KR20250111395A - 캡시드 변형에 의한 증가하는 조직 특정 유전자 전달 - Google Patents

캡시드 변형에 의한 증가하는 조직 특정 유전자 전달

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Publication number
KR20250111395A
KR20250111395A KR1020257023347A KR20257023347A KR20250111395A KR 20250111395 A KR20250111395 A KR 20250111395A KR 1020257023347 A KR1020257023347 A KR 1020257023347A KR 20257023347 A KR20257023347 A KR 20257023347A KR 20250111395 A KR20250111395 A KR 20250111395A
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KR
South Korea
Prior art keywords
gly
modified
aavrh74
aav
pro
Prior art date
Legal status (The legal status 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 status listed.)
Pending
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KR1020257023347A
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English (en)
Korean (ko)
Inventor
스코트 알렌 로일러
Original Assignee
더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈
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Application filed by 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 filed Critical 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈
Publication of KR20250111395A publication Critical patent/KR20250111395A/ko
Pending legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • 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
    • C12N2750/14145—Special targeting system for viral vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Mycology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
KR1020257023347A 2018-03-16 2019-03-14 캡시드 변형에 의한 증가하는 조직 특정 유전자 전달 Pending KR20250111395A (ko)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862644317P 2018-03-16 2018-03-16
US62/644,317 2018-03-16
PCT/US2019/022353 WO2019178412A1 (en) 2018-03-16 2019-03-14 Increasing tissue specific gene delivery by capsid modification
KR1020207028750A KR102834868B1 (ko) 2018-03-16 2019-03-14 캡시드 변형에 의한 증가하는 조직 특정 유전자 전달

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
KR1020207028750A Division KR102834868B1 (ko) 2018-03-16 2019-03-14 캡시드 변형에 의한 증가하는 조직 특정 유전자 전달

Publications (1)

Publication Number Publication Date
KR20250111395A true KR20250111395A (ko) 2025-07-22

Family

ID=67906893

Family Applications (3)

Application Number Title Priority Date Filing Date
KR1020257023347A Pending KR20250111395A (ko) 2018-03-16 2019-03-14 캡시드 변형에 의한 증가하는 조직 특정 유전자 전달
KR1020257023337A Pending KR20250111393A (ko) 2018-03-16 2019-03-14 캡시드 변형에 의한 증가하는 조직 특정 유전자 전달
KR1020207028750A Active KR102834868B1 (ko) 2018-03-16 2019-03-14 캡시드 변형에 의한 증가하는 조직 특정 유전자 전달

Family Applications After (2)

Application Number Title Priority Date Filing Date
KR1020257023337A Pending KR20250111393A (ko) 2018-03-16 2019-03-14 캡시드 변형에 의한 증가하는 조직 특정 유전자 전달
KR1020207028750A Active KR102834868B1 (ko) 2018-03-16 2019-03-14 캡시드 변형에 의한 증가하는 조직 특정 유전자 전달

Country Status (11)

Country Link
US (2) US12385062B2 (https=)
EP (1) EP3765624A4 (https=)
JP (3) JP7304878B2 (https=)
KR (3) KR20250111395A (https=)
CN (2) CN111819286B (https=)
AU (2) AU2019234918C1 (https=)
BR (1) BR112020018354A2 (https=)
CA (3) CA3293599A1 (https=)
IL (2) IL317905A (https=)
SG (1) SG11202008687XA (https=)
WO (1) WO2019178412A1 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
EP3765624A4 (en) * 2018-03-16 2022-05-25 Research Institute at Nationwide Children's Hospital Increasing tissue specific gene delivery by capsid modification
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
US12611436B2 (en) 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
JP2022551739A (ja) 2019-10-17 2022-12-13 ストライドバイオ，インコーポレイテッド ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
US20240299586A1 (en) * 2021-05-06 2024-09-12 The Regents Of The University Of California Novel aav serotypes derived from a library screen
WO2023196862A1 (en) 2022-04-06 2023-10-12 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
US20240384271A1 (en) * 2023-04-05 2024-11-21 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5846782A (en) * 1995-11-28 1998-12-08 Genvec, Inc. Targeting adenovirus with use of constrained peptide motifs
EP2345731B1 (en) * 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
WO2012109570A1 (en) 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
NZ701693A (en) * 2012-04-18 2017-02-24 The Children’S Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants
IL297919A (en) * 2013-07-22 2023-01-01 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
CN107073051B (zh) * 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
ES2865487T3 (es) * 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
ES2977730T3 (es) * 2016-04-15 2024-08-29 Res Inst Nationwide Childrens Hospital Administración de microARN-29 y micro-distrofina por virus adenoasociado para tratar la distrofia muscular
US11364308B2 (en) * 2016-05-13 2022-06-21 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
EP3765624A4 (en) * 2018-03-16 2022-05-25 Research Institute at Nationwide Children's Hospital Increasing tissue specific gene delivery by capsid modification
BR112021003174A2 (pt) * 2018-08-30 2021-05-11 Research Institute At Nationwide Children's Hospital terapia gênica para o tratamento de galactosemia

Also Published As

Publication number Publication date
IL277270B1 (en) 2025-01-01
JP2023115149A (ja) 2023-08-18
US12385062B2 (en) 2025-08-12
KR20250111393A (ko) 2025-07-22
CN111819286A (zh) 2020-10-23
KR20200131843A (ko) 2020-11-24
IL277270B2 (en) 2025-05-01
EP3765624A4 (en) 2022-05-25
AU2025204275A1 (en) 2025-07-03
EP3765624A1 (en) 2021-01-20
BR112020018354A2 (pt) 2020-12-29
IL317905A (en) 2025-02-01
CN118373887A (zh) 2024-07-23
CA3092353A1 (en) 2019-09-19
IL277270A (en) 2020-10-29
RU2020131998A (ru) 2022-04-18
JP2024144521A (ja) 2024-10-11
JP2021515572A (ja) 2021-06-24
JP7304878B2 (ja) 2023-07-07
SG11202008687XA (en) 2020-10-29
AU2019234918B2 (en) 2025-08-14
US20210087584A1 (en) 2021-03-25
AU2019234918A1 (en) 2020-09-17
CN111819286B (zh) 2024-05-24
AU2019234918C1 (en) 2026-03-05
CA3293598A1 (en) 2026-03-02
US20250346924A1 (en) 2025-11-13
KR102834868B1 (ko) 2025-07-17
CA3293599A1 (en) 2026-03-02
WO2019178412A1 (en) 2019-09-19

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