10-24
WO2020148458A1 (en) 2020-07-23
MX2021008622A (es) 2021-12-15
EP4295913A2 (en) 2023-12-27
SI3911354T1 (sl) 2023-12-29
JP7595013B2 (ja) 2024-12-05
DK3911354T3 (da) 2023-10-16
IL284742A (en) 2021-08-31
EP3911354A1 (en) 2021-11-24
AU2020208933A1 (en) 2021-07-29
JP2022522996A (ja) 2022-04-21
US20220125875A1 (en) 2022-04-28
JP2025032155A (ja) 2025-03-11
SMT202300359T1 (it) 2023-11-13
IL284742B2 (en) 2024-06-01

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