DK2276501T3 - Fremgangsmåder og sammensætninger til genetisk og retinal sygdom - Google Patents

Fremgangsmåder og sammensætninger til genetisk og retinal sygdom Download PDF

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Publication number
DK2276501T3
DK2276501T3 DK09719985.5T DK09719985T DK2276501T3 DK 2276501 T3 DK2276501 T3 DK 2276501T3 DK 09719985 T DK09719985 T DK 09719985T DK 2276501 T3 DK2276501 T3 DK 2276501T3
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Denmark
Prior art keywords
norrin
protein
cell
compound
retinal
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DK09719985.5T
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English (en)
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Kimberly Drenser
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Kimberly Drenser
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Publication of DK2276501T3 publication Critical patent/DK2276501T3/da

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/48Reproductive organs
    • A61K35/54Ovaries; Ova; Ovules; Embryos; Foetal cells; Germ cells
    • A61K35/545Embryonic stem cells; Pluripotent stem cells; Induced pluripotent stem cells; Uncharacterised stem cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18Growth factors; Growth regulators
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Developmental Biology & Embryology (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Reproductive Health (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Immunology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Ophthalmology & Optometry (AREA)
  • Virology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Claims (4)

1. Norrin-forbindelse til anvendelse ved behandling af en patologisk tilstand i retina, hvor Norrin-forbindelsen er en nukleotidsekvens, der koder for et Norrin-protein, en celle, som er transficeret med en nukleotidsekvens, der koder for et Norrin-protein, og omfatter den pågældende nukleotidsekvens, eller et Norrin-protein, hvor Norrin-proteinet er valgt fra gruppen bestående af et polypeptid med sekvensen ifølge SEQ ID NO: 1, SEQ ID NO: 2, SEQ ID NO: 3, et oligomert kompleks, som omfatter et Norrin-protein, og en muteret form af et Norrin-protein, hvor den muterede form er valgt fra gruppen bestående af R64E-mutanten, T27A-, S28A- og S29A-mutanten; P36A-, R37A- og R38A-mutanten og H127A-, E129A- og E130A-mutanten af polypeptidet ifølge SEQ ID NO: 1, hvor den retinale sygdom er valgt fra gruppen bestående af vitreoretinopati, præmaturitetsretinopati og familiær ekssudativ vitreoretinopati.
2. Norrin-forbindelse til anvendelse ifølge krav 1, hvor nukleotidsekvensen, der koder for et Norrin-protein, er en genoverførselsvektor.
3. Norrin-forbindelse til anvendelse ifølge et hvilket som helst af de foregående krav, hvor forbindelsen skal administreres intraokulært, ved topisk applikation i øjet, ved intraokulær injektion, lokalt ved indføring eller injektion i vævet omkring øjet, systemisk ad oral vej, eller ved subkutan, intravenøs eller intramuskulær injektion.
4. Norrin-forbindelse til anvendelse ifølge krav 3, hvor den intraokulære injektion sker i corpus vitreum-rummet eller subretinalt.
DK09719985.5T 2008-03-14 2009-03-20 Fremgangsmåder og sammensætninger til genetisk og retinal sygdom DK2276501T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US3671708P 2008-03-14 2008-03-14
PCT/US2009/037792 WO2009114878A2 (en) 2008-03-14 2009-03-20 Methods and compositions for genetic and retinal disease

Publications (1)

Publication Number Publication Date
DK2276501T3 true DK2276501T3 (da) 2017-03-20

Family

ID=41065889

Family Applications (1)

Application Number Title Priority Date Filing Date
DK09719985.5T DK2276501T3 (da) 2008-03-14 2009-03-20 Fremgangsmåder og sammensætninger til genetisk og retinal sygdom

Country Status (3)

Country Link
EP (1) EP2276501B1 (da)
DK (1) DK2276501T3 (da)
WO (1) WO2009114878A2 (da)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2367564A1 (en) * 2008-12-22 2011-09-28 Universität Regensburg Norrin in the treatment of diseases associated with an increased tgf-beta activity
EP2968452A4 (en) * 2013-03-15 2016-10-05 Michael Thomas Trese COMPOSITIONS AND METHOD FOR THE TREATMENT OF NETWORK DISEASES
US10202429B2 (en) 2015-06-08 2019-02-12 Retinal Solutions Llc Norrin regulation of cellular production of junction proteins and use to treat retinal vasculature edema
US10206978B2 (en) 2015-06-08 2019-02-19 Retinal Solutions Llc Norrin regulation of junction proteins and the use thereof to treat epithelial or endothelial membrane leakage induced edema
US10669321B2 (en) 2015-06-08 2020-06-02 Retinal Solutions Llc Retinal capillary regeneration with synthetic norrin protein
CN111926021B (zh) * 2020-07-29 2024-06-21 武汉纽福斯生物科技有限公司 重组人norrin胱氨酸结生长因子表达载体及其应用

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5667968A (en) * 1989-08-30 1997-09-16 Regeneron Pharmaceuticals, Inc. Prevention of retinal injury and degeneration by specific factors
CA2346262A1 (en) 1998-09-17 2000-03-23 University Of Florida Methods for treatment of degenerative retinal diseases
AU2002346611A1 (en) 2001-12-03 2003-06-17 The Regents Of The University Of California Expression of glial-derived neurotrophic factor for treatment of diseases of the eye
US20040086572A1 (en) 2002-07-17 2004-05-06 Dailey James P. Delivery of therapeutic agent affixed to magnetic particle
US6899717B2 (en) 2002-09-18 2005-05-31 Allergan, Inc. Methods and apparatus for delivery of ocular implants
WO2005110374A1 (en) 2004-04-30 2005-11-24 Allergan, Inc. Intraocular drug delivery systems containing a therapeutic component, a cyclodextrin, and a polymeric component
US20070154465A1 (en) 2005-12-30 2007-07-05 Alexandar Kharazi Stem cell therapy for retinal disease

Also Published As

Publication number Publication date
WO2009114878A3 (en) 2009-12-03
EP2276501A4 (en) 2011-06-01
EP2276501B1 (en) 2017-02-15
WO2009114878A2 (en) 2009-09-17
EP2276501A2 (en) 2011-01-26

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