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)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Power Engineering (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Computer Networks & Wireless Communication (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Bioinformatics & Cheminformatics (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)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Liquid Crystal Substances (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicinal Preparation (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)

Applications Claiming Priority (2)

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

Publications (2)

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

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

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KR101685532B1 (ko) * 2016-04-26 2016-12-13 한국프라임제약주식회사 혈관내피성장인자 수용체 융합단백질
EP4653536A3 (en) 2017-03-17 2026-02-25 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression
KR102205830B1 (ko) * 2017-10-26 2021-01-21 주식회사 큐로진생명과학 솔루블 VEGFR-1 변이체 cDNA를 함유하는 rAAV를 포함하는 황반변성 치료용 조성물
KR20200120649A (ko) * 2018-02-14 2020-10-21 제너레이션 바이오 컴퍼니 비-바이러스 dna 벡터 및 항체 및 융합 단백질 생산을 위한 이의 용도
WO2020077295A1 (en) * 2018-10-11 2020-04-16 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
WO2025061000A1 (zh) * 2023-09-21 2025-03-27 成都弘基生物科技有限公司 一种用于治疗受试者中与血管新生相关的眼病的方法

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

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