JP2011511090A - 治療用タンパク質をコードする発現ベクターの硝子体内投与による光受容体のレスキュー方法 - Google Patents

治療用タンパク質をコードする発現ベクターの硝子体内投与による光受容体のレスキュー方法 Download PDF

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JP2011511090A
JP2011511090A JP2010546007A JP2010546007A JP2011511090A JP 2011511090 A JP2011511090 A JP 2011511090A JP 2010546007 A JP2010546007 A JP 2010546007A JP 2010546007 A JP2010546007 A JP 2010546007A JP 2011511090 A JP2011511090 A JP 2011511090A
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cells
aav
vector
retinal
eye
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JP2011511090A5 (https=
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レイモンド ティー． バータス
キャシー エム． ビショップ
メディ ガスミ
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セレジーン インコーポレイテッド
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    • 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

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  • 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)
JP2010546007A 2008-02-07 2009-02-05 治療用タンパク質をコードする発現ベクターの硝子体内投与による光受容体のレスキュー方法 Ceased JP2011511090A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US2699008P 2008-02-07 2008-02-07
US9322808P 2008-08-29 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
JP2011511090A true JP2011511090A (ja) 2011-04-07
JP2011511090A5 JP2011511090A5 (https=) 2013-03-28

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ID=40939054

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JP2010546007A Ceased JP2011511090A (ja) 2008-02-07 2009-02-05 治療用タンパク質をコードする発現ベクターの硝子体内投与による光受容体のレスキュー方法

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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
JP2002539176A (ja) * 1999-03-15 2002-11-19 カイロン コーポレイション 眼の疾患を処置または予防するための組換え遺伝子送達ベクターの使用

Family Cites Families (12)

* 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
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
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

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
JP2002539176A (ja) * 1999-03-15 2002-11-19 カイロン コーポレイション 眼の疾患を処置または予防するための組換え遺伝子送達ベクターの使用

Also Published As

Publication number Publication date
AU2009212309A1 (en) 2009-08-13
AU2009212309B2 (en) 2014-04-03
CN101980668A (zh) 2011-02-23
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
CA2713755A1 (en) 2009-08-13
EP2247258A4 (en) 2012-01-18

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