KR20190096329A - 녹내장에서 신경보호 요법으로서 sfasl의 aav2-매개된 유전자 전달 - Google Patents

녹내장에서 신경보호 요법으로서 sfasl의 aav2-매개된 유전자 전달 Download PDF

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KR20190096329A
KR20190096329A KR1020197003089A KR20197003089A KR20190096329A KR 20190096329 A KR20190096329 A KR 20190096329A KR 1020197003089 A KR1020197003089 A KR 1020197003089A KR 20197003089 A KR20197003089 A KR 20197003089A KR 20190096329 A KR20190096329 A KR 20190096329A
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sfasl
raav
subject
mice
glaucoma
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Ceased
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KR1020197003089A
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Korean (ko)
Inventor
안 마샥-로스스타인
메레디스 그레고리-크샌더
브루스 크샌더
Original Assignee
유니버시티 오브 매사추세츠
매사추세츠 아이 앤드 이어 인퍼머리
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Publication of KR20190096329A publication Critical patent/KR20190096329A/ko
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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
    • C—CHEMISTRY; METALLURGY
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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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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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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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  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
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  • Biochemistry (AREA)
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  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Virology (AREA)
  • General Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
KR1020197003089A 2016-07-05 2017-07-05 녹내장에서 신경보호 요법으로서 sfasl의 aav2-매개된 유전자 전달 Ceased KR20190096329A (ko)

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
KR20190096329A true KR20190096329A (ko) 2019-08-19

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KR1020197003089A Ceased KR20190096329A (ko) 2016-07-05 2017-07-05 녹내장에서 신경보호 요법으로서 sfasl의 aav2-매개된 유전자 전달

Country Status (7)

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

Cited By (1)

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Publication number Priority date Publication date Assignee Title
WO2023048529A1 (ko) * 2021-09-27 2023-03-30 (주) 씨드모젠 Aav2-f11 단백질을 유효성분으로 포함하는 녹내장 예방 또는 치료용 조성물

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JP6884755B2 (ja) 2015-05-01 2021-06-09 オーエヌエル セラピューティクス，インコーポレーテッド ペプチド組成物および使用方法
EP3768301A4 (en) * 2018-03-20 2022-06-08 ONL Therapeutics, Inc. COMPOSITIONS AND METHODS FOR 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
WO2023129766A1 (en) * 2021-12-27 2023-07-06 Onl Therapeutics, Inc. Sfasl for inhibiting rpe cell death and associated disorders
TW202346600A (zh) * 2022-03-30 2023-12-01 高等教育聯邦系統 匹茲堡大學 用於將核酸遞送至視網膜神經節細胞和/或視網膜色素上皮細胞的腺相關病毒載體

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US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
WO1998010088A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US6544523B1 (en) * 1996-11-13 2003-04-08 Chiron Corporation Mutant forms of Fas ligand and uses thereof
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US6962815B2 (en) * 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
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GB0820262D0 (en) * 2008-11-05 2008-12-10 Tmo Renewables Ltd Microorganisms
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US9238679B2 (en) * 2011-02-11 2016-01-19 The Trustees Of The University Of Pennslyvania Nucleic acid molecule encoding hepatitis B virus core protein and surface antigen protein and vaccine comprising the same
EP2586793A1 (en) * 2011-10-27 2013-05-01 Centre National de la Recherche Scientifique Chimeric molecule involving oligomerized FasL extracellular domain
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023048529A1 (ko) * 2021-09-27 2023-03-30 (주) 씨드모젠 Aav2-f11 단백질을 유효성분으로 포함하는 녹내장 예방 또는 치료용 조성물

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Publication number Publication date
WO2018009553A1 (en) 2018-01-11
EP3481433B1 (en) 2024-07-03
JP2019520405A (ja) 2019-07-18
JP7493334B2 (ja) 2024-05-31
MX2019000221A (es) 2020-02-07
US20250001015A1 (en) 2025-01-02
CA3029864A1 (en) 2018-01-11
JP2022084594A (ja) 2022-06-07
EP3481433A4 (en) 2020-02-26
US20190240353A1 (en) 2019-08-08
EP3481433A1 (en) 2019-05-15
CN110177577A (zh) 2019-08-27

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