CA2938828A1 - Compositions and methods for treating and preventing macular degeneration - Google Patents

Compositions and methods for treating and preventing macular degeneration Download PDF

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Publication number
CA2938828A1
CA2938828A1 CA2938828A CA2938828A CA2938828A1 CA 2938828 A1 CA2938828 A1 CA 2938828A1 CA 2938828 A CA2938828 A CA 2938828A CA 2938828 A CA2938828 A CA 2938828A CA 2938828 A1 CA2938828 A1 CA 2938828A1
Authority
CA
Canada
Prior art keywords
seq
eye
domain
raav
aav
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CA2938828A
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English (en)
French (fr)
Inventor
Abraham Scaria
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Genzyme Corp
Original Assignee
Genzyme Corp
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of CA2938828A1 publication Critical patent/CA2938828A1/en
Pending legal-status Critical Current

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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
    • 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
    • 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

Landscapes

  • 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)
CA2938828A 2014-02-06 2015-02-06 Compositions and methods for treating and preventing macular degeneration Pending CA2938828A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461936797P 2014-02-06 2014-02-06
US61/936,797 2014-02-06
PCT/US2015/014872 WO2015120309A1 (en) 2014-02-06 2015-02-06 Compositions and methods for treating and preventing macular degeneration

Publications (1)

Publication Number Publication Date
CA2938828A1 true CA2938828A1 (en) 2015-08-13

Family

ID=52589781

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2938828A Pending CA2938828A1 (en) 2014-02-06 2015-02-06 Compositions and methods for treating and preventing macular degeneration

Country Status (14)

Country Link
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)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
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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
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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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Also Published As

Publication number Publication date
BR112016017817A2 (pt) 2017-10-10
EP3102246A1 (en) 2016-12-14
TWI687225B (zh) 2020-03-11
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
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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