CN101980668A - 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 - Google Patents

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 Download PDF

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Publication number
CN101980668A
CN101980668A CN2009801043603A CN200980104360A CN101980668A CN 101980668 A CN101980668 A CN 101980668A CN 2009801043603 A CN2009801043603 A CN 2009801043603A CN 200980104360 A CN200980104360 A CN 200980104360A CN 101980668 A CN101980668 A CN 101980668A
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CN
China
Prior art keywords
cell
aav
retina
eye
vector
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Pending
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CN2009801043603A
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English (en)
Chinese (zh)
Inventor
R·T·巴尔图斯
K·M·毕晓普
M·加斯米
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Ceregene Inc
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Ceregene Inc
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Application filed by Ceregene Inc filed Critical Ceregene Inc
Publication of CN101980668A publication Critical patent/CN101980668A/zh
Pending legal-status Critical Current

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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, ocular 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)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Psychology (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN2009801043603A 2008-02-07 2009-02-05 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 Pending CN101980668A (zh)

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
CN101980668A true CN101980668A (zh) 2011-02-23

Family

ID=40939054

Family Applications (1)

Application Number Title Priority Date Filing Date
CN2009801043603A Pending CN101980668A (zh) 2008-02-07 2009-02-05 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

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)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107937341A (zh) * 2011-02-25 2018-04-20 国立研究开发法人理化学研究所 视网膜色素上皮细胞片层的生产方法

Families Citing this family (11)

* 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
CA2878171C (en) * 2012-07-11 2021-04-27 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
WO2015082690A1 (en) * 2013-12-06 2015-06-11 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the retinal pigment epithelium of a subject
US10293059B2 (en) * 2015-04-09 2019-05-21 Cornell University Gene therapy to prevent reactions to allergens
US11390866B2 (en) * 2015-12-04 2022-07-19 Sorbonne Université Promoters and uses thereof
EP3448411A1 (en) * 2016-04-29 2019-03-06 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
CN116916937A (zh) * 2020-10-22 2023-10-20 维斯基因股份有限公司 用于人类基因疗法的elovl2构建体
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 (3)

* 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
US20050208103A1 (en) * 1999-01-05 2005-09-22 Adamis Anthony P Targeted transscleral controlled release drug delivery to the retina and choroid
US20060188481A1 (en) * 2005-02-22 2006-08-24 Saitama Medical School Methods for prophylactically or therapeutically treating an animal for an ocular-related disorder

Family Cites Families (11)

* 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
WO1998056404A1 (en) * 1997-06-11 1998-12-17 Acorda Therapeutics Cns neuroregenerative compositions and methods of use
CA2367375A1 (en) * 1999-03-15 2000-09-21 Chiron Corporation Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
CA2401855A1 (en) 2000-02-29 2001-09-07 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
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
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 (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050208103A1 (en) * 1999-01-05 2005-09-22 Adamis Anthony P Targeted transscleral controlled release drug delivery to the retina and choroid
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
US20060188481A1 (en) * 2005-02-22 2006-08-24 Saitama Medical School Methods for prophylactically or therapeutically treating an animal for an ocular-related disorder

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107937341A (zh) * 2011-02-25 2018-04-20 国立研究开发法人理化学研究所 视网膜色素上皮细胞片层的生产方法

Also Published As

Publication number Publication date
WO2009100253A8 (en) 2010-08-19
AU2009212309B2 (en) 2014-04-03
WO2009100253A1 (en) 2009-08-13
US8420619B2 (en) 2013-04-16
AU2009212309A1 (en) 2009-08-13
US8242093B2 (en) 2012-08-14
US20090202505A1 (en) 2009-08-13
EP2247258A1 (en) 2010-11-10
US20120316112A1 (en) 2012-12-13
CA2713755A1 (en) 2009-08-13
JP2011511090A (ja) 2011-04-07
EP2247258A4 (en) 2012-01-18

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Application publication date: 20110223