TW202405181A - 用於跨視網膜區域核酸遞送的腺相關病毒載體 - Google Patents

用於跨視網膜區域核酸遞送的腺相關病毒載體 Download PDF

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TW202405181A
TW202405181A TW112112172A TW112112172A TW202405181A TW 202405181 A TW202405181 A TW 202405181A TW 112112172 A TW112112172 A TW 112112172A TW 112112172 A TW112112172 A TW 112112172A TW 202405181 A TW202405181 A TW 202405181A
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polypeptide
amino acid
acid sequence
seq
vector
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TW112112172A
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Chinese (zh)
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莉亞 卡羅琳 伯恩
莫莉 Ｅ 約翰遜
威廉 理查德 斯塔弗
比爾吉 艾辛 奧茲特克
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高等教育聯邦系統 匹茲堡大學
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Publication of TW202405181A publication Critical patent/TW202405181A/zh

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    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/1783—Nuclear receptors, e.g. retinoic acid receptor [RAR], RXR, nuclear orphan receptors
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0083—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 administration regime
    • 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
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
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    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
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    • 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
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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
    • 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
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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
    • C12N2750/14145—Special targeting system for viral vectors

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  • Wood Science & Technology (AREA)
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  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
TW112112172A 2022-03-30 2023-03-30 用於跨視網膜區域核酸遞送的腺相關病毒載體 TW202405181A (zh)

Applications Claiming Priority (12)

Application Number Priority Date Filing Date Title
US202263325542P 2022-03-30 2022-03-30
US202263325544P 2022-03-30 2022-03-30
US202263325543P 2022-03-30 2022-03-30
US202263325553P 2022-03-30 2022-03-30
US202263325558P 2022-03-30 2022-03-30
US202263325562P 2022-03-30 2022-03-30
US63/325,544 2022-03-30
US63/325,562 2022-03-30
US63/325,542 2022-03-30
US63/325,553 2022-03-30
US63/325,543 2022-03-30
US63/325,558 2022-03-30

Publications (1)

Publication Number Publication Date
TW202405181A true TW202405181A (zh) 2024-02-01

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TW112112172A TW202405181A (zh) 2022-03-30 2023-03-30 用於跨視網膜區域核酸遞送的腺相關病毒載體

Country Status (11)

Country Link
US (1) US20250222135A1 (OSRAM)
EP (1) EP4499847A4 (OSRAM)
JP (1) JP2025510946A (OSRAM)
KR (1) KR20240168434A (OSRAM)
CN (1) CN119213134A (OSRAM)
AU (1) AU2023245716A1 (OSRAM)
CA (1) CA3255242A1 (OSRAM)
IL (1) IL315751A (OSRAM)
MX (1) MX2024011862A (OSRAM)
TW (1) TW202405181A (OSRAM)
WO (1) WO2023192454A2 (OSRAM)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025231339A1 (en) * 2024-05-02 2025-11-06 Avista Therapeutics, Inc. Adeno-associated virus vectors for therapeutic nucleic acid delivery to retinal cells

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6156303A (en) * 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US7504490B1 (en) * 1998-10-16 2009-03-17 Oscient Pharmaceuticals Corporation Nucleic acid and amino acid sequences relating to Apergillus fumigatus for diagnostics and therapeutics
US20040031072A1 (en) * 1999-05-06 2004-02-12 La Rosa Thomas J. Soy nucleic acid molecules and other molecules associated with transcription plants and uses thereof for plant improvement
CN107012171B (zh) * 2011-04-22 2021-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
CA3040483A1 (en) * 2016-10-13 2018-04-19 University Of Massachusetts Aav capsid designs
EP3645052A4 (en) * 2017-06-30 2021-07-28 Regents of the University of California ADENO-ASSOCIATED VIRUS VIRIONS WITH ASSOCIATED CAPSIDE VARIANTS AND METHODS OF USE
CA3190214A1 (en) * 2020-07-29 2022-02-03 University Of Florida Research Foundation, Incorporated Improved aav-mediated x-linked retinoschisis therapies

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JP2025510946A (ja) 2025-04-15
MX2024011862A (es) 2024-11-08
US20250222135A1 (en) 2025-07-10
IL315751A (en) 2024-11-01
KR20240168434A (ko) 2024-11-29
WO2023192454A2 (en) 2023-10-05
CA3255242A1 (en) 2023-10-05
EP4499847A2 (en) 2025-02-05
CN119213134A (zh) 2024-12-27
WO2023192454A3 (en) 2023-11-02
AU2023245716A1 (en) 2024-10-03
EP4499847A4 (en) 2026-03-25

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