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

캡시드 변형에 의한 증가하는 조직 특정 유전자 전달

Info

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
Authority
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
Application number
KR1020257023347A
Other languages
English (en)
Korean (ko)
Inventor
스코트 알렌 로일러
Original Assignee
더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈
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
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

Links

Classifications

    • 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
KR1020207028750A KR102834868B1 (ko) 2018-03-16 2019-03-14 캡시드 변형에 의한 증가하는 조직 특정 유전자 전달
PCT/US2019/022353 WO2019178412A1 (en) 2018-03-16 2019-03-14 Increasing tissue specific gene delivery by capsid modification

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
KR1020207028750A Active KR102834868B1 (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

Family Applications After (2)

Application Number Title Priority Date Filing Date
KR1020207028750A Active KR102834868B1 (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

Country Status (11)

Country Link
US (2) US12385062B2 (https=)
EP (1) EP3765624A4 (https=)
JP (3) JP7304878B2 (https=)
KR (3) KR20250111395A (https=)
CN (2) CN118373887A (https=)
AU (2) AU2019234918C1 (https=)
BR (1) BR112020018354A2 (https=)
CA (3) CA3293599A1 (https=)
IL (2) IL277270B2 (https=)
SG (1) SG11202008687XA (https=)
WO (1) WO2019178412A1 (https=)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112018003665A2 (en) 2015-09-28 2018-09-25 The University Of North Carolina At Chapel Hill methods and compositions for antibody evasion viral vectors
CN118373887A (zh) 2018-03-16 2024-07-23 国家儿童医院研究所 通过衣壳修饰增加组织特异性的基因递送
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
US20210363191A1 (en) 2018-04-03 2021-11-25 Stridebio, Inc. Antibody-evading virus vectors
BR112021018776A2 (pt) 2019-03-21 2021-11-30 Stridebio Inc Vetores de vírus adenoassociado recombinante
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
EP4200408A1 (en) 2020-08-19 2023-06-28 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
CA3247417A1 (en) 2022-04-06 2023-10-12 Genzyme Corporation TARGETED GENE THERAPY FOR DM-1 MYOTONIC DYSTROPHY
JP2026511976A (ja) * 2023-04-05 2026-04-14 ジェンザイム・コーポレーション Ｄｍ－１筋強直性ジストロフィーの標的遺伝子療法

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
CN1856576B (zh) * 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
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
AU2013249202B2 (en) 2012-04-18 2018-08-09 The Children's Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using AAV capsid variants
KR20240090694A (ko) 2013-07-22 2024-06-21 더 칠드런스 호스피탈 오브 필라델피아 변종 ａａｖ 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
AU2015335923B2 (en) * 2014-10-21 2021-04-29 University Of Massachusetts Recombinant AAV variants and uses thereof
BR112018003665A2 (en) * 2015-09-28 2018-09-25 The University Of North Carolina At Chapel Hill methods and compositions for antibody evasion viral vectors
AU2017250791B2 (en) * 2016-04-15 2022-11-24 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy
FI3445773T3 (fi) * 2016-05-13 2023-03-30 4D Molecular Therapeutics Inc Adenoassosioituneen viruksen kapsidimuunnoksia ja niiden käyttömenetelmiä
CN118373887A (zh) 2018-03-16 2024-07-23 国家儿童医院研究所 通过衣壳修饰增加组织特异性的基因递送
WO2020047472A1 (en) 2018-08-30 2020-03-05 Research Institute At Nationwide Children's Hospital Gene therapy for the treatment of galactosemia

Also Published As

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

Similar Documents

Publication Publication Date Title
KR102834868B1 (ko) 캡시드 변형에 의한 증가하는 조직 특정 유전자 전달
US12454701B2 (en) Recombinant adeno-associated viral vector for gene delivery
JP7702949B2 (ja) ヒトｍｅｃｐ２遺伝子を発現するように設計されたトランスジーンカセット
US12534741B2 (en) Compositions and methods for treatment of neurological disorders
JP2023534026A (ja) 多部分遺伝子送達のための組み換えアデノ随伴ウイルスベクター
WO2024220719A1 (en) Modified aav capsids for gene delivery
RU2801217C2 (ru) Повышение тканеспецифической доставки генов путем модификации капсида
Kwiatek et al. Directed evolution of AAV capsids for improved efficacy and specificity of delivery to preclinical models of human liver
WO2024163335A2 (en) Pten gene therapy vectors and uses thereof
WO2023102518A1 (en) Gnao1 gene therapy vectors and uses thereof

Legal Events

Date Code Title Description
A16 Divisional, continuation or continuation in part application filed

Free format text: ST27 STATUS EVENT CODE: A-0-1-A10-A16-DIV-PA0104 (AS PROVIDED BY THE NATIONAL OFFICE)

A18 Application divided or continuation or continuation in part accepted

Free format text: ST27 STATUS EVENT CODE: A-0-1-A10-A18-DIV-PA0104 (AS PROVIDED BY THE NATIONAL OFFICE)

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P13-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PA0104 Divisional application for international application

St.27 status event code: A-0-1-A10-A18-div-PA0104

St.27 status event code: A-0-1-A10-A16-div-PA0104

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

Q12 Application published

Free format text: ST27 STATUS EVENT CODE: A-1-1-Q10-Q12-NAP-PG1501 (AS PROVIDED BY THE NATIONAL OFFICE)