WO2010133975A3 - Reducible polyamino disulfides as transfectants - Google Patents

Reducible polyamino disulfides as transfectants Download PDF

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Publication number
WO2010133975A3
WO2010133975A3 PCT/IB2010/001472 IB2010001472W WO2010133975A3 WO 2010133975 A3 WO2010133975 A3 WO 2010133975A3 IB 2010001472 W IB2010001472 W IB 2010001472W WO 2010133975 A3 WO2010133975 A3 WO 2010133975A3
Authority
WO
WIPO (PCT)
Prior art keywords
reducible
disulfide
polynucleotide
polymer
transfectants
Prior art date
Application number
PCT/IB2010/001472
Other languages
French (fr)
Other versions
WO2010133975A2 (en
Inventor
Abderahim Aissaoui
Karsten Hipler
Original Assignee
Imuthes Limited
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 Imuthes Limited filed Critical Imuthes Limited
Publication of WO2010133975A2 publication Critical patent/WO2010133975A2/en
Publication of WO2010133975A3 publication Critical patent/WO2010133975A3/en

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    • 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
    • A61K9/0043—Nose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/20—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing sulfur, e.g. dimethyl sulfoxide [DMSO], docusate, sodium lauryl sulfate or aminosulfonic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/34—Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6921—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere
    • 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
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B82—NANOTECHNOLOGY
    • B82Y—SPECIFIC USES OR APPLICATIONS OF NANOSTRUCTURES; MEASUREMENT OR ANALYSIS OF NANOSTRUCTURES; MANUFACTURE OR TREATMENT OF NANOSTRUCTURES
    • B82Y5/00—Nanobiotechnology or nanomedicine, e.g. protein engineering or drug delivery
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Nanotechnology (AREA)
  • Biophysics (AREA)
  • Otolaryngology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Pulmonology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Medical Informatics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Inorganic Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The invention relates to polynucleotide transfection (e.g. of pDNA or siRNA), and in particular, the use of polyplexes (complexes of a polynucleotide and a reducible (disulfide) polymer) as delivery vehicles. Compositions of the invention contain a reducing agent, such as glutathione (GSH) or N-acetyl cysteine (NAC). These are able to reduce, and so break, S- S bonds in the reducible disulfide polymer formed form polymerisation of thiol-containing monomers. This can result in the release of the polynucleotide (e.g. pDNA or siRNA). The reducible (disulfide) polymer is preferably formed from constituent monomer(s) units having a thiol group at each end, and a middle repeating section or unit of a polyamine, such as a spermine molecule.
PCT/IB2010/001472 2009-05-22 2010-05-21 Reducible polyamino disulfides as transfectants WO2010133975A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GB0908963.2 2009-05-22
GBGB0908963.2A GB0908963D0 (en) 2009-05-22 2009-05-22 Compositions

Publications (2)

Publication Number Publication Date
WO2010133975A2 WO2010133975A2 (en) 2010-11-25
WO2010133975A3 true WO2010133975A3 (en) 2011-11-03

Family

ID=40862931

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IB2010/001472 WO2010133975A2 (en) 2009-05-22 2010-05-21 Reducible polyamino disulfides as transfectants

Country Status (2)

Country Link
GB (1) GB0908963D0 (en)
WO (1) WO2010133975A2 (en)

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999055825A1 (en) * 1998-04-30 1999-11-04 Mirus Corporation A process of making a compound by forming a polymer from a template drug
WO2000075162A1 (en) * 1999-06-07 2000-12-14 Mirus Corporation A compound containing a labile disulfide bond
WO2004074509A2 (en) * 2003-02-18 2004-09-02 Mirus Corporation Reversible attachment of a membrane active polymer to a polynucleotide
WO2005016355A1 (en) * 2003-07-28 2005-02-24 Mirus Corporation Intravascular delivery of non-viral nucleic acid

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2911197A (en) 1996-05-24 1998-01-05 Imperial College Of Science, Technology And Medicine Polycationic sterol derivatives as transfection agents
RU2003120456A (en) 2000-12-12 2005-01-27 Митсубиси Кемикэл Корп. (Jp) COMPOUND

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999055825A1 (en) * 1998-04-30 1999-11-04 Mirus Corporation A process of making a compound by forming a polymer from a template drug
WO2000075162A1 (en) * 1999-06-07 2000-12-14 Mirus Corporation A compound containing a labile disulfide bond
WO2004074509A2 (en) * 2003-02-18 2004-09-02 Mirus Corporation Reversible attachment of a membrane active polymer to a polynucleotide
WO2005016355A1 (en) * 2003-07-28 2005-02-24 Mirus Corporation Intravascular delivery of non-viral nucleic acid

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
LEE, Y. ET AL: "Visualization of the Degradation of a Disulfide Polymer, Linear Poly(ethylenimine sulfide), for Gene Delivery", BIOCONJUGATE CHEM., no. 18, 23 December 2006 (2006-12-23), pages 13 - 18, XP055003439, Retrieved from the Internet <URL:http://pubs.acs.org/doi/pdfplus/10.1021/bc060113t> [retrieved on 20110722] *
LIN, C. ET AL: "Novel Bioreducible Poly(amido amine)s for Highly Efficient Gene Delivery", BIOCONJUGATE CHEM., no. 18, 22 December 2006 (2006-12-22), pages 138 - 145, XP055003407, Retrieved from the Internet <URL:http://pubs.acs.org/doi/pdfplus/10.1021/bc060200l> [retrieved on 20110722] *

Also Published As

Publication number Publication date
GB0908963D0 (en) 2009-07-01
WO2010133975A2 (en) 2010-11-25

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