US20130323302A1 - Treatment of amd using aav sflt-1 - Google Patents

Treatment of amd using aav sflt-1 Download PDF

Info

Publication number
US20130323302A1
US20130323302A1 US13/889,275 US201313889275A US2013323302A1 US 20130323302 A1 US20130323302 A1 US 20130323302A1 US 201313889275 A US201313889275 A US 201313889275A US 2013323302 A1 US2013323302 A1 US 2013323302A1
Authority
US
United States
Prior art keywords
seq
sflt
sequence
pharmaceutical composition
aspects
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
US13/889,275
Other languages
English (en)
Inventor
Ian J. Constable
Elizabeth P. Rakoczy
Chooi-May Lai
Thomas W. Chalberg, JR.
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.)
Avalanche Australia Pty Ltd
Original Assignee
Avalanche Australia Pty Ltd
Lions Eye Institute of Western Australia Inc
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 Avalanche Australia Pty Ltd, Lions Eye Institute of Western Australia Inc filed Critical Avalanche Australia Pty Ltd
Priority to US13/889,275 priority Critical patent/US20130323302A1/en
Assigned to AVALANCHE AUSTRALIA PTY LTD. reassignment AVALANCHE AUSTRALIA PTY LTD. ASSIGNMENT OF ASSIGNORS INTEREST (SEE DOCUMENT FOR DETAILS). Assignors: CHALBERG, THOMAS W., JR.
Assigned to LIONS EYE INSTITUTE LIMITED reassignment LIONS EYE INSTITUTE LIMITED ASSIGNMENT OF ASSIGNORS INTEREST (SEE DOCUMENT FOR DETAILS). Assignors: LAI, Chooi-may, CONSTABLE, IAN J., RAKOCZY, ELIZABETH P.
Publication of US20130323302A1 publication Critical patent/US20130323302A1/en
Priority to US14/281,749 priority patent/US9943573B2/en
Priority to US14/281,765 priority patent/US20140341977A1/en
Priority to US14/281,783 priority patent/US20140371438A1/en
Assigned to AVALANCHE AUSTRALIA PTY LTD. reassignment AVALANCHE AUSTRALIA PTY LTD. ASSIGNMENT OF ASSIGNORS INTEREST (SEE DOCUMENT FOR DETAILS). Assignors: LIONS EYE INSTITUTE LIMITED
Assigned to LIONS EYE INSTITUTE LIMITED reassignment LIONS EYE INSTITUTE LIMITED CORRECTIVE ASSIGNMENT TO CORRECT THE THE LISTING OF ASSIGNOR NAME. PREVIOUSLY RECORDED ON REEL 030457 FRAME 0046. ASSIGNOR(S) HEREBY CONFIRMS THE ASSIGNOR'S NAME. Assignors: RAKOCZY, P. ELIZABETH, LAI, Chooi-may, CONSTABLE, IAN J.
Priority to US15/851,650 priority patent/US10004788B2/en
Priority to US15/961,654 priority patent/US20180311319A1/en
Priority to US17/328,705 priority patent/US20220111015A1/en
Abandoned legal-status Critical Current

Links

Images

Classifications

    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45—Transferases (2)
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61B—DIAGNOSIS; SURGERY; IDENTIFICATION
    • A61B3/00—Apparatus for testing the eyes; Instruments for examining the eyes
    • A61B3/02—Subjective types, i.e. testing apparatus requiring the active assistance of the patient
    • A61B3/028—Subjective types, i.e. testing apparatus requiring the active assistance of the patient for testing visual acuity; for determination of refraction, e.g. phoropters
    • A61B3/032—Devices for presenting test symbols or characters, e.g. test chart projectors
    • 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
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • 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
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/179—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K49/00—Preparations for testing in vivo
    • A61K49/0004—Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
    • 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
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/10—Ophthalmic agents for accommodation disorders, e.g. myopia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/14—Vasoprotectives; Antihaemorrhoidals; Drugs for varicose therapy; Capillary stabilisers
    • 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
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/08—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from viruses
    • C07K16/081—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from viruses from DNA viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/22—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against growth factors ; against growth regulators
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/10—Protein-tyrosine kinases (2.7.10)
    • C12Y207/10001—Receptor protein-tyrosine kinase (2.7.10.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/10—Protein-tyrosine kinases (2.7.10)
    • C12Y207/10002—Non-specific protein-tyrosine kinase (2.7.10.2), i.e. spleen tyrosine kinase
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/24—Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/55—Fab or Fab'
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/73—Inducing cell death, e.g. apoptosis, necrosis or inhibition of cell proliferation
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/32—Fusion polypeptide fusions with soluble part of a cell surface receptor, "decoy receptors"
    • 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
    • C12N2710/14011—Baculoviridae
    • C12N2710/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14044—Chimeric viral vector comprising heterologous viral elements for production of another viral vector
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/10—Protein-tyrosine kinases (2.7.10)

Definitions

  • VEGF vascular endothelial growth factor
  • VEGFR VEGF receptor
  • PDGF platelet-derived growth factor
  • HIF hypoxia inducible factor
  • Ang angiopoietin
  • MAPK mitogen-activated protein kinases
  • the disclosure provides for a nucleic acid encoding the sFLT-1 which is operatively linked to a promoter selected from the group consisting of: cytomegalovirus (CMV) promoter, Rous sarcoma virus (RSV) promoter, MMT promoter, EF-1 alpha promoter, UB6 promoter, chicken beta-actin promoter, CAG promoter, RPE65 promoter and opsin promoter.
  • a promoter selected from the group consisting of: cytomegalovirus (CMV) promoter, Rous sarcoma virus (RSV) promoter, MMT promoter, EF-1 alpha promoter, UB6 promoter, chicken beta-actin promoter, CAG promoter, RPE65 promoter and opsin promoter.
  • the disclosure provides for the human subject as resistant to treatment with VEGF inhibitors.
  • the disclosure provides for administering of the pharmaceutical composition at a frequency less than 3 times a year in the human subject.
  • the disclosure provides for a pharmaceutical composition followed by gas/fluid exchange in the vitreous chamber.
  • the disclosure provides for a unit dose of a pharmaceutical composition
  • a pharmaceutical composition comprising recombinant viruses of 1 ⁇ 10 6 to 1 ⁇ 10 15 vector genomes, wherein the recombinant viruses comprise a nucleic acid encoding sFLT-1 operatively linked to a promoter.
  • the unit dose of the pharmaceutical composition comprises 1 ⁇ 10 10 to 3 ⁇ 10 12 vector genomes.
  • the disclosure provides for the nucleic acid for use, wherein the pharmaceutical composition is capable of elevating levels of sFLT-1 protein in the vitreous of the human subject after at least 72 hours after administration of said pharmaceutical composition to said human subject, compared to levels of sFLT-1 protein in the vitreous of said human prior to said administration.
  • VEGF has been implicated in eye diseases, including but not limited to ischemic retinopathy, intraocular neovascularization, age-related macular degeneration (AMD), wet-AMD, dry-AMD, retinal neovascularization, diabetic macular edema, diabetic retina ischemia, diabetic retinal edema, proliferative diabetic retinopathy, retinal vein occlusion, central retinal vein occlusion, branched retinal vein occlusion.
  • AMD age-related macular degeneration
  • AMD age-related macular degeneration
  • AMD age-related macular degeneration
  • AMD age-AMD
  • dry-AMD dry-AMD
  • retinal neovascularization diabetic macular edema
  • diabetic retina ischemia diabetic retinal edema
  • proliferative diabetic retinopathy retinal vein occlusion
  • central retinal vein occlusion central retinal vein occlusion
  • recombinant viruses of the disclosure are at most about 1 ⁇ 10 1 , 1 ⁇ 10 2 , 1 ⁇ 10 3 , 1 ⁇ 10 4 , 1 ⁇ 10 5 , 1 ⁇ 10 6 , 1 ⁇ 10 7 , 1 ⁇ 10 8 , 1 ⁇ 10 10 , 1 ⁇ 10 11 , 1 ⁇ 10 12 , 1 ⁇ 10 13 , 1 ⁇ 10 14 , 1 ⁇ 10 15 , 1 ⁇ 10 16 , 1 ⁇ 10 17 , and 1 ⁇ 10 18 vector genomes.
  • the nucleic acid may be delivered without the use of a virus (i.e. with a non-viral vector), and may be measured as the quantity of nucleic acid.
  • a virus i.e. with a non-viral vector
  • any suitable amount of nucleic acid may be used with the compositions and methods of this disclosure.
  • a polyA or termination sequence may include but is not limited to sequences such as SEQ ID No. 49, SEQ ID No. 50, SEQ ID No. 51, SEQ ID No. 52, SEQ ID No. 53, SEQ ID No. 54, and SEQ ID No. 55.
  • 5′ UTRs sequences may comprise various sequence elements.
  • the present disclosure provides for 5′ UTR sequences that may include but are not limited to sequence elements such as one or more ribosome binding sites (RBS), linker sequences, spacer sequences, regulatory sequences, regulatory response elements, riboswitches, sequences that promote or inhibit translation initiation, regulatory sequences for mRNA transport or variants and/or combinations thereof.
  • RBS ribosome binding sites
  • linker sequences spacer sequences
  • regulatory sequences regulatory response elements
  • riboswitches sequences that promote or inhibit translation initiation, regulatory sequences for mRNA transport or variants and/or combinations thereof.
  • the recombinant virus and/or plasmid used to generate recombinant virus comprises nucleic acid elements in the following order: a) a first ITR sequence; b) a first linker sequence; c) a promoter sequence; d) a second linker sequence; e) an intron sequence; f) a third linker sequence; g) a first UTR sequence; h) a sequence encoding a VEGF inhibitor; i) a second UTR sequence; j) a fourth linker sequence; k) a poly A sequence; l) a fifth linker sequence; and m) a second ITR sequence.
  • salts formed with cations such as sodium, potassium, ammonium, magnesium, calcium, polyamides such as spermine and spermidine, and the like
  • inorganic acids for example hydrochloric acid, hydrobromic acid, sulfuric acid, phosphoric acid, nitric acid and the like
  • salts formed with organic acids such as, for example, acetic acid, oxalic acid, tartaric acid, succinic acid, maleic acid, fumaric acid, gluconic acid, citric acid, malic acid, ascorbic acid, benzoic acid, tannic acid, palmitic acid, alginic acid, polyglutamic acid, napthalenesulfonic acid, methanesulfonic acid, p-toluenesulfonic acid, naphthalenedisulfonic acid, polygalacturonic
  • the nucleic acid may be delivered without the use of a virus (i.e. with a non-viral vector), and may be measured as the quantity of nucleic acid.
  • a virus i.e. with a non-viral vector
  • any suitable amount of nucleic acid may be used with the compositions and methods of this disclosure.
  • CNV choroidal neovascularization
  • One or multiple (e.g. 2, 3, or more) blebs can be created.
  • the total volume of bleb or blebs created by the methods and systems of the disclosure cannot exceed the fluid volume of the eye, for example about 4 ml in a typical human subject.
  • the total volume of each individual bleb is preferably at about 0.1-0.2 ml.
  • a patient's best corrected visual acuity improves by 1, 2 3, 4, 5 or more lines.
  • sFLT-1 was first identified in human umbilical vein endothelial cells (HUVEC), but it has since been found to occur naturally in the placenta and circulating systematically in pregnant women.
  • the sFLT-1 used in generating rAAV.sFlt-1 contains an open reading frame encoding only the first six extracellular immunoglobulin-like domains of the full length membrane-spanning FLT-1, followed by a unique 31-amino acid long C-terminal extension, representing the alternatively splices, secreted soluble FLT-1 isoform described earlier.
  • Frag001m was formed from the following sequential nucleic acid elements which were chemically synthesized by Blue Heron Biotech, LLC (Bothell, Wash.) and cloned into a BHKan backbone: an ITR (AAV serotype 2), CMV-IE promoter, chimeric intron, 5′ untranslated region (UTR), sFlt-1 coding sequence, SV40 polyA region, ITR (AAV serotype 2).
  • the plasmid V109-pFB-AAV-CMV-SV40pA-Kan was obtained from Virovek, Inc. (Hayward, Calif.).
  • results of the studies for rAAV.sFlt-1 and rAAV(bv).sFlt-1 are presented in FIG. 25A and FIG. 25B .
  • the concentration of sFlt-1 protein expressed by HEK293 cells 72 hours after transduction with rAAV.sFlt-1 and rAAV(bv).sFlt-1 ranged from 100-1,000 pg/mL at an MOI of 1 ⁇ 10 4 , 100-10,000 pg/mL at an MOI of 1 ⁇ 10 5 and 1,000-10,000 pg/mL at an MOI of 1 ⁇ 10 6 .
  • mice from either the rAAV.sFlt-1 or control treatment groups were then assessed at one week and one month post-injection for the presence of immune cells (leucocytes, macrophages and B- and T-lymphocytes).
  • Flow cytometric examination of the posterior eye cup showed that at one week post-injection there was a statistically significant increase in CD45+ leucocytes (6.6-fold increase compared to control; P ⁇ 0.05) and CD11b+ macrophages (5.7-fold increase compared to control; P ⁇ 0.036).
  • CD19+, CD8+ and CD4+ B- and T-lymphocytes
  • Plasma hsFLT-1 levels in the monkeys did not show any trend at the different sampling times ( FIG. 3B ). This suggests that the injection of rAAV.sFlt-1 did not have an obvious effect on the plasma hsFLT-1 level. The fluctuating levels did not have any effect on the well-being of the monkeys.

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Immunology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Diabetes (AREA)
  • Cell Biology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Cardiology (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • Endocrinology (AREA)
  • Urology & Nephrology (AREA)
US13/889,275 2012-05-15 2013-05-07 Treatment of amd using aav sflt-1 Abandoned US20130323302A1 (en)

Priority Applications (7)

Application Number Priority Date Filing Date Title
US13/889,275 US20130323302A1 (en) 2012-05-15 2013-05-07 Treatment of amd using aav sflt-1
US14/281,749 US9943573B2 (en) 2012-05-15 2014-05-19 Treatment of ocular neovascularization using anti-VEGF proteins
US14/281,765 US20140341977A1 (en) 2012-05-15 2014-05-19 Treatment of amd using aav sflt-1
US14/281,783 US20140371438A1 (en) 2012-05-15 2014-05-19 Treatment of amd using aav sflt-1
US15/851,650 US10004788B2 (en) 2012-05-15 2017-12-21 Treatment of ocular neovascularization using anti-VEGF proteins
US15/961,654 US20180311319A1 (en) 2012-05-15 2018-04-24 Treatment of ocular neovascularization using anti-vegf proteins
US17/328,705 US20220111015A1 (en) 2012-05-15 2021-05-24 Treatment of ocular neovascularization using anti-vegf proteins

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201261647461P 2012-05-15 2012-05-15
US201261670535P 2012-07-11 2012-07-11
US201261678555P 2012-08-01 2012-08-01
US201261691660P 2012-08-21 2012-08-21
US201361775440P 2013-03-08 2013-03-08
US13/889,275 US20130323302A1 (en) 2012-05-15 2013-05-07 Treatment of amd using aav sflt-1

Related Child Applications (3)

Application Number Title Priority Date Filing Date
US14/281,765 Continuation US20140341977A1 (en) 2012-05-15 2014-05-19 Treatment of amd using aav sflt-1
US14/281,783 Division US20140371438A1 (en) 2012-05-15 2014-05-19 Treatment of amd using aav sflt-1
US14/281,749 Continuation US9943573B2 (en) 2012-05-15 2014-05-19 Treatment of ocular neovascularization using anti-VEGF proteins

Publications (1)

Publication Number Publication Date
US20130323302A1 true US20130323302A1 (en) 2013-12-05

Family

ID=49584435

Family Applications (7)

Application Number Title Priority Date Filing Date
US13/889,275 Abandoned US20130323302A1 (en) 2012-05-15 2013-05-07 Treatment of amd using aav sflt-1
US14/281,765 Abandoned US20140341977A1 (en) 2012-05-15 2014-05-19 Treatment of amd using aav sflt-1
US14/281,749 Active US9943573B2 (en) 2012-05-15 2014-05-19 Treatment of ocular neovascularization using anti-VEGF proteins
US14/281,783 Abandoned US20140371438A1 (en) 2012-05-15 2014-05-19 Treatment of amd using aav sflt-1
US15/851,650 Active US10004788B2 (en) 2012-05-15 2017-12-21 Treatment of ocular neovascularization using anti-VEGF proteins
US15/961,654 Abandoned US20180311319A1 (en) 2012-05-15 2018-04-24 Treatment of ocular neovascularization using anti-vegf proteins
US17/328,705 Pending US20220111015A1 (en) 2012-05-15 2021-05-24 Treatment of ocular neovascularization using anti-vegf proteins

Family Applications After (6)

Application Number Title Priority Date Filing Date
US14/281,765 Abandoned US20140341977A1 (en) 2012-05-15 2014-05-19 Treatment of amd using aav sflt-1
US14/281,749 Active US9943573B2 (en) 2012-05-15 2014-05-19 Treatment of ocular neovascularization using anti-VEGF proteins
US14/281,783 Abandoned US20140371438A1 (en) 2012-05-15 2014-05-19 Treatment of amd using aav sflt-1
US15/851,650 Active US10004788B2 (en) 2012-05-15 2017-12-21 Treatment of ocular neovascularization using anti-VEGF proteins
US15/961,654 Abandoned US20180311319A1 (en) 2012-05-15 2018-04-24 Treatment of ocular neovascularization using anti-vegf proteins
US17/328,705 Pending US20220111015A1 (en) 2012-05-15 2021-05-24 Treatment of ocular neovascularization using anti-vegf proteins

Country Status (27)

Country Link
US (7) US20130323302A1 (ja)
EP (3) EP2849802B1 (ja)
JP (5) JP6466322B2 (ja)
KR (3) KR102218067B1 (ja)
CN (2) CN104994882A (ja)
AU (3) AU2013263159B2 (ja)
BR (1) BR112014028633A8 (ja)
CA (1) CA2873628C (ja)
CY (1) CY1124495T1 (ja)
DK (1) DK3501549T3 (ja)
ES (2) ES2967616T3 (ja)
HK (1) HK1207568A1 (ja)
HR (1) HRP20211453T1 (ja)
HU (1) HUE056327T2 (ja)
IL (2) IL235679B (ja)
LT (1) LT3501549T (ja)
MX (2) MX362452B (ja)
NZ (2) NZ702637A (ja)
PL (1) PL3501549T3 (ja)
PT (1) PT3501549T (ja)
RS (1) RS62359B1 (ja)
RU (1) RU2014150340A (ja)
SG (2) SG10201609412QA (ja)
SI (1) SI3501549T1 (ja)
TW (3) TWI775096B (ja)
WO (1) WO2013173129A2 (ja)
ZA (1) ZA201409025B (ja)

Cited By (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9193956B2 (en) 2011-04-22 2015-11-24 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9457103B2 (en) 2010-10-06 2016-10-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
WO2017075335A1 (en) 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
US9681951B2 (en) 2013-03-14 2017-06-20 Edwards Lifesciences Cardiaq Llc Prosthesis with outer skirt and anchors
EP3088527A4 (en) * 2013-12-27 2017-09-06 Mogam Biotechnology Institute Expression vector having improved ability to express gene
US9943573B2 (en) 2012-05-15 2018-04-17 Avalanche Australia Pty Ltd. Treatment of ocular neovascularization using anti-VEGF proteins
US20180142260A1 (en) * 2015-05-08 2018-05-24 Children's Medical Research Institute Promoters for expression of heterologous genes
US10000741B2 (en) 2014-03-17 2018-06-19 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
WO2018170473A1 (en) * 2017-03-17 2018-09-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression
US10166097B2 (en) 2009-09-29 2019-01-01 Edwards Lifesciences Cardiaq Llc Replacement heart valve and method
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
US10335466B2 (en) 2014-11-05 2019-07-02 Voyager Therapeutics, Inc. AADC polynucleotides for the treatment of parkinson's disease
WO2019173434A1 (en) 2018-03-06 2019-09-12 Voyager Therapeutics, Inc. Insect cell manufactured partial self-complementary aav genomes
WO2019217582A1 (en) * 2018-05-08 2019-11-14 Rutgers, The State University Of New Jersey Aav-compatible laminin-linker polymerization proteins
WO2019222444A2 (en) 2018-05-16 2019-11-21 Voyager Therapeutics, Inc. Directed evolution
WO2019222441A1 (en) 2018-05-16 2019-11-21 Voyager Therapeutics, Inc. Aav serotypes for brain specific payload delivery
WO2020010042A1 (en) 2018-07-02 2020-01-09 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
WO2020023612A1 (en) 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
US10570395B2 (en) 2014-11-14 2020-02-25 Voyager Therapeutics, Inc. Modulatory polynucleotides
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
US10584328B2 (en) 2015-12-23 2020-03-10 Adverum Biotechnologies, Inc. Mutant viral capsid libraries and related systems and methods
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
US10597660B2 (en) 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
WO2020072849A1 (en) 2018-10-04 2020-04-09 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
WO2020150556A1 (en) 2019-01-18 2020-07-23 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
WO2020219933A1 (en) 2019-04-26 2020-10-29 Adverum Biotechnologies, Inc. Variant aav capsids for intravitreal delivery
WO2020223274A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
US10883117B2 (en) 2015-03-24 2021-01-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
WO2021030125A1 (en) 2019-08-09 2021-02-18 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
WO2021041485A1 (en) 2019-08-26 2021-03-04 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US11021519B2 (en) 2015-03-02 2021-06-01 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
US11066465B2 (en) 2015-12-30 2021-07-20 Kodiak Sciences Inc. Antibodies and conjugates thereof
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
US11155610B2 (en) 2014-06-28 2021-10-26 Kodiak Sciences Inc. Dual PDGF/VEGF antagonists
US11192925B2 (en) 2016-10-19 2021-12-07 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
US11242530B2 (en) * 2016-12-08 2022-02-08 Shanghai Unicar-Therapy Bio-Medicine Technology Co., Ltd siRNA knocking down human PD-1 and recombinant expression CAR-T vector and their construction methods and applications
WO2022032153A1 (en) 2020-08-06 2022-02-10 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11298041B2 (en) 2016-08-30 2022-04-12 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187473A2 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US11497576B2 (en) 2017-07-17 2022-11-15 Voyager Therapeutics, Inc. Trajectory array guide system
WO2022247917A1 (zh) 2021-05-28 2022-12-01 上海瑞宏迪医药有限公司 衣壳变异的重组腺相关病毒及其应用
US11554180B2 (en) 2016-07-29 2023-01-17 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
US11603542B2 (en) 2017-05-05 2023-03-14 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
US11680249B2 (en) 2017-08-28 2023-06-20 The Regents Of The University Of California Adeno-associated virus capsid variants and methods of use thereof
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
US11752181B2 (en) 2017-05-05 2023-09-12 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
US11759506B2 (en) 2017-06-15 2023-09-19 Voyager Therapeutics, Inc. AADC polynucleotides for the treatment of Parkinson's disease
US11912784B2 (en) 2019-10-10 2024-02-27 Kodiak Sciences Inc. Methods of treating an eye disorder
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US11951121B2 (en) 2016-05-18 2024-04-09 Voyager Therapeutics, Inc. Compositions and methods for treating Huntington's disease

Families Citing this family (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI687225B (zh) * 2014-02-06 2020-03-11 美商健臻公司 用於治療及預防黃斑部病變的組成物及方法
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
SI3122878T1 (sl) * 2014-03-24 2019-05-31 Translate Bio, Inc. MRNA terapija za zdravljenje očesnih bolezni
US10507232B2 (en) 2014-04-02 2019-12-17 University Of Florida Research Foundation, Incorporated Materials and methods for the treatment of latent viral infection
IL292951B1 (en) 2014-05-02 2024-06-01 Genzyme Corp AAV vectors for gene therapy in the central nervous system and retina
GB201502137D0 (en) 2015-02-09 2015-03-25 Ucl Business Plc Treatment
US20180043034A1 (en) * 2015-03-06 2018-02-15 Massachusetts Eye And Ear Infirmary Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene
JP6929230B2 (ja) * 2015-05-14 2021-09-01 セント ジュード チルドレンズ リサーチ ホスピタル インコーポレイテッド スペーサーを含む核酸分子およびその使用の方法
US10016514B2 (en) 2015-05-15 2018-07-10 New Hope Research Foundation Polynucleotides, vectors and methods for insertion and expression of transgenes
US11230720B2 (en) 2015-10-14 2022-01-25 Audentes Therapeutics, Inc. Nucleic acid molecules containing spacers and methods of use thereof
GB201519086D0 (en) * 2015-10-28 2015-12-09 Syncona Partners Llp Gene Therapy
WO2017093935A1 (en) * 2015-12-03 2017-06-08 Friedrich Miescher Institute For Biomedical Research Synp161, a promoter for the specific expression of genes in rod photoreceptors
JP7046828B2 (ja) 2016-04-15 2022-04-04 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 滲出型加齢性黄斑変性の治療のための組成物
WO2017181021A1 (en) * 2016-04-15 2017-10-19 Regenxbio Inc. Treatment of ocular diseases with fully-human post-translationally modified anti-vegf fab
RU2018142273A (ru) * 2016-05-03 2020-06-03 Чжо-хуа Пань Способ улучшения опосредованной вирусами доставки генов в глаз с использованием ингибиторов протеасом
KR102622823B1 (ko) * 2016-06-16 2024-01-10 애드베룸 바이오테크놀로지스, 인코포레이티드 안구 혈관신생을 저하시키기 위한 조성물 및 방법
CN117531025A (zh) * 2016-06-16 2024-02-09 阿德夫拉姆生物技术股份有限公司 使用aav2变体和阿柏西普治疗amd
EP3595687A1 (en) * 2017-03-16 2020-01-22 Lineage Cell Therapeutics, Inc. Methods for measuring therapeutic effects of retinal disease therapies
US20210228738A1 (en) 2017-07-17 2021-07-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins
CA3076905A1 (en) * 2017-09-27 2019-04-04 The Johns Hopkins University Treatment of ocular diseases with fully-human post-translationally modified anti-vegf fab
KR102205830B1 (ko) * 2017-10-26 2021-01-21 주식회사 큐로진생명과학 솔루블 VEGFR-1 변이체 cDNA를 함유하는 rAAV를 포함하는 황반변성 치료용 조성물
EP3727468A4 (en) * 2017-12-19 2021-09-22 Akouos, Inc. AAV-MEDIATED DELIVERY OF THERAPEUTIC ANTIBODIES TO THE INNER EAR
US10610606B2 (en) * 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
EP3886809A4 (en) 2018-11-28 2022-03-30 Genascence Corporation METHODS AND COMPOSITIONS FOR THE TREATMENT OF OSTEOARTHRITIS
WO2020136384A1 (en) * 2018-12-28 2020-07-02 Constable Ian Jeffery Methods of retinal administration
AU2020231505A1 (en) 2019-03-04 2021-08-19 Adverum Biotechnologies, Inc. Sequential intravitreal administration of AAV gene therapy to contralateral eyes
WO2021055906A1 (en) * 2019-09-20 2021-03-25 Bullseye Therapeutics, Inc. Injection systems and methods of their use
CN112552410A (zh) * 2019-09-26 2021-03-26 三生国健药业（上海）股份有限公司 一种抗体融合蛋白及其制法和在抗肿瘤中的应用
AU2020396699A1 (en) * 2019-12-04 2022-07-14 Cdmogen Co., Ltd Composition for treating diabetic retinopathy, comprising rAAV containing soluble VEGFR-1 variant cDNA
US20220267795A1 (en) * 2021-02-24 2022-08-25 Kinase Pharma Inc. Compositions and methods for regulating production of an angiogensis inhibitor
WO2023206134A1 (en) * 2022-04-27 2023-11-02 Beijing Sightnovo Medical Technology Co., Ltd Compositions and methods for eye diseases

Citations (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5527533A (en) * 1994-10-27 1996-06-18 Board Of Trustees Of The University Of Illinois Method of retarding and ameliorating central nervous system and eye damage
US6054485A (en) * 1996-08-20 2000-04-25 Regents Of The University Of California Eye treatments using synthetic thyroid hormone compositions
US6458157B1 (en) * 1997-08-04 2002-10-01 Suaning Gregg Joergen Retinal stimulator
US20030087889A1 (en) * 2001-02-06 2003-05-08 Strong H. Andrew Photodynamic therapy of occult age-related macular degeneration
US20050260203A1 (en) * 1999-06-08 2005-11-24 Wiegand Stanley J Use of VEGF inhibitors for treatment of eye disorders
US20070190058A1 (en) * 2004-10-21 2007-08-16 Genentech, Inc. Method for treating intraocular neovascular diseases
US20080152654A1 (en) * 2006-06-12 2008-06-26 Exegenics, Inc., D/B/A Opko Health, Inc. COMPOSITIONS AND METHODS FOR siRNA INHIBITION OF ANGIOGENESIS
US20090285826A1 (en) * 2006-05-04 2009-11-19 Fovea Pharmaceuticals Sa Combination drugs comprising vegf inhibitor and serine proteases/thrombolytic compounds for treating neovascular disorders
US7666405B2 (en) * 1998-08-28 2010-02-23 Duke University Deleted adenovirus vectors and methods of making and administering the same
US20100081707A1 (en) * 2008-02-21 2010-04-01 Ali Robin R Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
US20110052678A1 (en) * 2010-11-05 2011-03-03 Shantha Totada R Method for treating age related macular degeneration
US20120141422A1 (en) * 2009-07-31 2012-06-07 Association Institute De Myologie Widespread gene delivery to the retina using systemic administration of aav vectors

Family Cites Families (108)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
US4874237A (en) 1987-05-07 1989-10-17 Lions Eye Inst. Of Western Australia Electroretinogram apparatus
WO1990005538A1 (en) 1988-11-14 1990-05-31 The United States Of America, As Represented By The Secretary, U.S. Department Of Commerce Parvovirus capsids
FR2643252B1 (fr) 1989-02-21 1991-06-07 Technomed Int Sa Appareil de destruction selective de cellules incluant les tissus mous et les os a l'interieur du corps d'un etre vivant par implosion de bulles de gaz
US5112946A (en) 1989-07-06 1992-05-12 Repligen Corporation Modified pf4 compositions and methods of use
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US6379885B1 (en) 1989-09-14 2002-04-30 Rijksuniversiteit Te Leiden Human parvovirus B19 proteins and virus-like particles, their production and their use in diagnostic assays and vaccines
DE69128037T2 (de) 1990-11-13 1998-05-07 Immunex Corp Bifunktionelle wählbare fusionsgene
US5383917A (en) 1991-07-05 1995-01-24 Jawahar M. Desai Device and method for multi-phase radio-frequency ablation
JP3734262B2 (ja) 1993-03-25 2006-01-11 メルク エンド カンパニー インコーポレーテッド 血管内皮細胞増殖因子阻害剤
AU6953394A (en) 1993-05-21 1994-12-20 Targeted Genetics Corporation Bifunctional selectable fusion genes based on the cytosine deaminase (cd) gene
US5814618A (en) 1993-06-14 1998-09-29 Basf Aktiengesellschaft Methods for regulating gene expression
JPH10501681A (ja) 1994-02-22 1998-02-17 ダナ−ファーバー キャンサー インスティチュート 核酸送達システムならびにその合成および使用方法
US6551618B2 (en) 1994-03-15 2003-04-22 University Of Birmingham Compositions and methods for delivery of agents for neuronal regeneration and survival
FR2718150B1 (fr) 1994-03-29 1996-04-26 Rhone Poulenc Rorer Sa Virus recombinants, préparation et utilisation en thérapie génique.
US5639725A (en) 1994-04-26 1997-06-17 Children's Hospital Medical Center Corp. Angiostatin protein
US20020168342A1 (en) 1994-11-03 2002-11-14 Cell Genesys, Inc. Novel adenoviral vectors, packaging cell lines, recombinant adenoviruses and methods
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US5641749A (en) 1995-11-29 1997-06-24 Amgen Inc. Method for treating retinal ganglion cell injury using glial cell line-derived neurothrophic factor (GDNF) protein product
AU738806B2 (en) 1996-09-24 2001-09-27 Merck & Co., Inc. Gene therapy for inhibition of angiogenesis
FR2756297B1 (fr) 1996-11-22 1999-01-08 Centre Nat Rech Scient Procede de production de virus recombinants
US6153436A (en) 1997-01-10 2000-11-28 The Board Of Trustees Of The University Of Arkansas Method of gene delivery using wildtype adeno associated viral (AAV) vectors with insertions
JP4135120B2 (ja) 1997-04-14 2008-08-20 セル ジェネシス，インコーポレーテッド 組換えａａｖ生産の効率を上昇させる方法
AU7383298A (en) 1997-05-13 1998-12-08 Regents Of The University Of California, The Novel antiangiogenic peptide agents and their therapeutic and diagnostic use
WO1999014354A1 (en) * 1997-09-19 1999-03-25 The Trustees Of The University Of The Pennsylvania Methods and vector constructs useful for production of recombinant aav
JPH11100327A (ja) 1997-09-26 1999-04-13 Toray Ind Inc 網膜変性症治療剤
AU9673998A (en) 1997-10-01 1999-04-23 G.D. Searle & Co. Fusion proteins comprising an angiostatin moiety and their use in anti-tumor treatment
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
EP1045921A2 (en) 1998-01-16 2000-10-25 Chiron Corporation Feline immunodeficiency virus gene therapy vectors
ATE226444T1 (de) 1998-03-12 2002-11-15 Genentech Inc Verwendung von fgf-5 polypetiden zur vorbeugung des absterbens retinaler neuronen und behandlung okularer krankheiten
US6593133B1 (en) 1998-07-06 2003-07-15 Nsgene A/S Neurotrophic factors
EP1114171A1 (en) 1998-09-17 2001-07-11 University of Florida Methods for treatment of degenerative retinal diseases
US20040234505A1 (en) 1998-09-23 2004-11-25 Stuart Naylor Polynucleotide constructs and uses thereof
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
EP1183051A2 (en) * 1999-03-15 2002-03-06 Chiron Corporation Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
US7070959B1 (en) 1999-06-08 2006-07-04 Regeneron Pharmaceuticals, Inc. Modified chimeric polypeptides with improved pharmacokinetic properties
US7087411B2 (en) 1999-06-08 2006-08-08 Regeneron Pharmaceuticals, Inc. Fusion protein capable of binding VEGF
EP1229839A4 (en) 1999-10-25 2005-12-07 Therus Corp USING FOCUSED ULTRASOUND FOR VASCULAR SEALING
ATE511784T1 (de) 2000-04-17 2011-06-15 Univ Sydney Verfahren zur objektiven elektrophysiologischen bewertung der visuellen funktion
US6329181B1 (en) 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
EP1372552B1 (de) 2001-03-27 2017-03-01 WaveLight GmbH Vorrichtung zur bearbeitung und diagnose von augengewebe
WO2002082904A2 (en) 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness
JP2002363107A (ja) 2001-06-04 2002-12-18 Noriyuki Azuma 色覚不全動物の色覚復元方法
WO2003012054A2 (en) 2001-08-02 2003-02-13 Institut Clayton De La Recherche Methods and compositions relating to improved lentiviral vector production systems
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
EP1490113A4 (en) 2002-03-20 2007-05-02 Univ Florida ADENO-ASSOCIATED RECOMBINANT VIRAL VECTOR (RAAV) COMPOSITIONS AND CORRESPONDING METHODS FOR THE TREATMENT OF CHOROIDAL NEOVASCULARIZATION
AU2003223775A1 (en) 2002-04-30 2003-11-17 Duke University Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use
US8304233B2 (en) 2002-06-04 2012-11-06 Poetic Genetics, Llc Methods of unidirectional, site-specific integration into a genome, compositions and kits for practicing the same
AU2003265855A1 (en) 2002-08-28 2004-03-19 University Of Florida Modified aav
GB0220467D0 (en) 2002-09-03 2002-10-09 Oxford Biomedica Ltd Composition
WO2004079332A2 (en) * 2003-03-04 2004-09-16 National Institute Of Advanced Industrial Science And Technology Composition and method for increasing efficiency of introduction of target substance into cell
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
WO2005072364A2 (en) 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector
SI1809750T1 (sl) 2004-11-08 2012-08-31 Chromagenics Bv Izbira gostiteljskih celic, ki imajo visok nivo izraĹľanja proteina
US7922670B2 (en) 2005-02-24 2011-04-12 Warren Jones System and method for quantifying and mapping visual salience
JP5702519B2 (ja) 2005-04-07 2015-04-15 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Ａａｖベクターの機能を上げる方法
US20060234347A1 (en) * 2005-04-13 2006-10-19 Harding Thomas C Targeting multiple angiogenic pathways for cancer therapy using soluble tyrosine kinase receptors
EP2311967B1 (en) 2005-10-20 2017-09-20 UniQure IP B.V. Improved AAV vectors produced in insect cells
US20090191597A1 (en) 2006-01-20 2009-07-30 Asklepios Biopharmaceutical, Inc. Enhanced production of infectious parvovirus vectors in insect cells
US20070190028A1 (en) 2006-02-13 2007-08-16 Jihong Qu Method and apparatus for heat or electromagnetic control of gene expression
US7384145B2 (en) 2006-02-16 2008-06-10 The Board Of Trustees Of The University Of Illinois Mapping retinal function using corneal electrode array
US8118752B2 (en) 2006-02-16 2012-02-21 The Board Of Trustees Of The University Of Illinois Apparatus and methods for mapping retinal function
US8216575B2 (en) * 2006-03-31 2012-07-10 Chengdu Kanghong Biotechnologies Co., Ltd. Inhibition of neovascularization with a soluble chimeric protein comprising VEGF FLT-1 and KDR domains
GB0612096D0 (en) 2006-06-19 2006-07-26 Greater Glasgow Nhs Board Functional imaging of the retina
RU2457252C2 (ru) 2006-06-21 2012-07-27 Амстердам Молекьюла Терапьютикс (Амт) Ип Б.В. AAV ВЕКТОРЫ С УСОВЕРШЕНСТВОВАННЫМИ Rep-КОДИРУЮЩИМИ ПОСЛЕДОВАТЕЛЬНОСТЯМИ, ИСПОЛЬЗУЕМЫМИ В СИСТЕМАХ ПРОДУКЦИИ НА ОСНОВЕ КЛЕТОК НАСЕКОМЫХ
US20100008170A1 (en) 2006-06-27 2010-01-14 Shinya Sato Semiconductor tester and testing method of semiconductor memory
US8945918B2 (en) 2006-08-24 2015-02-03 Virovek, Inc. Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor
WO2008098310A1 (en) 2007-02-16 2008-08-21 Objectivision Limited Stimulus method for multifocal visual evoked potential
EP1995309A1 (en) * 2007-05-21 2008-11-26 Vivalis Recombinant protein production in avian EBx® cells
US10696983B2 (en) 2007-05-30 2020-06-30 The Trustees Of The University Of Pennsylvania Method for transducing cells with primary cilia
US8540369B2 (en) 2007-08-16 2013-09-24 The Research Foundation Of State University Of New York Led variable light source
US8518037B2 (en) 2007-10-30 2013-08-27 Boston Scientific Scimed, Inc. Radiofrequency ablation device
US20110053274A1 (en) * 2007-11-30 2011-03-03 Scarab Genomics Llc Lac expression system
WO2009104964A1 (en) 2008-02-19 2009-08-27 Amsterdam Molecular Therapeutics B.V. Optimisation of expression of parvoviral rep and cap proteins in insect cells
CN101951925A (zh) 2008-02-20 2011-01-19 建新公司 血管发生抑制
ITFI20080081A1 (it) 2008-04-18 2009-10-19 Strumenti Oftalmici C S O S R Procedimento e sistema per la registrazione di risposte elettrofunzionali erg, perg e vep multifocali in tempo reale
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
ES2330826B1 (es) 2008-06-04 2010-07-26 Proyecto De Biomedicina Cima, S.L. Sistema para empaquetamiento de adenovirus de alta capacidad.
PT2403867T (pt) 2009-03-04 2019-08-29 Deutsches Krebsforsch Proteína de ativação de montagem (aap) e a sua utilização para o fabrico de partículas de parvovírus essenciais consistindo de vp3
WO2010114948A2 (en) 2009-04-02 2010-10-07 University Of Florida Research Foundation, Inc. An inducible system for highly efficient production of recombinant adeno-associated virus (raav) vectors
TWI466158B (zh) 2009-07-03 2014-12-21 Univ Lunghwa Sci & Technology 電漿測量裝置、電漿系統及測量電漿特性之方法
EP2292781A1 (en) 2009-08-17 2011-03-09 Genethon Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions
WO2011034947A2 (en) 2009-09-15 2011-03-24 University Of Washington Reagents and methods for modulating cone photoreceptor activity
US20110270256A1 (en) 2009-09-24 2011-11-03 Medicinelodge, Inc. Dba Imds Co-Innovation Surgical rasp with radiofrequency ablation
WO2011088081A1 (en) 2010-01-12 2011-07-21 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
US9228174B2 (en) 2010-03-11 2016-01-05 Uniqure Ip B.V. Mutated rep encoding sequences for use in AAV production
US20130023033A1 (en) * 2010-03-29 2013-01-24 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
CN201704771U (zh) 2010-04-30 2011-01-12 陈福环 无水箱节水型抽水马桶
US9102718B2 (en) * 2010-04-30 2015-08-11 Lpath, Inc. Anti-S1P antibody treatment of patients with ocular disease
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
JP6092782B2 (ja) 2010-11-16 2017-03-08 エクセリミューン， インコーポレイテッド 組換えタンパク質の製造方法
WO2012094560A2 (en) 2011-01-07 2012-07-12 Applied Genetic Technologies Corporation Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
RS60207B1 (sr) 2011-04-22 2020-06-30 Univ California Adeno-povezani virioni virusa sa varijantama kapsida i postupci za njihovu primenu
WO2013063601A1 (en) 2011-10-28 2013-05-02 University Of Florida Research Foundation, Inc. Chimeric promoter for cone photoreceptor targeted gene therapy
TWI775096B (zh) 2012-05-15 2022-08-21 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
WO2013188316A1 (en) 2012-06-11 2013-12-19 Avalanche Biotechnologies, Inc. Optical regulation of gene expression in the retina
EP3013946B8 (en) 2013-06-28 2019-04-10 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for expressing a polynucleotide of interest in the retina of a subject
US20150025939A1 (en) 2013-07-18 2015-01-22 Somnath Chatterjee Company Centric Social Media Platfonn for Content Sharing aud Tracking
EP3561062A1 (en) 2013-09-13 2019-10-30 California Institute of Technology Selective recovery
EP3633041A3 (en) 2013-09-26 2020-07-29 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
CA2941640A1 (en) 2014-03-04 2015-09-11 University Of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
SG10201810150UA (en) 2014-03-17 2018-12-28 Adverum Biotechnologies Inc Compositions and methods for enhanced gene expression in cone cells
AU2016226289B2 (en) 2015-03-02 2021-04-29 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods

Patent Citations (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5527533A (en) * 1994-10-27 1996-06-18 Board Of Trustees Of The University Of Illinois Method of retarding and ameliorating central nervous system and eye damage
US6054485A (en) * 1996-08-20 2000-04-25 Regents Of The University Of California Eye treatments using synthetic thyroid hormone compositions
US6458157B1 (en) * 1997-08-04 2002-10-01 Suaning Gregg Joergen Retinal stimulator
US7666405B2 (en) * 1998-08-28 2010-02-23 Duke University Deleted adenovirus vectors and methods of making and administering the same
US20050260203A1 (en) * 1999-06-08 2005-11-24 Wiegand Stanley J Use of VEGF inhibitors for treatment of eye disorders
US20030087889A1 (en) * 2001-02-06 2003-05-08 Strong H. Andrew Photodynamic therapy of occult age-related macular degeneration
US20070190058A1 (en) * 2004-10-21 2007-08-16 Genentech, Inc. Method for treating intraocular neovascular diseases
US20090285826A1 (en) * 2006-05-04 2009-11-19 Fovea Pharmaceuticals Sa Combination drugs comprising vegf inhibitor and serine proteases/thrombolytic compounds for treating neovascular disorders
US20080152654A1 (en) * 2006-06-12 2008-06-26 Exegenics, Inc., D/B/A Opko Health, Inc. COMPOSITIONS AND METHODS FOR siRNA INHIBITION OF ANGIOGENESIS
US20100081707A1 (en) * 2008-02-21 2010-04-01 Ali Robin R Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
US20120141422A1 (en) * 2009-07-31 2012-06-07 Association Institute De Myologie Widespread gene delivery to the retina using systemic administration of aav vectors
US20110052678A1 (en) * 2010-11-05 2011-03-03 Shantha Totada R Method for treating age related macular degeneration

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Shah et al, Outcomes and risk Factors Associated with Endophthalmitis After Intravitreal Injection of Anti-Vascular Endothelial Growth Factor Agents. Jefferson Digital Commons, 2011. 1-14 *

Cited By (87)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10214566B2 (en) 2003-06-30 2019-02-26 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US10046016B2 (en) 2003-06-30 2018-08-14 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US10166097B2 (en) 2009-09-29 2019-01-01 Edwards Lifesciences Cardiaq Llc Replacement heart valve and method
US9457103B2 (en) 2010-10-06 2016-10-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
US10494612B2 (en) 2010-10-06 2019-12-03 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
US9458517B2 (en) 2011-04-22 2016-10-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
US11236402B2 (en) 2011-04-22 2022-02-01 The Regents Of The University Of California Adeno-associated virus virions with variant capsid
US9856539B2 (en) 2011-04-22 2018-01-02 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
US9193956B2 (en) 2011-04-22 2015-11-24 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
US9587282B2 (en) 2011-04-22 2017-03-07 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
US10214785B2 (en) 2011-04-22 2019-02-26 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
US10202657B2 (en) 2011-04-22 2019-02-12 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
US9943573B2 (en) 2012-05-15 2018-04-17 Avalanche Australia Pty Ltd. Treatment of ocular neovascularization using anti-VEGF proteins
US10004788B2 (en) 2012-05-15 2018-06-26 Avalanche Australia Pty Ltd. Treatment of ocular neovascularization using anti-VEGF proteins
US9681951B2 (en) 2013-03-14 2017-06-20 Edwards Lifesciences Cardiaq Llc Prosthesis with outer skirt and anchors
US11634691B2 (en) 2013-05-31 2023-04-25 The Regents Of The University Of California Compositions and methods of treatment
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
US10301645B2 (en) 2013-12-27 2019-05-28 Mogam Biotechnology Institute Expression vector for expressing heterogeneous gene
EP3088527A4 (en) * 2013-12-27 2017-09-06 Mogam Biotechnology Institute Expression vector having improved ability to express gene
US10000741B2 (en) 2014-03-17 2018-06-19 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
US11248214B2 (en) 2014-03-17 2022-02-15 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
US11155610B2 (en) 2014-06-28 2021-10-26 Kodiak Sciences Inc. Dual PDGF/VEGF antagonists
US11027000B2 (en) 2014-11-05 2021-06-08 Voyager Therapeutics, Inc. AADC polynucleotides for the treatment of Parkinson's disease
US11975056B2 (en) 2014-11-05 2024-05-07 Voyager Therapeutics, Inc. AADC polynucleotides for the treatment of Parkinson's disease
US10335466B2 (en) 2014-11-05 2019-07-02 Voyager Therapeutics, Inc. AADC polynucleotides for the treatment of parkinson's disease
US10920227B2 (en) 2014-11-14 2021-02-16 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
US11198873B2 (en) 2014-11-14 2021-12-14 Voyager Therapeutics, Inc. Modulatory polynucleotides
US10570395B2 (en) 2014-11-14 2020-02-25 Voyager Therapeutics, Inc. Modulatory polynucleotides
US10597660B2 (en) 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
US11542506B2 (en) 2014-11-14 2023-01-03 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
US11021519B2 (en) 2015-03-02 2021-06-01 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
US10883117B2 (en) 2015-03-24 2021-01-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
US11104917B2 (en) * 2015-05-08 2021-08-31 Children's Medical Research Institute Promoters for expression of heterologous genes
US20180142260A1 (en) * 2015-05-08 2018-05-24 Children's Medical Research Institute Promoters for expression of heterologous genes
WO2017075335A1 (en) 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
US11427931B2 (en) 2015-12-23 2022-08-30 Adverum Biotechnologies, Inc. Mutant viral capsid libraries and related systems and methods
US10584328B2 (en) 2015-12-23 2020-03-10 Adverum Biotechnologies, Inc. Mutant viral capsid libraries and related systems and methods
US11066465B2 (en) 2015-12-30 2021-07-20 Kodiak Sciences Inc. Antibodies and conjugates thereof
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
US11951121B2 (en) 2016-05-18 2024-04-09 Voyager Therapeutics, Inc. Compositions and methods for treating Huntington's disease
US11193129B2 (en) 2016-05-18 2021-12-07 Voyager Therapeutics, Inc. Modulatory polynucleotides
US11565000B2 (en) 2016-07-29 2023-01-31 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
US11554180B2 (en) 2016-07-29 2023-01-17 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
US11565001B2 (en) 2016-07-29 2023-01-31 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
US11298041B2 (en) 2016-08-30 2022-04-12 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
US11192925B2 (en) 2016-10-19 2021-12-07 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
US11242530B2 (en) * 2016-12-08 2022-02-08 Shanghai Unicar-Therapy Bio-Medicine Technology Co., Ltd siRNA knocking down human PD-1 and recombinant expression CAR-T vector and their construction methods and applications
US11352644B2 (en) 2017-03-17 2022-06-07 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression
AU2018234918B2 (en) * 2017-03-17 2023-11-02 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression
US11773406B2 (en) 2017-03-17 2023-10-03 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression
WO2018170473A1 (en) * 2017-03-17 2018-09-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression
US11603542B2 (en) 2017-05-05 2023-03-14 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
US11752181B2 (en) 2017-05-05 2023-09-12 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
US11759506B2 (en) 2017-06-15 2023-09-19 Voyager Therapeutics, Inc. AADC polynucleotides for the treatment of Parkinson's disease
US11497576B2 (en) 2017-07-17 2022-11-15 Voyager Therapeutics, Inc. Trajectory array guide system
US11680249B2 (en) 2017-08-28 2023-06-20 The Regents Of The University Of California Adeno-associated virus capsid variants and methods of use thereof
US11434502B2 (en) 2017-10-16 2022-09-06 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
US11931375B2 (en) 2017-10-16 2024-03-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
EP4124658A2 (en) 2017-10-16 2023-02-01 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
WO2019173434A1 (en) 2018-03-06 2019-09-12 Voyager Therapeutics, Inc. Insect cell manufactured partial self-complementary aav genomes
WO2019217582A1 (en) * 2018-05-08 2019-11-14 Rutgers, The State University Of New Jersey Aav-compatible laminin-linker polymerization proteins
IL278393B1 (en) * 2018-05-08 2023-07-01 Univ Rutgers aav-compatible proteins for polymerization bind to laminin
WO2019222444A2 (en) 2018-05-16 2019-11-21 Voyager Therapeutics, Inc. Directed evolution
WO2019222441A1 (en) 2018-05-16 2019-11-21 Voyager Therapeutics, Inc. Aav serotypes for brain specific payload delivery
WO2020010042A1 (en) 2018-07-02 2020-01-09 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
WO2020023612A1 (en) 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
WO2020072849A1 (en) 2018-10-04 2020-04-09 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
WO2020150556A1 (en) 2019-01-18 2020-07-23 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
WO2020219933A1 (en) 2019-04-26 2020-10-29 Adverum Biotechnologies, Inc. Variant aav capsids for intravitreal delivery
US11510950B2 (en) 2019-04-26 2022-11-29 Adverum Biotechnologies, Inc. Variant AAV capsids for intravitreal delivery
WO2020223274A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
WO2021030125A1 (en) 2019-08-09 2021-02-18 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
WO2021041485A1 (en) 2019-08-26 2021-03-04 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US11912784B2 (en) 2019-10-10 2024-02-27 Kodiak Sciences Inc. Methods of treating an eye disorder
WO2022032153A1 (en) 2020-08-06 2022-02-10 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
WO2022187473A2 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022247917A1 (zh) 2021-05-28 2022-12-01 上海瑞宏迪医药有限公司 衣壳变异的重组腺相关病毒及其应用
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins

Also Published As

Publication number Publication date
RU2014150340A (ru) 2016-07-10
US20220111015A1 (en) 2022-04-14
BR112014028633A2 (pt) 2017-08-01
JP2019071908A (ja) 2019-05-16
KR20200119276A (ko) 2020-10-19
JP2018015008A (ja) 2018-02-01
US20180125948A1 (en) 2018-05-10
CY1124495T1 (el) 2022-07-22
EP4371615A2 (en) 2024-05-22
EP2849802A4 (en) 2016-01-20
US20150004101A1 (en) 2015-01-01
PT3501549T (pt) 2021-09-30
AU2021200253A1 (en) 2021-03-18
TWI702955B (zh) 2020-09-01
US20180311319A1 (en) 2018-11-01
KR102154225B1 (ko) 2020-09-11
MX2019000586A (es) 2021-06-15
HK1207568A1 (en) 2016-02-05
SG11201407548UA (en) 2014-12-30
JP6466322B2 (ja) 2019-02-06
BR112014028633A8 (pt) 2020-01-21
NZ702637A (en) 2017-05-26
PL3501549T3 (pl) 2022-01-17
LT3501549T (lt) 2021-10-11
TWI698240B (zh) 2020-07-11
TW201828951A (zh) 2018-08-16
JP2021045170A (ja) 2021-03-25
SG10201609412QA (en) 2017-01-27
KR20150014965A (ko) 2015-02-09
EP2849802A2 (en) 2015-03-25
US20140341977A1 (en) 2014-11-20
AU2018211212A1 (en) 2018-08-16
EP2849802B1 (en) 2023-12-20
JP6667486B2 (ja) 2020-03-18
JP2022106929A (ja) 2022-07-20
CA2873628A1 (en) 2013-11-21
AU2013263159B2 (en) 2018-05-31
EP3501549B1 (en) 2021-06-23
KR102218067B1 (ko) 2021-02-22
WO2013173129A3 (en) 2014-01-30
CA2873628C (en) 2020-11-24
AU2013263159A1 (en) 2015-01-15
SI3501549T1 (sl) 2021-11-30
MX2014013933A (es) 2015-06-03
MX362452B (es) 2019-01-18
US9943573B2 (en) 2018-04-17
RS62359B1 (sr) 2021-10-29
WO2013173129A2 (en) 2013-11-21
NZ727516A (en) 2018-05-25
HRP20211453T1 (hr) 2021-12-24
TWI775096B (zh) 2022-08-21
TW202103711A (zh) 2021-02-01
JP2015523060A (ja) 2015-08-13
US20140371438A1 (en) 2014-12-18
EP4371615A3 (en) 2024-06-26
EP2849802C0 (en) 2023-12-20
AU2018211212B2 (en) 2020-10-15
DK3501549T3 (da) 2021-09-13
TW201408307A (zh) 2014-03-01
IL281833A (en) 2021-05-31
EP3501549A1 (en) 2019-06-26
CN115337407A (zh) 2022-11-15
ES2967616T3 (es) 2024-05-03
KR20210021111A (ko) 2021-02-24
IL235679A0 (en) 2015-02-26
CN104994882A (zh) 2015-10-21
IL235679B (en) 2021-04-29
US10004788B2 (en) 2018-06-26
HUE056327T2 (hu) 2022-02-28
ZA201409025B (en) 2019-04-24
ES2890813T3 (es) 2022-01-24

Similar Documents

Publication Publication Date Title
US20220111015A1 (en) Treatment of ocular neovascularization using anti-vegf proteins
US20230322911A1 (en) Compositions and methods for reducing ocular neovascularization
US20210100856A1 (en) Methods of treating ocular neovascular diseases using aav2 variants encoding aflibercept

Legal Events

Date Code Title Description
AS Assignment

Owner name: AVALANCHE AUSTRALIA PTY LTD., AUSTRALIA

Free format text: ASSIGNMENT OF ASSIGNORS INTEREST;ASSIGNOR:CHALBERG, THOMAS W., JR.;REEL/FRAME:030462/0370

Effective date: 20130429

Owner name: LIONS EYE INSTITUTE LIMITED, AUSTRALIA

Free format text: ASSIGNMENT OF ASSIGNORS INTEREST;ASSIGNORS:CONSTABLE, IAN J.;RAKOCZY, ELIZABETH P.;LAI, CHOOI-MAY;SIGNING DATES FROM 20130425 TO 20130429;REEL/FRAME:030457/0046

AS Assignment

Owner name: AVALANCHE AUSTRALIA PTY LTD., AUSTRALIA

Free format text: ASSIGNMENT OF ASSIGNORS INTEREST;ASSIGNOR:LIONS EYE INSTITUTE LIMITED;REEL/FRAME:034138/0491

Effective date: 20141103

AS Assignment

Owner name: LIONS EYE INSTITUTE LIMITED, AUSTRALIA

Free format text: CORRECTIVE ASSIGNMENT TO CORRECT THE THE LISTING OF ASSIGNOR NAME. PREVIOUSLY RECORDED ON REEL 030457 FRAME 0046. ASSIGNOR(S) HEREBY CONFIRMS THE ASSIGNOR'S NAME;ASSIGNORS:CONSTABLE, IAN J.;RAKOCZY, P. ELIZABETH;LAI, CHOOI-MAY;SIGNING DATES FROM 20130426 TO 20140828;REEL/FRAME:034944/0001

STCB Information on status: application discontinuation

Free format text: ABANDONED -- FAILURE TO RESPOND TO AN OFFICE ACTION