TWI687225B - 用於治療及預防黃斑部病變的組成物及方法 - Google Patents

用於治療及預防黃斑部病變的組成物及方法 Download PDF

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TWI687225B
TWI687225B TW104104067A TW104104067A TWI687225B TW I687225 B TWI687225 B TW I687225B TW 104104067 A TW104104067 A TW 104104067A TW 104104067 A TW104104067 A TW 104104067A TW I687225 B TWI687225 B TW I687225B
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aav
sequence
seq
amino acid
raav
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TW104104067A
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TW201542217A (zh
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亞伯拉罕 史瑞亞
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美商健臻公司
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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
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C07K14/71—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • H—ELECTRICITY
    • H02—GENERATION; CONVERSION OR DISTRIBUTION OF ELECTRIC POWER
    • H02J—ELECTRIC POWER NETWORKS; CIRCUIT ARRANGEMENTS OR SYSTEMS FOR SUPPLYING OR DISTRIBUTING ELECTRIC POWER; SYSTEMS FOR STORING ELECTRIC ENERGY
    • H02J50/00—Circuit arrangements or systems for wireless supply or distribution of electric power
    • H02J50/10—Circuit arrangements or systems for wireless supply or distribution of electric power using inductive coupling
    • H—ELECTRICITY
    • H02—GENERATION; CONVERSION OR DISTRIBUTION OF ELECTRIC POWER
    • H02J—ELECTRIC POWER NETWORKS; CIRCUIT ARRANGEMENTS OR SYSTEMS FOR SUPPLYING OR DISTRIBUTING ELECTRIC POWER; SYSTEMS FOR STORING ELECTRIC ENERGY
    • H02J50/00—Circuit arrangements or systems for wireless supply or distribution of electric power
    • H02J50/40—Circuit arrangements or systems for wireless supply or distribution of electric power using two or more transmitting or receiving devices
    • H—ELECTRICITY
    • H02—GENERATION; CONVERSION OR DISTRIBUTION OF ELECTRIC POWER
    • H02J—ELECTRIC POWER NETWORKS; CIRCUIT ARRANGEMENTS OR SYSTEMS FOR SUPPLYING OR DISTRIBUTING ELECTRIC POWER; SYSTEMS FOR STORING ELECTRIC ENERGY
    • H02J7/00—Circuit arrangements for charging or discharging batteries or for supplying loads from batteries
    • H02J7/02—Circuit arrangements for charging or discharging batteries or for supplying loads from batteries for charging batteries from AC mains by converters
    • H—ELECTRICITY
    • H02—GENERATION; CONVERSION OR DISTRIBUTION OF ELECTRIC POWER
    • H02J—ELECTRIC POWER NETWORKS; CIRCUIT ARRANGEMENTS OR SYSTEMS FOR SUPPLYING OR DISTRIBUTING ELECTRIC POWER; SYSTEMS FOR STORING ELECTRIC ENERGY
    • H02J7/00—Circuit arrangements for charging or discharging batteries or for supplying loads from batteries
    • H02J7/50—Circuit arrangements for charging or discharging batteries or for supplying loads from batteries acting upon multiple batteries simultaneously or sequentially
    • H—ELECTRICITY
    • H02—GENERATION; CONVERSION OR DISTRIBUTION OF ELECTRIC POWER
    • H02J—ELECTRIC POWER NETWORKS; CIRCUIT ARRANGEMENTS OR SYSTEMS FOR SUPPLYING OR DISTRIBUTING ELECTRIC POWER; SYSTEMS FOR STORING ELECTRIC ENERGY
    • H02J7/00—Circuit arrangements for charging or discharging batteries or for supplying loads from batteries
    • H02J7/70—Circuit arrangements for charging or discharging batteries or for supplying loads from batteries characterised by the mechanical construction
    • H—ELECTRICITY
    • H02—GENERATION; CONVERSION OR DISTRIBUTION OF ELECTRIC POWER
    • H02J—ELECTRIC POWER NETWORKS; CIRCUIT ARRANGEMENTS OR SYSTEMS FOR SUPPLYING OR DISTRIBUTING ELECTRIC POWER; SYSTEMS FOR STORING ELECTRIC ENERGY
    • H02J7/00—Circuit arrangements for charging or discharging batteries or for supplying loads from batteries
    • H02J7/70—Circuit arrangements for charging or discharging batteries or for supplying loads from batteries characterised by the mechanical construction
    • H02J7/751—Circuit arrangements for charging or discharging batteries or for supplying loads from batteries characterised by the mechanical construction concerning the insertion or the connection of the batteries
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • 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
    • C12N2750/14171—Demonstrated in vivo effect
    • H—ELECTRICITY
    • H02—GENERATION; CONVERSION OR DISTRIBUTION OF ELECTRIC POWER
    • H02J—ELECTRIC POWER NETWORKS; CIRCUIT ARRANGEMENTS OR SYSTEMS FOR SUPPLYING OR DISTRIBUTING ELECTRIC POWER; SYSTEMS FOR STORING ELECTRIC ENERGY
    • H02J7/00—Circuit arrangements for charging or discharging batteries or for supplying loads from batteries
    • H02J7/34—Parallel operation in networks using both storage and other DC sources, e.g. providing buffering
    • H02J7/35—Parallel operation in networks using both storage and other DC sources, e.g. providing buffering with light sensitive cells

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Power Engineering (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Zoology (AREA)
  • Toxicology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Ophthalmology & Optometry (AREA)
  • Computer Networks & Wireless Communication (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Peptides Or Proteins (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)
  • Liquid Crystal Substances (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
TW104104067A 2014-02-06 2015-02-06 用於治療及預防黃斑部病變的組成物及方法 TWI687225B (zh)

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US201461936797P 2014-02-06 2014-02-06
US61/936,797 2014-02-06

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TW201542217A TW201542217A (zh) 2015-11-16
TWI687225B true TWI687225B (zh) 2020-03-11

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US (3) US20170007719A1 (enExample)
EP (1) EP3102246B1 (enExample)
JP (1) JP6719381B2 (enExample)
KR (1) KR102234695B1 (enExample)
CN (3) CN111068072A (enExample)
AU (4) AU2015213770A1 (enExample)
BR (1) BR112016017817A2 (enExample)
CA (1) CA2938828A1 (enExample)
IL (1) IL247116B (enExample)
MX (2) MX2016010215A (enExample)
RU (1) RU2703145C2 (enExample)
SG (1) SG11201606101WA (enExample)
TW (1) TWI687225B (enExample)
WO (1) WO2015120309A1 (enExample)

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KR102234695B1 (ko) * 2014-02-06 2021-04-02 젠자임 코포레이션 황반 변성을 치료하고 예방하기 위한 조성물 및 방법
KR101685532B1 (ko) * 2016-04-26 2016-12-13 한국프라임제약주식회사 혈관내피성장인자 수용체 융합단백질
KR102616820B1 (ko) 2017-03-17 2023-12-21 애드베룸 바이오테크놀로지스, 인코포레이티드 향상된 유전자 발현을 위한 조성물 및 방법
KR102205830B1 (ko) * 2017-10-26 2021-01-21 주식회사 큐로진생명과학 솔루블 VEGFR-1 변이체 cDNA를 함유하는 rAAV를 포함하는 황반변성 치료용 조성물
AU2019221642A1 (en) * 2018-02-14 2020-07-09 Generation Bio Co. Non-viral DNA vectors and uses thereof for antibody and fusion protein production
US12534739B2 (en) * 2018-10-11 2026-01-27 Decibel Therapeutics, Inc. AAV1 vectors and uses thereof for treatment of otic indications
CN114729009A (zh) 2019-10-31 2022-07-08 伯尔尼大学 用于眼部基因递送的aav载体变体
CN113952473A (zh) * 2020-07-21 2022-01-21 英斯培瑞有限公司 用于治疗眼部疾病的组合物和方法
CN119656336A (zh) * 2023-09-21 2025-03-21 成都弘基生物科技有限公司 一种重组腺相关病毒在制备治疗与血管新生相关眼病的药物中的用途

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BR112016017817A2 (pt) 2017-10-10
EP3102246A1 (en) 2016-12-14
CN120131999A (zh) 2025-06-13
SG11201606101WA (en) 2016-08-30
AU2025203206A1 (en) 2025-05-29
RU2016135771A3 (enExample) 2018-10-15
AU2021250986A1 (en) 2021-11-18
MX2023011453A (es) 2023-10-18
CN106163573A (zh) 2016-11-23
AU2021250986B2 (en) 2025-02-06
US12483047B2 (en) 2025-11-25
US20170007719A1 (en) 2017-01-12
RU2016135771A (ru) 2018-03-14
US20260166177A1 (en) 2026-06-18
AU2019203413A1 (en) 2019-06-06
KR102234695B1 (ko) 2021-04-02
TW201542217A (zh) 2015-11-16
MX2016010215A (es) 2016-11-15
CN111068072A (zh) 2020-04-28
AU2019203413B2 (en) 2021-07-15
CA2938828A1 (en) 2015-08-13
EP3102246B1 (en) 2020-03-25
US20220288232A1 (en) 2022-09-15
JP6719381B2 (ja) 2020-07-08
JP2017506230A (ja) 2017-03-02
RU2703145C2 (ru) 2019-10-15
WO2015120309A1 (en) 2015-08-13
IL247116B (en) 2020-06-30
AU2015213770A1 (en) 2016-09-01
KR20160110523A (ko) 2016-09-21
IL247116A0 (en) 2016-09-29

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