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

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18Growth factors; Growth regulators
    • A61K38/185Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0048Eye, e.g. artificial tears
    • A61K9/0051Ocular inserts, ocular implants
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2799/00Uses of viruses
    • C12N2799/02Uses of viruses as vector
    • C12N2799/021Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025Uses 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)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (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 (cg-RX-API-DMAC7.html)
EP (1) EP2247258A4 (cg-RX-API-DMAC7.html)
JP (1) JP2011511090A (cg-RX-API-DMAC7.html)
CN (1) CN101980668A (cg-RX-API-DMAC7.html)
AU (1) AU2009212309B2 (cg-RX-API-DMAC7.html)
CA (1) CA2713755A1 (cg-RX-API-DMAC7.html)
WO (1) WO2009100253A1 (cg-RX-API-DMAC7.html)

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
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
CN105120901A (zh) 2012-07-11 2015-12-02 宾夕法尼亚大学托管会 Rpgr x染色体连锁视网膜退化的aav介导的基因治疗
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
WO2016164920A1 (en) * 2015-04-09 2016-10-13 Cornell University Gene therapy to prevent reactions to allergens
CA3004807C (en) * 2015-12-04 2022-02-22 Universite Pierre Et Marie Curie (Paris 6) Promoters and uses thereof
US20190269755A1 (en) * 2016-04-29 2019-09-05 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
CA3199230A1 (en) * 2020-10-22 2022-04-28 Visgenx, Inc. Elovl2 constructs for human gene therapy
JP2025540499A (ja) * 2022-12-05 2025-12-11 ザ ユナイテッド ステイツ オブ アメリカ, アズ リプレゼンテッド バイ ザ セクレタリー, デパートメント オブ ヘルス アンド ヒューマン サービシーズ 色素上皮由来因子ペプチドおよび網膜変性症を処置するための使用

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
ATE246935T1 (de) * 1997-06-11 2003-08-15 Acorda Therapeutics Zns neuroregenerative zusammensetzungen und deren verwendungen
WO2001072959A2 (en) 2000-03-01 2001-10-04 Auburn University Plastid transformation vectors for expressing human proteins in plants
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
WO2000054813A2 (en) * 1999-03-15 2000-09-21 Chiron Corporation Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
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
CA2401855A1 (en) 2000-02-29 2001-09-07 Auburn University Genetic engineering of drought tolerance via a plastid genome
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
EP1885173A4 (en) 2005-05-27 2009-02-18 Univ Central Florida CHLOROPLASTE DEVELOPED FOR THE EXPRESSION OF 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

Also Published As

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

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Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20140110

FZDE Dead

Effective date: 20160205