WO2001058494A3 - Gene therapy for treating ocular-related disorders - Google Patents

Gene therapy for treating ocular-related disorders Download PDF

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Publication number
WO2001058494A3
WO2001058494A3 PCT/US2001/004203 US0104203W WO0158494A3 WO 2001058494 A3 WO2001058494 A3 WO 2001058494A3 US 0104203 W US0104203 W US 0104203W WO 0158494 A3 WO0158494 A3 WO 0158494A3
Authority
WO
WIPO (PCT)
Prior art keywords
nucleic acid
acid sequence
sequence encoding
ocular
gene therapy
Prior art date
Application number
PCT/US2001/004203
Other languages
French (fr)
Other versions
WO2001058494A2 (en
Inventor
Imre Kovesdi
Douglas E Brough
Duncan L Mcvey
Lisa Wei
Original Assignee
Genvec Inc
Imre Kovesdi
Douglas E Brough
Duncan L Mcvey
Lisa Wei
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
Priority claimed from US09/599,997 external-priority patent/US6821775B1/en
Application filed by Genvec Inc, Imre Kovesdi, Douglas E Brough, Duncan L Mcvey, Lisa Wei filed Critical Genvec Inc
Priority to EP01909022A priority Critical patent/EP1253948A2/en
Priority to AU36818/01A priority patent/AU780634C/en
Priority to CA002398571A priority patent/CA2398571A1/en
Priority to JP2001557602A priority patent/JP2003522160A/en
Publication of WO2001058494A2 publication Critical patent/WO2001058494A2/en
Publication of WO2001058494A3 publication Critical patent/WO2001058494A3/en
Priority to US10/211,701 priority patent/US20030045498A1/en
Priority to AU2005202935A priority patent/AU2005202935A1/en
Priority to US11/610,364 priority patent/US20070098692A1/en
Priority to US12/237,036 priority patent/US20090018100A1/en

Links

Classifications

    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/71—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81—Protease inhibitors
    • C07K14/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
    • C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The present invention is directed to a method of prophylactically or therapeutically treating an animal for at least one ocular-related disorder, e.g., ocular neovascularization or age-related macular degeneration. The method comprises contacting an ocular cell with an expression vector comprising a nucleic acid sequence encoding an inhibitor of angiogenesis and the same or different nucleic acid sequence encoding a neurotrophic agent. The method also can comprise contacting an ocular cell with different expression vectors, each comprising a nucleic acid sequence encoding an inhibitor of angiogenesis and/or a nucleic acid sequence encoding a neurotrophic agent. In addition, the present invention provides a viral vector comprising a nucleic acid sequence encoding pigment epithelium-derived factor (PEDF) or a therapeutic fragment thereof.
PCT/US2001/004203 2000-02-11 2001-02-09 Gene therapy for treating ocular-related disorders WO2001058494A2 (en)

Priority Applications (8)

Application Number Priority Date Filing Date Title
EP01909022A EP1253948A2 (en) 2000-02-11 2001-02-09 Gene therapy for treating ocular-related disorders
AU36818/01A AU780634C (en) 2000-02-11 2001-02-09 Gene Therapy for treating ocular-related disorders
CA002398571A CA2398571A1 (en) 2000-02-11 2001-02-09 Materials and methods for treating ocular-related disorders
JP2001557602A JP2003522160A (en) 2000-02-11 2001-02-09 Gene therapy for the treatment of eye-related disorders
US10/211,701 US20030045498A1 (en) 2000-02-11 2002-08-02 Materials and methods for treating ocular-related disorders
AU2005202935A AU2005202935A1 (en) 2000-02-11 2005-07-06 Gene therapy for treating ocular-related disorders
US11/610,364 US20070098692A1 (en) 2000-02-11 2006-12-13 Materials and methods for treating ocular-related disorders
US12/237,036 US20090018100A1 (en) 2000-02-11 2008-09-24 Materials and methods for treating ocular-related disorders

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US18174300P 2000-02-11 2000-02-11
US60/181,743 2000-02-11
US09/599,997 2000-06-23
US09/599,997 US6821775B1 (en) 2000-02-11 2000-06-23 Viral vector encoding pigment epithelium-derived factor
US22833700P 2000-08-28 2000-08-28
US60/228,337 2000-08-28

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
US09/599,997 Continuation-In-Part US6821775B1 (en) 2000-02-11 2000-06-23 Viral vector encoding pigment epithelium-derived factor

Related Child Applications (1)

Application Number Title Priority Date Filing Date
US10/211,701 Continuation US20030045498A1 (en) 2000-02-11 2002-08-02 Materials and methods for treating ocular-related disorders

Publications (2)

Publication Number Publication Date
WO2001058494A2 WO2001058494A2 (en) 2001-08-16
WO2001058494A3 true WO2001058494A3 (en) 2002-04-04

Family

ID=27391455

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2001/004203 WO2001058494A2 (en) 2000-02-11 2001-02-09 Gene therapy for treating ocular-related disorders

Country Status (5)

Country Link
EP (1) EP1253948A2 (en)
JP (1) JP2003522160A (en)
AU (1) AU780634C (en)
CA (1) CA2398571A1 (en)
WO (1) WO2001058494A2 (en)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7470659B2 (en) 2001-12-07 2008-12-30 The Regents Of The University Of California Methods to increase reverse cholesterol transport in the retinal pigment epithelium (RPE) and Bruch's membrane (BM)
AU2003297607A1 (en) * 2002-12-02 2004-06-23 Genvec, Inc Materials and methods for treating ocular-related disorders
US20040166091A1 (en) * 2003-02-24 2004-08-26 Genvec, Inc. Materials and methods for treating disorders of the ear
WO2006054278A2 (en) 2004-11-16 2006-05-26 Yeda Research And Development Co. Ltd. At The Weizmann Institute Of Science Variants of pigment epithelium derived factor and uses thereof
ATE513052T1 (en) * 2005-04-18 2011-07-15 Inst Nat Sante Rech Med METHOD AND DEVICE FOR DELIVERING A THERAPEUTIC PRODUCT TO THE EYEBALL OF A PATIENT
JP2007291009A (en) * 2006-04-25 2007-11-08 Seitai Shigen Laboratory Inc Agent for inhibiting intimal thickening
WO2009126894A2 (en) * 2008-04-11 2009-10-15 Massachusetts Eye And Ear Infirmary Methods and compositions for the diagnosis and treatment of angiogenic disorders
US9790264B2 (en) 2012-06-25 2017-10-17 The Brigham And Women's Hospital, Inc. Compounds and methods for modulating pharmacokinetics
ES2651113T3 (en) * 2012-06-25 2018-01-24 The Brigham And Women's Hospital, Inc. Targeted therapeutic treatments
EP2968612B1 (en) 2013-03-14 2018-02-14 Asklepios Biopharmaceutical, Inc. Modified soluble vegf receptor-1 genes and vectors for gene therapy
TWI710635B (en) 2014-10-09 2020-11-21 美商珍維克公司 Adenoviral vector encoding human atonal homolog-1 (hath1)
US20210207166A1 (en) * 2018-01-23 2021-07-08 Southern Eye Equipment Expression vector and method
WO2019246141A1 (en) * 2018-06-19 2019-12-26 Cella Therapeutics, Llc DRUG DELIVERY SYSTEMS COMPRISING A NEUROTROPHIC AGENT, AN APOPTOSIS SIGNALING FRAGMENT INHIBITOR (FAS) OR FAS LIGAND (FASL) INHIBITOR, A TUMOR NECROSIS FACTOR-α (TNF-α) OR TNF RECEPTOR INHIBITOR, A MITOCHONDRIAL PEPTIDE, AN OLIGONUCLEOTIDE, A CHEMOKINE INHIBITOR, OR A CYSTEINE-ASPARTIC PROTEASE
EP3810082A1 (en) * 2018-06-19 2021-04-28 Cella Therapeutics, LLC Sustained-release drug delivery systems comprising an intraocular pressure lowering agent, a cnp compound, an npr-b compound, a tie-2 agonist, or neurotrophic agent for use for treating glaucoma or ocular hypertension
US20230094423A1 (en) * 2019-12-18 2023-03-30 Cella Therapeutics, Llc Drug delivery systems comprising a neurotrophic agent, an apoptosis signaling fragment inhibitor (fas) or fas ligand (fasl) inhibitor, a tumor necrosis factor-alpha (tnf-alpha) or tnf receptor inhibitor, a mitochondrial peptide, an oligonucleotide, a chemokine inhibitor, or a cysteine-aspartic protease inhibitor

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5461749A (en) * 1994-05-31 1995-10-31 Minnesota Mining And Manufacturing Company Floor mop and cleaning system
WO1995033480A1 (en) * 1994-06-07 1995-12-14 The Government Of The United States Of America, Re Pigment epithelium-derived factor: characterization, genomic organization and sequence of the pedf gene
US5840686A (en) * 1992-09-24 1998-11-24 The United States Of America As Represented By The Department Of Health And Human Services Pigment epithelium-derived factor: characterization of its novel biological activity and sequences encoding and expressing the protein and methods of use
WO1999026480A1 (en) * 1997-11-20 1999-06-03 Genetix Pharmaceuticals, Inc. Anti-angiogenic gene therapy vectors and their use in treating angiogenesis-related diseases
WO2000054813A2 (en) * 1999-03-15 2000-09-21 Chiron Corporation Use of recombinant gene delivery vectors for treating or preventing diseases of the eye

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5904144A (en) * 1996-03-22 1999-05-18 Cytotherapeutics, Inc. Method for treating ophthalmic diseases
CA2298018C (en) * 1997-07-23 2008-12-16 Northwestern University Methods and compositions for inhibiting angiogenesis
WO1999015686A1 (en) * 1997-09-23 1999-04-01 Genvec, Inc. Plasmids for construction of eukaryotic viral vectors

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5840686A (en) * 1992-09-24 1998-11-24 The United States Of America As Represented By The Department Of Health And Human Services Pigment epithelium-derived factor: characterization of its novel biological activity and sequences encoding and expressing the protein and methods of use
US5461749A (en) * 1994-05-31 1995-10-31 Minnesota Mining And Manufacturing Company Floor mop and cleaning system
WO1995033480A1 (en) * 1994-06-07 1995-12-14 The Government Of The United States Of America, Re Pigment epithelium-derived factor: characterization, genomic organization and sequence of the pedf gene
WO1999026480A1 (en) * 1997-11-20 1999-06-03 Genetix Pharmaceuticals, Inc. Anti-angiogenic gene therapy vectors and their use in treating angiogenesis-related diseases
WO2000054813A2 (en) * 1999-03-15 2000-09-21 Chiron Corporation Use of recombinant gene delivery vectors for treating or preventing diseases of the eye

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
D. W. DAWSON ET AL: "Pigment Epithelium-Derive Factor: A Potent Inhibitor of Angiogenesis", SCIENCE, vol. 285, 9 July 1999 (1999-07-09), pages 245 - 248, XP002135983 *
See also references of EP1253948A2 *
STEELE F R ET AL: "PIGMENT EPITHELIUM-DERIVED FACTOR: NEUROTROPHIC ACTIVITY AND IDENTIFICATION AS A MEMBER OF THE SERINE PROTEASE INHIBITOR GENE FAMILY", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF USA, NATIONAL ACADEMY OF SCIENCE. WASHINGTON, US, vol. 90, 1 February 1993 (1993-02-01), pages 1526 - 1530, XP000606398, ISSN: 0027-8424 *

Also Published As

Publication number Publication date
AU3681801A (en) 2001-08-20
AU780634B2 (en) 2005-04-07
AU780634C (en) 2006-02-23
WO2001058494A2 (en) 2001-08-16
JP2003522160A (en) 2003-07-22
CA2398571A1 (en) 2001-08-16
EP1253948A2 (en) 2002-11-06

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