WO2014160196A9 - Sonochemical induction of abca1 expression and compositions therefor - Google Patents

Sonochemical induction of abca1 expression and compositions therefor Download PDF

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Publication number
WO2014160196A9
WO2014160196A9 PCT/US2014/026018 US2014026018W WO2014160196A9 WO 2014160196 A9 WO2014160196 A9 WO 2014160196A9 US 2014026018 W US2014026018 W US 2014026018W WO 2014160196 A9 WO2014160196 A9 WO 2014160196A9
Authority
WO
WIPO (PCT)
Prior art keywords
abca1
microspheres
sonochemical
induction
sonochemically
Prior art date
Application number
PCT/US2014/026018
Other languages
French (fr)
Other versions
WO2014160196A3 (en
WO2014160196A2 (en
Inventor
Eric COLES
Michael Davidson
Original Assignee
Sonogene, Llc
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 Sonogene, Llc filed Critical Sonogene, Llc
Priority to EP14772576.6A priority Critical patent/EP2970991B1/en
Priority to JP2016502027A priority patent/JP6550039B2/en
Priority to AU2014244014A priority patent/AU2014244014B2/en
Priority to CA2902047A priority patent/CA2902047A1/en
Publication of WO2014160196A2 publication Critical patent/WO2014160196A2/en
Publication of WO2014160196A3 publication Critical patent/WO2014160196A3/en
Publication of WO2014160196A9 publication Critical patent/WO2014160196A9/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/22—Echographic preparations; Ultrasound imaging preparations ; Optoacoustic imaging preparations
    • A61K49/222—Echographic preparations; Ultrasound imaging preparations ; Optoacoustic imaging preparations characterised by a special physical form, e.g. emulsions, liposomes
    • A61K49/223—Microbubbles, hollow microspheres, free gas bubbles, gas microspheres
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K41/00—Medicinal preparations obtained by treating materials with wave energy or particle radiation ; Therapies using these preparations
    • A61K41/0028—Disruption, e.g. by heat or ultrasounds, sonophysical or sonochemical activation, e.g. thermosensitive or heat-sensitive liposomes, disruption of calculi with a medicinal preparation and ultrasounds
    • 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
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61M—DEVICES FOR INTRODUCING MEDIA INTO, OR ONTO, THE BODY; DEVICES FOR TRANSDUCING BODY MEDIA OR FOR TAKING MEDIA FROM THE BODY; DEVICES FOR PRODUCING OR ENDING SLEEP OR STUPOR
    • A61M37/00—Other apparatus for introducing media into the body; Percutany, i.e. introducing medicines into the body by diffusion through the skin
    • A61M37/0092—Other apparatus for introducing media into the body; Percutany, i.e. introducing medicines into the body by diffusion through the skin using ultrasonic, sonic or infrasonic vibrations, e.g. phonophoresis
    • 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
    • A61P3/06—Antihyperlipidemics
    • 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
    • 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
    • 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

Abstract

The present invention provides compositions useful for transfecting cells (e.g., liver cells) to express ABCA1. The compositions described herein comprise a pharmaceutically acceptable aqueous carrier containing sonochemically-active microspheres together with a plasmid DNA construct encoding an active form of ABCA1 and at least one promoter for the expression thereof. Preferably, the sonochemically-active microspheres comprise, consist essentially of, or consist of gas bubbles (e.g., a fluorocarbon gas, such as octafluoropropane) encapsulated within protein-containing or lipid-containing shells (e.g., human serum albumin shells). The microspheres are disruptable by exposure to ultrasonic acoustic energy to release the encapsulated gas.
PCT/US2014/026018 2013-03-14 2014-03-13 Sonochemical induction of abca1 expression and compositions therefor WO2014160196A2 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
EP14772576.6A EP2970991B1 (en) 2013-03-14 2014-03-13 Sonochemical induction of abca1 expression and compositions therefor
JP2016502027A JP6550039B2 (en) 2013-03-14 2014-03-13 Sonochemical induction of ABCA1 expression and composition therefor
AU2014244014A AU2014244014B2 (en) 2013-03-14 2014-03-13 Sonochemical induction of ABCA1 expression and compositions therefor
CA2902047A CA2902047A1 (en) 2013-03-14 2014-03-13 Sonochemical induction of abca1 expression and compositions therefor

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US13/826,066 2013-03-14
US13/826,066 US9101745B2 (en) 2013-03-14 2013-03-14 Sonochemical induction of ABCA1 expression and compositions therefor

Publications (3)

Publication Number Publication Date
WO2014160196A2 WO2014160196A2 (en) 2014-10-02
WO2014160196A3 WO2014160196A3 (en) 2014-11-27
WO2014160196A9 true WO2014160196A9 (en) 2015-01-29

Family

ID=51530678

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2014/026018 WO2014160196A2 (en) 2013-03-14 2014-03-13 Sonochemical induction of abca1 expression and compositions therefor

Country Status (6)

Country Link
US (2) US9101745B2 (en)
EP (1) EP2970991B1 (en)
JP (1) JP6550039B2 (en)
AU (1) AU2014244014B2 (en)
CA (1) CA2902047A1 (en)
WO (1) WO2014160196A2 (en)

Family Cites Families (27)

* Cited by examiner, † Cited by third party
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US5585112A (en) 1989-12-22 1996-12-17 Imarx Pharmaceutical Corp. Method of preparing gas and gaseous precursor-filled microspheres
US5542935A (en) 1989-12-22 1996-08-06 Imarx Pharmaceutical Corp. Therapeutic delivery systems related applications
US5580575A (en) 1989-12-22 1996-12-03 Imarx Pharmaceutical Corp. Therapeutic drug delivery systems
JPH03297475A (en) 1990-04-16 1991-12-27 Ken Ishihara Controlling method for emission of medicine by means of resonance sound wave
DE69215722T3 (en) 1991-03-22 2001-03-08 Katsuro Tachibana Amplifiers for ultrasound therapy of diseases and liquid pharmaceutical compositions containing them
CN1056124A (en) 1991-06-05 1991-11-13 清华大学 High efficient ultrosonic wave genetic conduction method
BR9405798A (en) 1993-02-22 1995-12-12 Vivorx Pharmaceuticals Inc Methods for in vivo release of biological material and useful compositions thereof
DE59409568D1 (en) 1993-09-09 2000-11-30 Schering Ag ACTIVE SUBSTANCES AND GAS CONTAINING MICROPARTICLES
US6048903A (en) 1994-05-03 2000-04-11 Robert Toppo Treatment for blood cholesterol with trans-resveratrol
US5558092A (en) 1995-06-06 1996-09-24 Imarx Pharmaceutical Corp. Methods and apparatus for performing diagnostic and therapeutic ultrasound simultaneously
US6139819A (en) 1995-06-07 2000-10-31 Imarx Pharmaceutical Corp. Targeted contrast agents for diagnostic and therapeutic use
US6265387B1 (en) 1995-10-11 2001-07-24 Mirus, Inc. Process of delivering naked DNA into a hepatocyte via bile duct
US20010009904A1 (en) 1997-12-30 2001-07-26 Jon A. Wolff Process of delivering a polynucleotide to a cell via the vascular system
US5849727A (en) 1996-06-28 1998-12-15 Board Of Regents Of The University Of Nebraska Compositions and methods for altering the biodistribution of biological agents
JP2001501458A (en) 1996-08-15 2001-02-06 アメリカ合衆国 Spatial and temporal control of gene expression using a combination of heat shock protein promoter and local heating
JP2002515887A (en) 1996-10-19 2002-05-28 クウォドラント、ヘルスケアー、（ユーケー）、リミテッド Use of hollow microcapsules in diagnosis and therapy
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WO2000042988A1 (en) 1999-01-21 2000-07-27 University Of Virginia Method of identifying and treating inflamed tissue
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KR20020012612A (en) * 1999-06-18 2002-02-16 씨브이 쎄러퓨틱스, 인코포레이티드 Compositions and methods for increasing cholesterol efflux and raising hdl using atp binding cassette transporter protein abc1
DE60026313D1 (en) 1999-07-23 2006-04-27 Uutech Ltd SENSITIZATION OF RED BLOOD BODIES AGAINST ULTRASOUND BY IMPACT OF AN ELECTRIC FIELD
JP2002145784A (en) * 2000-11-10 2002-05-22 Ryuichi Morishita Biologically active medicament-introducing composition and method for using the same
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Also Published As

Publication number Publication date
EP2970991B1 (en) 2022-04-27
EP2970991A4 (en) 2016-11-30
JP6550039B2 (en) 2019-07-24
WO2014160196A3 (en) 2014-11-27
CA2902047A1 (en) 2014-10-02
US20140276363A1 (en) 2014-09-18
US9101745B2 (en) 2015-08-11
WO2014160196A2 (en) 2014-10-02
AU2014244014A1 (en) 2015-09-17
JP2016514155A (en) 2016-05-19
AU2014244014B2 (en) 2019-08-01
US20150343098A1 (en) 2015-12-03
EP2970991A2 (en) 2016-01-20
US9393328B2 (en) 2016-07-19

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