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
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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)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Immunology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (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 (https=)
EP (1) EP2247258A4 (https=)
JP (1) JP2011511090A (https=)
CN (1) CN101980668A (https=)
AU (1) AU2009212309B2 (https=)
CA (1) CA2713755A1 (https=)
WO (1) WO2009100253A1 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101980668A (zh) * 2008-02-07 2011-02-23 赛拉基因公司 通过玻璃体内施用编码治疗用蛋白质的表达载体救援光感器
GB201103062D0 (en) * 2011-02-22 2011-04-06 Isis Innovation Method
US9902933B2 (en) * 2011-02-25 2018-02-27 Riken Method of producing retinal pigment epithelial cell sheet
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
US20160256571A1 (en) * 2013-09-30 2016-09-08 Sanofi Invention
KR102281881B1 (ko) * 2013-12-06 2021-07-27 엥스띠뛰 나씨오날 드 라 쌍떼 에 드 라 흐쉐르슈 메디깔 (인쎄름) 대상체의 망막 색소 상피에서 목적 폴리뉴클레오티드를 발현시키기 위한 방법 및 약학적 조성물
CN107635584B (zh) * 2015-04-09 2022-06-17 康奈尔大学 基因治疗以预防对过敏原的反应
WO2017093566A1 (en) * 2015-12-04 2017-06-08 Universite Pierre Et Marie Curie (Paris 6) Promoters and uses thereof
WO2017187272A1 (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
IL302164A (en) * 2020-10-22 2023-06-01 Visgenx Inc Elovl2 constructs for human gene therapy
WO2024123461A1 (en) * 2022-12-05 2024-06-13 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
JP2002512627A (ja) * 1997-06-11 2002-04-23 アコーダ セラピーティックス Ｃｎｓ神経再生組成物及びその使用方法
EP1154691A4 (en) 1999-01-05 2004-07-07 Massachusetts Eye & Ear Infirm TARGETED ADMINISTRATION OF REGULATED RELEASE MEDICINE TO RETINA AND CHOROID THROUGH SCLEROTIC
AU3755900A (en) * 1999-03-15 2000-10-04 Chiron Corporation Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
WO2001064850A1 (en) 2000-02-29 2001-09-07 Auburn University Genetic engineering of drought tolerance via a plastid genome
EP1274846A4 (en) 2000-03-01 2004-09-29 Univ Auburn PLASTIDE TRANSFORMATION VECTOR FOR THE EXPRESSION OF HUMAN PROTEINS 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
CA2598029A1 (en) 2005-02-24 2006-10-05 The Scripps Research Institute Isolated myeloid-like bone marrow cell populations and methods of treatment therewith
US20080274143A1 (en) 2005-05-27 2008-11-06 Henry Daniell Chloroplasts Engineering to Express Pharmaceutical Proteins
CN101980668A (zh) * 2008-02-07 2011-02-23 赛拉基因公司 通过玻璃体内施用编码治疗用蛋白质的表达载体救援光感器

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