KR20240154633A - 개선된 폐 향성을 갖는 아데노-관련 바이러스 변이체 캡시드 및 이의 용도 - Google Patents

개선된 폐 향성을 갖는 아데노-관련 바이러스 변이체 캡시드 및 이의 용도 Download PDF

Info

Publication number
KR20240154633A
KR20240154633A KR1020247032207A KR20247032207A KR20240154633A KR 20240154633 A KR20240154633 A KR 20240154633A KR 1020247032207 A KR1020247032207 A KR 1020247032207A KR 20247032207 A KR20247032207 A KR 20247032207A KR 20240154633 A KR20240154633 A KR 20240154633A
Authority
KR
South Korea
Prior art keywords
seq
aav
capsid protein
amino acid
variant
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
KR1020247032207A
Other languages
English (en)
Korean (ko)
Inventor
피터 프랜시스
멜리사 A. 코터맨
줄리 나이
록산느 크로즈
Original Assignee
4디 몰레큘러 테라퓨틱스 아이엔씨.
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 4디 몰레큘러 테라퓨틱스 아이엔씨. filed Critical 4디 몰레큘러 테라퓨틱스 아이엔씨.
Publication of KR20240154633A publication Critical patent/KR20240154633A/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
    • 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
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/55—Protease inhibitors
    • A61K38/57—Protease inhibitors from animals; from humans
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory 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
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Virology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Pulmonology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020247032207A 2022-03-07 2023-03-06 개선된 폐 향성을 갖는 아데노-관련 바이러스 변이체 캡시드 및 이의 용도 Pending KR20240154633A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263317450P 2022-03-07 2022-03-07
US63/317,450 2022-03-07
PCT/US2023/063789 WO2023172873A2 (en) 2022-03-07 2023-03-06 Adeno-associated virus variant capsids with improved lung tropism and uses thereof

Publications (1)

Publication Number Publication Date
KR20240154633A true KR20240154633A (ko) 2024-10-25

Family

ID=87935918

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247032207A Pending KR20240154633A (ko) 2022-03-07 2023-03-06 개선된 폐 향성을 갖는 아데노-관련 바이러스 변이체 캡시드 및 이의 용도

Country Status (10)

Country Link
US (1) US20260014275A1 (https=)
EP (1) EP4490301A2 (https=)
JP (1) JP2025508059A (https=)
KR (1) KR20240154633A (https=)
CN (1) CN119213135A (https=)
AU (1) AU2023230885A1 (https=)
CA (1) CA3254571A1 (https=)
IL (1) IL315490A (https=)
MX (1) MX2024010938A (https=)
WO (1) WO2023172873A2 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025165162A1 (ko) 2024-02-01 2025-08-07 주식회사 글루진테라퓨틱스 평활근 세포 특이적 유전자 전달이 가능한 아데노연관바이러스 벡터

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2770922C2 (ru) * 2017-09-20 2022-04-25 4Д Молекьюлар Терапьютикс Инк. Капсиды вариантов аденоассоциированных вирусов и методы их применения
US11707487B2 (en) * 2020-04-13 2023-07-25 Affyimmune Therapeutics, Inc. EpCAM antibody and CAR-T cells
AU2021265768B2 (en) * 2020-04-27 2023-03-30 4D Molecular Therapeutics Inc. Adeno-associated variants, formulations and methods for pulmonary delivery

Also Published As

Publication number Publication date
EP4490301A2 (en) 2025-01-15
US20260014275A1 (en) 2026-01-15
MX2024010938A (es) 2025-04-02
AU2023230885A1 (en) 2024-09-26
CA3254571A1 (en) 2023-09-14
WO2023172873A3 (en) 2023-10-19
IL315490A (en) 2024-11-01
JP2025508059A (ja) 2025-03-21
CN119213135A (zh) 2024-12-27
WO2023172873A2 (en) 2023-09-14

Similar Documents

Publication Publication Date Title
US20240209031A1 (en) Methods and compositions for gene transfer across the vasculature
CN111836649B (zh) 腺相关病毒变异衣壳和其使用方法
EP4206216B1 (en) Adeno-associated virus variant capsids and methods of use thereof
CN105431170B (zh) 嵌合腺相关病毒/博卡病毒细小病毒载体
JP2023126919A (ja) バリアントカプシドを有するアデノ随伴ウイルスビリオン及びその使用方法
EP3277819B1 (en) Adeno-associated virus variants and methods of use thereof
US11999965B2 (en) Bocaparvovirus small noncoding RNA and uses thereof
JP2022107646A (ja) アデノ随伴ウイルス（ａａｖ）キャプシドタンパク質の変異体
KR20210008561A (ko) 합성 간-향성 아데노-연관 바이러스 캡시드 및 그의 용도
CA2387484A1 (en) Adeno-associated virus vectors encoding factor viii and methods of using the same
CN101511373A (zh) 用于基因治疗的修饰的因子viii和因子ix基因和载体
CN111718418A (zh) 一种增强基因编辑的融合蛋白及其应用
US20260014275A1 (en) Adeno-Associated Virus Variant Capsids with Improved Lung Tropism and Uses Thereof
HK40121415A (zh) 具有改善的肺向性的腺伴随病毒变体衣壳及其用途
US20250084129A1 (en) Aav5 capsid variants
WO2025024227A2 (en) Aav variants for cardiac delivery
HK40036161A (zh) 腺相关病毒变异衣壳和其使用方法
HK40014535A (en) Mutant of adeno-associated virus (aav) capsid protein
HK1260937A1 (en) Modified capsid proteins for enhanced delivery of parvovirus vectors
HK1217916B (zh) 嵌合腺相關病毒/博卡病毒細小病毒載體

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

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

E13 Pre-grant limitation requested

Free format text: ST27 STATUS EVENT CODE: A-2-3-E10-E13-LIM-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

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