CN110177577A - Sfasl的aav2介导的基因递送作为青光眼的神经保护疗法 - Google Patents

Sfasl的aav2介导的基因递送作为青光眼的神经保护疗法 Download PDF

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Publication number
CN110177577A
CN110177577A CN201780052351.9A CN201780052351A CN110177577A CN 110177577 A CN110177577 A CN 110177577A CN 201780052351 A CN201780052351 A CN 201780052351A CN 110177577 A CN110177577 A CN 110177577A
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CN
China
Prior art keywords
sfasl
raav
subject
mice
nucleic acid
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Pending
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CN201780052351.9A
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English (en)
Chinese (zh)
Inventor
A·马沙克-罗斯坦
M·格雷戈里-克桑德
B·克桑德
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Brigham and Womens Hospital Inc
University of Massachusetts Amherst
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Brigham and Womens Hospital Inc
University of Massachusetts Amherst
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Publication of CN110177577A publication Critical patent/CN110177577A/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
    • A61K48/0075—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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • 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
    • 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
    • A61P27/06—Antiglaucoma agents or miotics
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70575—NGF/TNF-superfamily, e.g. CD70, CD95L, CD153, CD154
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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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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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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
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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    • 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
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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
    • C12N2750/14171—Demonstrated in vivo effect

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  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Ophthalmology & Optometry (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
CN201780052351.9A 2016-07-05 2017-07-05 Sfasl的aav2介导的基因递送作为青光眼的神经保护疗法 Pending CN110177577A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662358541P 2016-07-05 2016-07-05
US62/358,541 2016-07-05
US201762511629P 2017-05-26 2017-05-26
US62/511,629 2017-05-26
PCT/US2017/040735 WO2018009553A1 (en) 2016-07-05 2017-07-05 Aav2-mediated gene delivery of sfasl as a neuroprotective therapy in glaucoma

Publications (1)

Publication Number Publication Date
CN110177577A true CN110177577A (zh) 2019-08-27

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CN201780052351.9A Pending CN110177577A (zh) 2016-07-05 2017-07-05 Sfasl的aav2介导的基因递送作为青光眼的神经保护疗法

Country Status (7)

Country Link
US (2) US20190240353A1 (https=)
EP (1) EP3481433B1 (https=)
JP (2) JP7493334B2 (https=)
KR (1) KR20190096329A (https=)
CN (1) CN110177577A (https=)
MX (1) MX2019000221A (https=)
WO (1) WO2018009553A1 (https=)

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LT3288379T (lt) 2015-05-01 2022-02-25 Onl Therapeutics, Inc. Peptidų kompozicijos ir naudojimo būdai
WO2019183246A1 (en) * 2018-03-20 2019-09-26 Onl Therapeutics, Inc. Compositions and methods of fas inhibition
US10862211B2 (en) * 2018-08-21 2020-12-08 Htc Corporation Integrated antenna structure
US10818758B2 (en) 2018-11-16 2020-10-27 Asm Ip Holding B.V. Methods for forming a metal silicate film on a substrate in a reaction chamber and related semiconductor device structures
WO2023048529A1 (ko) * 2021-09-27 2023-03-30 (주) 씨드모젠 Aav2-f11 단백질을 유효성분으로 포함하는 녹내장 예방 또는 치료용 조성물
WO2023129766A1 (en) * 2021-12-27 2023-07-06 Onl Therapeutics, Inc. Sfasl for inhibiting rpe cell death and associated disorders
EP4499672A4 (en) * 2022-03-30 2026-04-01 Univ Pittsburgh Commonwealth Sys Higher Education Adeno-associated viral vectors for the delivery of nucleic acids to retinal ganglion cells and/or retinal pigment epithelium cells

Citations (4)

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US20020192823A1 (en) * 2001-01-05 2002-12-19 Bartlett Jeffrey S. AAV2 vectors and methods
US20040126859A1 (en) * 1996-11-13 2004-07-01 Keting Chu Mutant forms of Fas ligand and uses thereof
US20140024598A1 (en) * 2010-11-01 2014-01-23 Demetrios Vavvas Methods and compositions for preserving retinal ganglion cells
WO2015044462A1 (en) * 2013-09-30 2015-04-02 Sanofi Use of neuroglobin agonist for preventing or treating mitochondrial rcci and/or rcciii deficiency disease

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NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
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US20140024598A1 (en) * 2010-11-01 2014-01-23 Demetrios Vavvas Methods and compositions for preserving retinal ganglion cells
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Also Published As

Publication number Publication date
MX2019000221A (es) 2020-02-07
US20250001015A1 (en) 2025-01-02
WO2018009553A1 (en) 2018-01-11
JP2022084594A (ja) 2022-06-07
US20190240353A1 (en) 2019-08-08
KR20190096329A (ko) 2019-08-19
CA3029864A1 (en) 2018-01-11
JP7493334B2 (ja) 2024-05-31
EP3481433A1 (en) 2019-05-15
EP3481433A4 (en) 2020-02-26
JP2019520405A (ja) 2019-07-18
EP3481433B1 (en) 2024-07-03

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Application publication date: 20190827