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WO1997004748A3 - Enhanced artificial viral envelopes for cellular delivery of therapeutic substances - Google Patents

Enhanced artificial viral envelopes for cellular delivery of therapeutic substances Download PDF

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Publication number
WO1997004748A3
WO1997004748A3 PCT/US1996/012750 US9612750W WO9704748A3 WO 1997004748 A3 WO1997004748 A3 WO 1997004748A3 US 9612750 W US9612750 W US 9612750W WO 9704748 A3 WO9704748 A3 WO 9704748A3
Authority
WO
WIPO (PCT)
Prior art keywords
vesicles
therapeutic substances
artificial viral
envelopes
nucleic acid
Prior art date
Application number
PCT/US1996/012750
Other languages
French (fr)
Other versions
WO1997004748A9 (en
WO1997004748A2 (en
WO1997004748B1 (en
Inventor
Jon T Conary
Hans Schreier
Original Assignee
Advanced Therapies Inc
Jon T Conary
Hans Schreier
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 Advanced Therapies Inc, Jon T Conary, Hans Schreier filed Critical Advanced Therapies Inc
Priority to AU66914/96A priority Critical patent/AU6691496A/en
Publication of WO1997004748A2 publication Critical patent/WO1997004748A2/en
Publication of WO1997004748A3 publication Critical patent/WO1997004748A3/en
Publication of WO1997004748B1 publication Critical patent/WO1997004748B1/en
Publication of WO1997004748A9 publication Critical patent/WO1997004748A9/en

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81Protease inhibitors
    • C07K14/8107Endopeptidase (E.C. 3.4.21-99) inhibitors
    • C07K14/811Serine protease (E.C. 3.4.21) inhibitors
    • C07K14/8121Serpins
    • C07K14/8125Alpha-1-antitrypsin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Dispersion Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)

Abstract

This invention provides artificial viral envelopes and other lipid vesicles that encapsulate therapeutic substances, such as expression vectors, targeted to mammalian cells. Polynucleotides may be packed into the envelopes by compressing them beforehand with a short peptide with a predominant positive charge. The compression step not only facilitates encapsulation, it also increases the number of vesicles containing nucleic acid, minimizes the amount of free nucleic acid, and may also increase the size and complexity of plasmids that can be encapsulated. The vesicles may be provided with a tissue targeting component that helps direct it towards certain tissue sites in an animal. The vesicles may also be provided with a fusogenic component that facilitates delivery of the therapeutic substance into the cell. The materials and reagents of this invention are effective, for example, in increasing expression of model proteins in both isolated cells and intact animals, and are expected to be useful for gene therapy.
PCT/US1996/012750 1995-08-01 1996-08-01 Enhanced artificial viral envelopes for cellular delivery of therapeutic substances WO1997004748A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU66914/96A AU6691496A (en) 1995-08-01 1996-08-01 Enhanced artificial viral envelopes for cellular delivery of therapeutic substances

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US173895P 1995-08-01 1995-08-01
US60/001,738 1995-08-01
US258095P 1995-08-21 1995-08-21
US60/002,580 1995-08-21
US69061396A 1996-07-31 1996-07-31
US60/690,613 1996-07-31

Publications (4)

Publication Number Publication Date
WO1997004748A2 WO1997004748A2 (en) 1997-02-13
WO1997004748A3 true WO1997004748A3 (en) 1997-05-29
WO1997004748B1 WO1997004748B1 (en) 1997-07-17
WO1997004748A9 WO1997004748A9 (en) 1997-10-09

Family

ID=27356985

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1996/012750 WO1997004748A2 (en) 1995-08-01 1996-08-01 Enhanced artificial viral envelopes for cellular delivery of therapeutic substances

Country Status (2)

Country Link
AU (1) AU6691496A (en)
WO (1) WO1997004748A2 (en)

Cited By (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7335509B2 (en) 1995-01-23 2008-02-26 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US8841071B2 (en) 2011-06-02 2014-09-23 Raindance Technologies, Inc. Sample multiplexing
US8871444B2 (en) 2004-10-08 2014-10-28 Medical Research Council In vitro evolution in microfluidic systems
US9012390B2 (en) 2006-08-07 2015-04-21 Raindance Technologies, Inc. Fluorocarbon emulsion stabilizing surfactants
US9228229B2 (en) 2010-02-12 2016-01-05 Raindance Technologies, Inc. Digital analyte analysis
US9273308B2 (en) 2006-05-11 2016-03-01 Raindance Technologies, Inc. Selection of compartmentalized screening method
US9328344B2 (en) 2006-01-11 2016-05-03 Raindance Technologies, Inc. Microfluidic devices and methods of use in the formation and control of nanoreactors
US9366632B2 (en) 2010-02-12 2016-06-14 Raindance Technologies, Inc. Digital analyte analysis
US9399797B2 (en) 2010-02-12 2016-07-26 Raindance Technologies, Inc. Digital analyte analysis
US9448172B2 (en) 2003-03-31 2016-09-20 Medical Research Council Selection by compartmentalised screening
US9498759B2 (en) 2004-10-12 2016-11-22 President And Fellows Of Harvard College Compartmentalized screening by microfluidic control
US9562897B2 (en) 2010-09-30 2017-02-07 Raindance Technologies, Inc. Sandwich assays in droplets
US9562837B2 (en) 2006-05-11 2017-02-07 Raindance Technologies, Inc. Systems for handling microfludic droplets

Families Citing this family (40)

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WO1997039019A1 (en) * 1996-04-12 1997-10-23 University Of Pittsburgh Novel cationic cholesteryl derivatives containing cyclic polar groups
US6258792B1 (en) 1996-04-12 2001-07-10 University Of Pittsburgh Cationic cholesteryl derivatives containing cyclic polar groups
FR2751222B1 (en) 1996-07-16 1998-10-09 Capsulis COMPOSITIONS CONTAINING AT LEAST ONE NUCLEIC ACID AND THEIR APPLICATIONS IN THE BIOMEDICAL FIELD, PARTICULARLY IN GENE THERAPY
ES2212305T3 (en) * 1997-06-23 2004-07-16 Alza Corporation POLINUCLEOTIDES ENCAPSULATED IN LIPOSOMES, COMPOSITION AND PROCEDURE.
ATE278013T1 (en) 1997-07-07 2004-10-15 Medical Res Council IN VITRO SELECTION METHOD
FR2766706B1 (en) * 1997-07-30 2001-05-25 Biovector Therapeutics Sa STABLE PARTICULATE COMPLEXES OF NEUTRAL OR NEGATIVE GLOBAL LOAD OF MULTILAMELLAR STRUCTURE COMPOSED OF AT LEAST ONE BIOLOGICALLY ACTIVE GLOBALLY ANIONIC SUBSTANCE AND A CATIONIC COMPONENT, THEIR PREPARATION AND USE
FR2766705B1 (en) * 1997-07-30 2001-05-25 Biovector Therapeutics PARTICLE COMPLEX OF NEUTRAL OR NEGATIVE GLOBAL CHARGE FORMED OF A CATIONIC OR NEUTRAL VESICLE AND A BIOLOGICALLY ACTIVE ANIONIC SUBSTANCE, THEIR PREPARATION AND THEIR USE
US6372720B1 (en) * 1998-02-05 2002-04-16 Kenneth J. Longmuir Liposome fusion and delivery vehicle
US6903196B1 (en) 1999-06-17 2005-06-07 Utah Ventures Ii, L.P. Methods for identifying and isolating tissue-specific lumen-exposed molecules
GB0015624D0 (en) * 2000-06-26 2000-08-16 Nat Cancer Centre Materials and methods relating to improved gene delivery
EP1209469A1 (en) * 2000-11-22 2002-05-29 Vectron Therapeutics AG Production and use of a targeted diagnostic system
US6713055B2 (en) 2000-11-27 2004-03-30 Geron Corporation Glycosyltransferase vectors for treating cancer
US7622446B2 (en) 2001-04-18 2009-11-24 The Open University Polypeptides, derivatives and uses thereof
US7491702B2 (en) 2001-04-18 2009-02-17 The Open University Polypeptides related to amyloid precursor protein, pharmaceutical compositions thereof, and methods of treatment using the same
EP2316849A2 (en) * 2001-04-18 2011-05-04 The Open University Polypeptides comprising RER, derivatives and uses thereof
AU2002256398A2 (en) * 2001-04-30 2002-11-11 Targeted Genetics Corporation Lipid-comprising drug delivery complexes and methods for their production
JP2005501011A (en) * 2001-06-08 2005-01-13 ユタ ベンチャー ザ セカンド リミテッド パートナーシップ Tissue-specific inner membrane protein
WO2004039406A1 (en) * 2002-11-01 2004-05-13 Genomidea Inc. Medicinal preparation having chemotherapeutic encapsulated therein
GB0307428D0 (en) 2003-03-31 2003-05-07 Medical Res Council Compartmentalised combinatorial chemistry
US20060078893A1 (en) 2004-10-12 2006-04-13 Medical Research Council Compartmentalised combinatorial chemistry by microfluidic control
EA037929B1 (en) 2005-03-23 2021-06-08 Генмаб А/С ANTIBODIES TO HUMAN CD38 AND THEIR APPLICATION
WO2008097559A2 (en) 2007-02-06 2008-08-14 Brandeis University Manipulation of fluids and reactions in microfluidic systems
WO2008130623A1 (en) 2007-04-19 2008-10-30 Brandeis University Manipulation of fluids, fluid components and reactions in microfluidic systems
DK2222697T3 (en) 2007-11-01 2013-03-11 Perseid Therapeutics Llc Immunosuppressive polypeptides and nucleic acids
US12038438B2 (en) 2008-07-18 2024-07-16 Bio-Rad Laboratories, Inc. Enzyme quantification
EP2315629B1 (en) 2008-07-18 2021-12-15 Bio-Rad Laboratories, Inc. Droplet libraries
WO2010111231A1 (en) 2009-03-23 2010-09-30 Raindance Technologies, Inc. Manipulation of microfluidic droplets
EP2486409A1 (en) 2009-10-09 2012-08-15 Universite De Strasbourg Labelled silica-based nanomaterial with enhanced properties and uses thereof
WO2011079176A2 (en) 2009-12-23 2011-06-30 Raindance Technologies, Inc. Microfluidic systems and methods for reducing the exchange of molecules between droplets
US10351905B2 (en) 2010-02-12 2019-07-16 Bio-Rad Laboratories, Inc. Digital analyte analysis
EP3412778A1 (en) 2011-02-11 2018-12-12 Raindance Technologies, Inc. Methods for forming mixed droplets
WO2012112804A1 (en) 2011-02-18 2012-08-23 Raindance Technoligies, Inc. Compositions and methods for molecular labeling
EP3709018A1 (en) 2011-06-02 2020-09-16 Bio-Rad Laboratories, Inc. Microfluidic apparatus for identifying components of a chemical reaction
US8658430B2 (en) 2011-07-20 2014-02-25 Raindance Technologies, Inc. Manipulating droplet size
US9340784B2 (en) * 2012-03-19 2016-05-17 Ionis Pharmaceuticals, Inc. Methods and compositions for modulating alpha-1-antitrypsin expression
US11901041B2 (en) 2013-10-04 2024-02-13 Bio-Rad Laboratories, Inc. Digital analysis of nucleic acid modification
US9944977B2 (en) 2013-12-12 2018-04-17 Raindance Technologies, Inc. Distinguishing rare variations in a nucleic acid sequence from a sample
US11193176B2 (en) 2013-12-31 2021-12-07 Bio-Rad Laboratories, Inc. Method for detecting and quantifying latent retroviral RNA species
US10647981B1 (en) 2015-09-08 2020-05-12 Bio-Rad Laboratories, Inc. Nucleic acid library generation methods and compositions
CA3099497A1 (en) * 2018-05-15 2019-11-21 Flagship Pioneering Innovations V, Inc. Fusosome compositions and uses thereof

Citations (5)

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EP0475178A1 (en) * 1990-08-27 1992-03-18 Kabushiki Kaisha Vitamin Kenkyusyo Liposome for entrapping gene, liposomal preparation and process for the manufacture of the preparation
EP0497997A1 (en) * 1991-02-02 1992-08-12 Nika Health Products Limited Synthetic membrane vesicles containing functionally active fusion peptides as drug delivery systems
EP0544292A2 (en) * 1991-11-27 1993-06-02 Roche Diagnostics GmbH Method of introduction of nucleic acids in cells
WO1994018947A1 (en) * 1993-02-16 1994-09-01 Sharifa Karali High purity protamine-dna complex and use of same
WO1996040964A2 (en) * 1995-06-07 1996-12-19 Inex Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0475178A1 (en) * 1990-08-27 1992-03-18 Kabushiki Kaisha Vitamin Kenkyusyo Liposome for entrapping gene, liposomal preparation and process for the manufacture of the preparation
EP0497997A1 (en) * 1991-02-02 1992-08-12 Nika Health Products Limited Synthetic membrane vesicles containing functionally active fusion peptides as drug delivery systems
EP0544292A2 (en) * 1991-11-27 1993-06-02 Roche Diagnostics GmbH Method of introduction of nucleic acids in cells
WO1994018947A1 (en) * 1993-02-16 1994-09-01 Sharifa Karali High purity protamine-dna complex and use of same
WO1996040964A2 (en) * 1995-06-07 1996-12-19 Inex Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer

Cited By (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7335509B2 (en) 1995-01-23 2008-02-26 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US7361640B2 (en) 1995-01-23 2008-04-22 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US9448172B2 (en) 2003-03-31 2016-09-20 Medical Research Council Selection by compartmentalised screening
US8871444B2 (en) 2004-10-08 2014-10-28 Medical Research Council In vitro evolution in microfluidic systems
US9186643B2 (en) 2004-10-08 2015-11-17 Medical Research Council In vitro evolution in microfluidic systems
US9498759B2 (en) 2004-10-12 2016-11-22 President And Fellows Of Harvard College Compartmentalized screening by microfluidic control
US9328344B2 (en) 2006-01-11 2016-05-03 Raindance Technologies, Inc. Microfluidic devices and methods of use in the formation and control of nanoreactors
US9410151B2 (en) 2006-01-11 2016-08-09 Raindance Technologies, Inc. Microfluidic devices and methods of use in the formation and control of nanoreactors
US9534216B2 (en) 2006-01-11 2017-01-03 Raindance Technologies, Inc. Microfluidic devices and methods of use in the formation and control of nanoreactors
US9273308B2 (en) 2006-05-11 2016-03-01 Raindance Technologies, Inc. Selection of compartmentalized screening method
US9562837B2 (en) 2006-05-11 2017-02-07 Raindance Technologies, Inc. Systems for handling microfludic droplets
US9012390B2 (en) 2006-08-07 2015-04-21 Raindance Technologies, Inc. Fluorocarbon emulsion stabilizing surfactants
US9498761B2 (en) 2006-08-07 2016-11-22 Raindance Technologies, Inc. Fluorocarbon emulsion stabilizing surfactants
US9228229B2 (en) 2010-02-12 2016-01-05 Raindance Technologies, Inc. Digital analyte analysis
US9366632B2 (en) 2010-02-12 2016-06-14 Raindance Technologies, Inc. Digital analyte analysis
US9399797B2 (en) 2010-02-12 2016-07-26 Raindance Technologies, Inc. Digital analyte analysis
US9562897B2 (en) 2010-09-30 2017-02-07 Raindance Technologies, Inc. Sandwich assays in droplets
US8841071B2 (en) 2011-06-02 2014-09-23 Raindance Technologies, Inc. Sample multiplexing

Also Published As

Publication number Publication date
AU6691496A (en) 1997-02-26
WO1997004748A2 (en) 1997-02-13

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