CA2713755A1 - 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
CA2713755A1
CA2713755A1 CA2713755A CA2713755A CA2713755A1 CA 2713755 A1 CA2713755 A1 CA 2713755A1 CA 2713755 A CA2713755 A CA 2713755A CA 2713755 A CA2713755 A CA 2713755A CA 2713755 A1 CA2713755 A1 CA 2713755A1
Authority
CA
Canada
Prior art keywords
cells
aav
vector
growth factor
retina
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.)
Abandoned
Application number
CA2713755A
Other languages
English (en)
French (fr)
Inventor
Raymond T. Bartus
Kathie M. Bishop
Mehdi Gasmi
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.)
Ceregene Inc
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of CA2713755A1 publication Critical patent/CA2713755A1/en
Abandoned legal-status Critical Current

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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)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Ophthalmology & Optometry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA2713755A 2008-02-07 2009-02-05 Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein Abandoned CA2713755A1 (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 (1)

Publication Number Publication Date
CA2713755A1 true CA2713755A1 (en) 2009-08-13

Family

ID=40939054

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2713755A Abandoned CA2713755A1 (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
AU2009212309B2 (en) * 2008-02-07 2014-04-03 Ceregene, Inc. Rescue of photoreceptors by intravitreal administration 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
AU2014359136B2 (en) * 2013-12-06 2020-06-25 Centre Hospitalier Universitaire De Montpellier Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the retinal pigment epithelium of a subject
WO2016164920A1 (en) * 2015-04-09 2016-10-13 Cornell University Gene therapy to prevent reactions to allergens
AU2016363830C1 (en) * 2015-12-04 2022-03-24 Centre National De La Recherche Scientifique Promoters and uses thereof
AU2017256910B2 (en) * 2016-04-29 2022-04-07 Centre National De La Recherche Scientifique Optogenetic visual restoration using Chrimson
PL239642B1 (pl) * 2018-08-16 2021-12-20 Pomorski Univ Medyczny W Szczecinie Kompozycja do doszklistkowego podawania białka leczniczego
CA3199230A1 (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

Family Cites Families (14)

* 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
DE69817180D1 (de) * 1997-06-11 2003-09-18 Acorda Therapeutics Hawthorne Zns neuroregenerative zusammensetzungen und deren verwendungen
JP2002534139A (ja) * 1999-01-05 2002-10-15 マサチューセッツ・アイ・アンド・イア・インファーマリー 網膜と脈絡膜への経強膜徐放性薬剤標的送達
US6943153B1 (en) * 1999-03-15 2005-09-13 The Regents Of The University Of California Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
AU3755900A (en) * 1999-03-15 2000-10-04 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
CA2402066C (en) 2000-03-01 2015-09-29 Auburn University Plastid transformation vectors for expressing 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
US20100172871A1 (en) 2005-02-17 2010-07-08 Flannery John G Muller 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
AU2009212309B2 (en) * 2008-02-07 2014-04-03 Ceregene, Inc. Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein

Also Published As

Publication number Publication date
AU2009212309A1 (en) 2009-08-13
AU2009212309B2 (en) 2014-04-03
CN101980668A (zh) 2011-02-23
JP2011511090A (ja) 2011-04-07
EP2247258A1 (en) 2010-11-10
WO2009100253A8 (en) 2010-08-19
US20090202505A1 (en) 2009-08-13
US20120316112A1 (en) 2012-12-13
US8420619B2 (en) 2013-04-16
US8242093B2 (en) 2012-08-14
WO2009100253A1 (en) 2009-08-13
EP2247258A4 (en) 2012-01-18

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Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20140110

FZDE Dead

Effective date: 20160205