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WO2018140411A3 - Therapeutic polymeric nanoparticles for tailored gene expression - Google Patents

Therapeutic polymeric nanoparticles for tailored gene expression Download PDF

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Publication number
WO2018140411A3
WO2018140411A3 PCT/US2018/014898 US2018014898W WO2018140411A3 WO 2018140411 A3 WO2018140411 A3 WO 2018140411A3 US 2018014898 W US2018014898 W US 2018014898W WO 2018140411 A3 WO2018140411 A3 WO 2018140411A3
Authority
WO
WIPO (PCT)
Prior art keywords
nanoparticles
gene expression
polymeric nanoparticles
tailored
therapeutic polymeric
Prior art date
Application number
PCT/US2018/014898
Other languages
French (fr)
Other versions
WO2018140411A2 (en
Inventor
Louis Joseph BORN
John W. Harmon
Frank Lay
Guy MARTI
Christopher Ng
Zahra ALIKHASSY
Amir Ansari
Original Assignee
The Johns Hopkins University
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 The Johns Hopkins University filed Critical The Johns Hopkins University
Publication of WO2018140411A2 publication Critical patent/WO2018140411A2/en
Publication of WO2018140411A3 publication Critical patent/WO2018140411A3/en

Links

Classifications

    • 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
    • A61K47/6927—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 the form being a solid microparticle having no hollow or gas-filled cores
    • A61K47/6929—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 the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle
    • A61K47/6931—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 the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle the material constituting the nanoparticle being a polymer
    • A61K47/6939—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 the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle the material constituting the nanoparticle being a polymer the polymer being a polysaccharide, e.g. starch, chitosan, chitin, cellulose or pectin
    • 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
    • A61K48/0041—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 polymeric
    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Nanotechnology (AREA)
  • Biochemistry (AREA)
  • Manufacturing & Machinery (AREA)
  • Immunology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)

Abstract

A composition for tailored, non-viral polynucleotide delivery to a cell including a plurality of nanoparticles and a polynucleotide attached to an exterior surface of at least one of the plurality of nanoparticles and methods of using and making the composition thereof. Each of the plurality of nanoparticles has a natural or synthetic cationic polymer electrostatically crosslinked with an anionic electrostatic crosslinking agent. Each of the plurality of nanoparticles have a positive zeta potential, and each of the plurality of nanoparticles are between 1 nm - 1000 nm in diameter.
PCT/US2018/014898 2017-01-24 2018-01-23 Therapeutic polymeric nanoparticles for tailored gene expression WO2018140411A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762449884P 2017-01-24 2017-01-24
US62/449,884 2017-01-24

Publications (2)

Publication Number Publication Date
WO2018140411A2 WO2018140411A2 (en) 2018-08-02
WO2018140411A3 true WO2018140411A3 (en) 2018-10-18

Family

ID=62978628

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2018/014898 WO2018140411A2 (en) 2017-01-24 2018-01-23 Therapeutic polymeric nanoparticles for tailored gene expression

Country Status (1)

Country Link
WO (1) WO2018140411A2 (en)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4048287A4 (en) * 2019-10-25 2023-12-06 The Johns Hopkins University POLYMERIC NANOPARTICLES FOR INTRACELLULAR PROTEIN DELIVERY
CN114317478B (en) * 2022-01-05 2023-10-20 北京化工大学 Application of sucrose phosphorylase and method for preparing 2-alpha-glyceroglycosides by using sucrose phosphorylase

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100086613A1 (en) * 2008-10-03 2010-04-08 Chang-Jer Wu Chitosan vehicle and method for making same

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100086613A1 (en) * 2008-10-03 2010-04-08 Chang-Jer Wu Chitosan vehicle and method for making same

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
CHEN, L. ET AL.: "Chitosan/beta-lactoglobulin core-shell nanoparticles as nutraceutical carriers", BIOMATERIALS, vol. 26, no. 30, October 2005 (2005-10-01), pages 6041 - 6053, XP055546989 *
CUI, Z. ET AL.: "Intranasal administration of plasmid DNA-coated nanoparticles results in enhanced immune responses", JOURNAL OF PHARMACY AND PHARMACOLOGY, vol. 54, 2002, pages 1195 - 1203, XP055546994 *
KUMAR, A. ET AL.: "Microneedle-mediated transcutaneous immunization with plasmid DNA coated on cationic PLGA nanoparticles", JOURNAL OF CONTROLLED RELEASE, vol. 163, no. 2, October 2012 (2012-10-01), pages 230 - 239, XP055546991 *
TIYABOONCHAI, W. ET AL.: "Chitosan nanoparticles: a promising system for drug delivery", NARESUAN UNIVERSITY JOURNAL, vol. 11, no. 3, 2013, pages 51 - 66, XP055546997 *

Also Published As

Publication number Publication date
WO2018140411A2 (en) 2018-08-02

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