AU2009212309B2 - Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein - Google Patents

Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein Download PDF

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Publication number
AU2009212309B2
AU2009212309B2 AU2009212309A AU2009212309A AU2009212309B2 AU 2009212309 B2 AU2009212309 B2 AU 2009212309B2 AU 2009212309 A AU2009212309 A AU 2009212309A AU 2009212309 A AU2009212309 A AU 2009212309A AU 2009212309 B2 AU2009212309 B2 AU 2009212309B2
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AU
Australia
Prior art keywords
cells
vector
aav
growth factor
layer
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.)
Ceased
Application number
AU2009212309A
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English (en)
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AU2009212309A1 (en
Inventor
Raymond T. Bartus
Kathie M. Bishop
Mehdi Gasmi
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Ceregene Inc
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Ceregene Inc
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Publication date
Application filed by Ceregene Inc filed Critical Ceregene Inc
Publication of AU2009212309A1 publication Critical patent/AU2009212309A1/en
Application granted granted Critical
Publication of AU2009212309B2 publication Critical patent/AU2009212309B2/en
Ceased legal-status Critical Current
Anticipated expiration legal-status Critical

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
    • 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
    • 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
    • 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
    • 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
    • A61K9/0051—Ocular inserts or implants
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Ophthalmology & Optometry (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2009212309A 2008-02-07 2009-02-05 Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein Ceased AU2009212309B2 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US2699008P 2008-02-07 2008-02-07
US61/026,990 2008-02-07
US9322808P 2008-08-29 2008-08-29
US61/093,228 2008-08-29
PCT/US2009/033275 WO2009100253A1 (en) 2008-02-07 2009-02-05 Rescue of photoreceptors by intravitreal administation of an expression vector encoding a therapeutic protein

Publications (2)

Publication Number Publication Date
AU2009212309A1 AU2009212309A1 (en) 2009-08-13
AU2009212309B2 true AU2009212309B2 (en) 2014-04-03

Family

ID=40939054

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2009212309A Ceased AU2009212309B2 (en) 2008-02-07 2009-02-05 Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein

Country Status (7)

Country Link
US (2) US8242093B2 (enExample)
EP (1) EP2247258A4 (enExample)
JP (1) JP2011511090A (enExample)
CN (1) CN101980668A (enExample)
AU (1) AU2009212309B2 (enExample)
CA (1) CA2713755A1 (enExample)
WO (1) WO2009100253A1 (enExample)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009100253A1 (en) * 2008-02-07 2009-08-13 Ceregene, Inc. Rescue of photoreceptors by intravitreal administation of an expression vector encoding a therapeutic protein
GB201103062D0 (en) * 2011-02-22 2011-04-06 Isis Innovation Method
CN103459593A (zh) * 2011-02-25 2013-12-18 独立行政法人理化学研究所 视网膜色素上皮细胞片层的生产方法
EP2872183B1 (en) * 2012-07-11 2018-09-26 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for rpgr x-linked retinal degeneration
WO2015044704A1 (en) * 2013-09-30 2015-04-02 Sanofi Use of neuroglobin agonist for preventing or treating mitochondrial rcci and/or rcciii deficiency disease
RU2723101C2 (ru) * 2013-12-06 2020-06-08 Инсерм (Инститьют Насьонал Де Ла Санте Ет Де Ла Решерш Медикаль) Вектор, способ лечения хороидермии, способ селективной экспрессии полинуклеотида
US10293059B2 (en) * 2015-04-09 2019-05-21 Cornell University Gene therapy to prevent reactions to allergens
EP3384036B1 (en) * 2015-12-04 2021-02-24 Sorbonne Université Promoters and uses thereof
CA3025975A1 (en) * 2016-04-29 2017-11-02 Gensight Biologics Sa Optogenetic visual restoration using chrimson
PL239642B1 (pl) * 2018-08-16 2021-12-20 Pomorski Univ Medyczny W Szczecinie Kompozycja do doszklistkowego podawania białka leczniczego
WO2022086990A1 (en) * 2020-10-22 2022-04-28 Visgenx, Inc. Elovl2 constructs for human gene therapy
AU2023390868A1 (en) * 2022-12-05 2025-06-19 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Pigment epithelium-derived factor peptides and use for treating retinal degeneration

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020194630A1 (en) * 1999-03-15 2002-12-19 Manning William C. Use of recombinant gene delivery vectors for treating or preventing diseases of the eye

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5932479A (en) 1988-09-26 1999-08-03 Auburn University Genetic engineering of plant chloroplasts
CA2293492A1 (en) * 1997-06-11 1998-12-17 Martin Berry Cns neuroregenerative compositions and methods of use
EP1154691A4 (en) * 1999-01-05 2004-07-07 Massachusetts Eye & Ear Infirm TARGETED ADMINISTRATION OF REGULATED RELEASE MEDICINE TO RETINA AND CHOROID THROUGH SCLEROTIC
WO2000054813A2 (en) * 1999-03-15 2000-09-21 Chiron Corporation Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
AU2001241809A1 (en) 2000-02-29 2001-09-12 Auburn University Genetic engineering of drought tolerance via a plastid genome
EP2080803B1 (en) 2000-03-01 2011-05-25 Auburn University Plastid transformation vectors for expressing proinsulin fused to the cholera toxin B-subunit in plants
US7354760B2 (en) 2001-12-26 2008-04-08 University Of Central Florida Research Foundation, Inc. Expression of protective antigens in transgenic chloroplasts
AU2003253781A1 (en) 2002-07-03 2004-01-23 University Of Central Florida Expression of human interferon in transgenic chloroplasts
WO2006101634A1 (en) 2005-02-17 2006-09-28 The Regents Of The University Of California Müller cell specific gene therapy
US20060188481A1 (en) * 2005-02-22 2006-08-24 Saitama Medical School Methods for prophylactically or therapeutically treating an animal for an ocular-related disorder
JP2008537478A (ja) 2005-02-24 2008-09-18 ザ・スクリプス・リサーチ・インステイチユート 単離骨髄球様骨髄細胞集団及びそれを用いた治療方法
AU2006309281B2 (en) 2005-05-27 2011-11-03 University Of Central Florida Chloroplasts engineered to express pharmaceutical proteins
WO2009100253A1 (en) * 2008-02-07 2009-08-13 Ceregene, Inc. Rescue of photoreceptors by intravitreal administation of an expression vector encoding a therapeutic protein

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020194630A1 (en) * 1999-03-15 2002-12-19 Manning William C. Use of recombinant gene delivery vectors for treating or preventing diseases of the eye

Also Published As

Publication number Publication date
US20120316112A1 (en) 2012-12-13
US8420619B2 (en) 2013-04-16
CN101980668A (zh) 2011-02-23
AU2009212309A1 (en) 2009-08-13
US8242093B2 (en) 2012-08-14
CA2713755A1 (en) 2009-08-13
JP2011511090A (ja) 2011-04-07
WO2009100253A8 (en) 2010-08-19
EP2247258A1 (en) 2010-11-10
WO2009100253A1 (en) 2009-08-13
EP2247258A4 (en) 2012-01-18
US20090202505A1 (en) 2009-08-13

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Legal Events

Date Code Title Description
DA3 Amendments made section 104

Free format text: THE NATURE OF THE AMENDMENT IS: AMEND THE INVENTION TITLE TO READ RESCUE OF PHOTORECEPTORS BY INTRAVITREAL ADMINISTRATION OF AN EXPRESSION VECTOR ENCODING A THERAPEUTIC PROTEIN

FGA Letters patent sealed or granted (standard patent)
MK14 Patent ceased section 143(a) (annual fees not paid) or expired