WO2014057432A3 - Multicomponent lipid nanoparticles and processes for the preparation thereof - Google Patents

Multicomponent lipid nanoparticles and processes for the preparation thereof Download PDF

Info

Publication number
WO2014057432A3
WO2014057432A3 PCT/IB2013/059230 IB2013059230W WO2014057432A3 WO 2014057432 A3 WO2014057432 A3 WO 2014057432A3 IB 2013059230 W IB2013059230 W IB 2013059230W WO 2014057432 A3 WO2014057432 A3 WO 2014057432A3
Authority
WO
WIPO (PCT)
Prior art keywords
processes
preparation
lipid nanoparticles
multicomponent lipid
multicomponent
Prior art date
Application number
PCT/IB2013/059230
Other languages
French (fr)
Other versions
WO2014057432A9 (en
WO2014057432A2 (en
Inventor
Giulio CARACCIOLO
Daniela POZZI
Simona MARCHINI
Augusto Amici
Original Assignee
Universita' Degli Studi Di Roma "La Sapienza"
Universita' Degli Studi Di Camerino
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 Universita' Degli Studi Di Roma "La Sapienza", Universita' Degli Studi Di Camerino filed Critical Universita' Degli Studi Di Roma "La Sapienza"
Publication of WO2014057432A2 publication Critical patent/WO2014057432A2/en
Publication of WO2014057432A9 publication Critical patent/WO2014057432A9/en
Publication of WO2014057432A3 publication Critical patent/WO2014057432A3/en

Links

Classifications

    • 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
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/337—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having four-membered rings, e.g. taxol
    • 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
    • A61K31/7028—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages
    • A61K31/7034—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin
    • A61K31/704—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin attached to a condensed carbocyclic ring system, e.g. sennosides, thiocolchicosides, escin, daunorubicin
    • 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
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine 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
    • 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
    • A61K47/6905—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 colloid or an emulsion
    • A61K47/6911—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 colloid or an emulsion the form being a liposome
    • 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
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • 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
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle

Abstract

The present invention relates to novel multicomponent lipid nanoparticles for the non-viral transport of nucleic acids and/or anti-tumoral drugs and to processes for the preparation thereof and their use in gene therapy and/or anti-tumoral therapy.
PCT/IB2013/059230 2012-10-09 2013-10-09 Multicomponent lipid nanoparticles and processes for the preparation thereof WO2014057432A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
ITRM2012A000480 2012-10-09
IT000480A ITRM20120480A1 (en) 2012-10-09 2012-10-09 MULTICOMPONENT LIPID NANOPARTICLES AND PROCEDURES FOR THEIR PREPARATION.

Publications (3)

Publication Number Publication Date
WO2014057432A2 WO2014057432A2 (en) 2014-04-17
WO2014057432A9 WO2014057432A9 (en) 2014-05-30
WO2014057432A3 true WO2014057432A3 (en) 2014-07-24

Family

ID=47278447

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/IB2013/059230 WO2014057432A2 (en) 2012-10-09 2013-10-09 Multicomponent lipid nanoparticles and processes for the preparation thereof

Country Status (2)

Country Link
IT (1) ITRM20120480A1 (en)
WO (1) WO2014057432A2 (en)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107106703A (en) * 2014-11-25 2017-08-29 纳米生物技术公司 Pharmaceutical composition, its preparation and use

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6507102B2 (en) 2013-02-05 2019-04-24 １グローブ ヘルス インスティテュート エルエルシー Biodegradable and Clinically Compatible Nanoparticles as Drug Delivery Carriers
MA38687A1 (en) 2013-05-30 2017-12-29 Nanobiotix Pharmaceutical composition, preparation and uses thereof
US10542918B2 (en) 2013-10-23 2020-01-28 Verily Life Sciences Llc Modulation of a response signal to distinguish between analyte and background signals
US9504405B2 (en) 2013-10-23 2016-11-29 Verily Life Sciences Llc Spatial modulation of magnetic particles in vasculature by external magnetic field
EA201791138A1 (en) * 2014-11-25 2017-09-29 Нанобиотикс PHARMACEUTICAL COMPOSITION, ITS PREPARATION AND APPLICATIONS
AR102781A1 (en) 2014-11-25 2017-03-22 Nanobiotix PHARMACEUTICAL COMPOSITION THAT COMBINES AT LEAST TWO DIFFERENT NANOPARTICLES AND A PHARMACEUTICAL COMPOUND, ITS PREPARATION AND ITS USES
ES2890662T3 (en) 2014-11-25 2022-01-21 Curadigm Sas Pharmaceutical compositions, preparation and uses thereof
PL3229843T3 (en) 2014-11-25 2020-06-29 Curadigm Sas Pharmaceutical composition, preparation and uses thereof
US9861710B1 (en) 2015-01-16 2018-01-09 Verily Life Sciences Llc Composite particles, methods, and in vivo diagnostic system
EP3265059A4 (en) 2015-03-03 2018-08-29 Cureport Inc. Combination liposomal pharmaceutical formulations
JP2018507227A (en) 2015-03-03 2018-03-15 キュアポート インコーポレイテッド Liposome drug formulation with two drugs
MA43100A (en) 2015-05-28 2018-09-05 Nanobiotix NANOPARTICLES FOR USE AS A THERAPEUTIC VACCINE
DE202016008594U1 (en) 2015-11-17 2018-08-23 Bioncotech Therapeutics S.L A novel pharmaceutical composition comprising particles comprising a complex of a double-stranded polyribonucleotide and a polyalkyleneimine
PL3448363T3 (en) 2017-05-17 2022-09-26 Highlight Therapeutics, S.L. Novel pharmaceutical composition comprising particles comprising a complex of a double-stranded polyribonucleotide and a polyalkyleneimine
JPWO2019131770A1 (en) * 2017-12-27 2020-12-24 武田薬品工業株式会社 Nucleic acid-containing lipid nanoparticles and their uses
WO2021016106A1 (en) * 2019-07-19 2021-01-28 University Of Florida Research Foundation Incorporated Multilamellar rna nanoparticles
CN110882383A (en) * 2019-11-26 2020-03-17 宁夏医科大学 Cationic liposome-protamine-mRNA tumor vaccine and preparation method and application method thereof
EP4099988A4 (en) * 2020-02-05 2024-03-13 Univ Florida Rna-loaded nanoparticles and use thereof for the treatment of cancer
EP4149425A1 (en) * 2020-05-15 2023-03-22 Translate Bio, Inc. Lipid nanoparticle formulations for mrna delivery
CN111603571A (en) * 2020-05-28 2020-09-01 中国药科大学 Mitochondrion targeting lipid-like carrier Mito-Alisposome, compound thereof, preparation method and application
CN113577039A (en) * 2021-07-01 2021-11-02 温州医科大学附属第二医院（温州医科大学附属育英儿童医院） Application of nano-particles formed by encapsulating protamine with erythrocyte membrane after compressing DNA and preparation method thereof
CN114657147B (en) * 2022-01-27 2023-12-01 安徽智飞龙科马生物制药有限公司 Protective agent for biological material and application thereof
CN114699538A (en) * 2022-04-02 2022-07-05 中国药科大学 Core-shell type efficient gene drug delivery system and preparation method thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010043630A2 (en) * 2008-10-13 2010-04-22 Sigmoid Pharma Limited A delivery system for rna
WO2012112730A2 (en) * 2011-02-15 2012-08-23 Merrimack Pharmaceuticals, Inc. Compositions and methods for delivering nucleic acid to a cell

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010043630A2 (en) * 2008-10-13 2010-04-22 Sigmoid Pharma Limited A delivery system for rna
WO2012112730A2 (en) * 2011-02-15 2012-08-23 Merrimack Pharmaceuticals, Inc. Compositions and methods for delivering nucleic acid to a cell

Non-Patent Citations (13)

* Cited by examiner, † Cited by third party
Title
CARACCIOLO ET AL: "Transfection efficiency boost by designer multicomponent lipoplexes", BIOCHIMICA ET BIOPHYSICA ACTA (BBA) - BIOMEMBRANES, ELSEVIER, AMSTERDAM, NL, vol. 1768, no. 9, 14 September 2007 (2007-09-14), pages 2280 - 2292, XP022244094, ISSN: 0005-2736, DOI: 10.1016/J.BBAMEM.2007.06.027 *
CARACCIOLO GIULIO ET AL: "DNA release from cationic liposome/DNA complexes by anionic lipids", APPLIED PHYSICS LETTERS, AIP, AMERICAN INSTITUTE OF PHYSICS, MELVILLE, NY, US, vol. 89, no. 23, 5 December 2006 (2006-12-05), pages 233903 - 233903, XP012087536, ISSN: 0003-6951, DOI: 10.1063/1.2399356 *
CARACCIOLO GIULIO ET AL: "On the correlation between phase evolution of lipoplexes/anionic lipid mixtures and DNA release", APPLIED PHYSICS LETTERS, AIP, AMERICAN INSTITUTE OF PHYSICS, MELVILLE, NY, US, vol. 91, no. 14, 4 October 2007 (2007-10-04), pages 143903 - 143903, XP012099609, ISSN: 0003-6951, DOI: 10.1063/1.2794436 *
CHEN J ET AL: "Transfection efficiency and intracellular fate of polycation liposomes combined with protamine", BIOMATERIALS, ELSEVIER SCIENCE PUBLISHERS BV., BARKING, GB, vol. 32, no. 5, 1 February 2011 (2011-02-01), pages 1412 - 1418, XP027537940, ISSN: 0142-9612, [retrieved on 20101102] *
COLONNA C ET AL: "Non-viral dried powders for respiratory gene delivery prepared by cationic and chitosan loaded liposomes", INTERNATIONAL JOURNAL OF PHARMACEUTICS, ELSEVIER BV, NL, vol. 364, no. 1, 19 November 2008 (2008-11-19), pages 108 - 118, XP025562425, ISSN: 0378-5173, [retrieved on 20080819], DOI: 10.1016/J.IJPHARM.2008.07.034 *
D. POZZI ET AL: "Mechanistic evaluation of the transfection barriers involved in lipid-mediated gene delivery: Interplay between nanostructure and composition", BIOCHIMICA ET BIOPHYSICA ACTA (BBA) - BIOMEMBRANES, vol. 1838, no. 3, 1 March 2014 (2014-03-01), pages 957 - 967, XP055107607, ISSN: 0005-2736, DOI: 10.1016/j.bbamem.2013.11.014 *
D. POZZI ET AL: "Mechanistic Understanding of Gene Delivery Mediated by Highly Efficient Multicomponent Envelope-Type Nanoparticle Systems", MOLECULAR PHARMACEUTICS, vol. 10, no. 12, 2 December 2013 (2013-12-02), pages 4654 - 4665, XP055106503, ISSN: 1543-8384, DOI: 10.1021/mp400470p *
GIULIO CARACCIOLO ET AL: "Efficient Escape from Endosomes Determines the Superior Efficiency of Multicomponent Lipoplexes", THE JOURNAL OF PHYSICAL CHEMISTRY B, vol. 113, no. 15, 16 April 2009 (2009-04-16), pages 4995 - 4997, XP055069393, ISSN: 1520-6106, DOI: 10.1021/jp811423r *
GIULIO CARACCIOLO ET AL: "Factors determining the superior performance of lipid/DNA/protammine nanoparticles over lipoplexes", JOURNAL OF MEDICINAL CHEMISTRY, 13 May 2011 (2011-05-13), pages 4160 - 4171, XP055069350, Retrieved from the Internet <URL:http://pubs.acs.org/doi/ipdf/10.1021/jm200237p> [retrieved on 20130703] *
MING-KUNG YEH ET AL: "Clinical development of liposome based drugs: formulation, characterization, and therapeutic efficacy", INTERNATIONAL JOURNAL OF NANOMEDICINE, 1 December 2011 (2011-12-01), pages 49, XP055069345, DOI: 10.2147/IJN.S26766 *
POZZI DANIELA ET AL: "Programmed packaging of multicomponent envelope-type nanoparticle system for gene delivery", APPLIED PHYSICS LETTERS, AIP, AMERICAN INSTITUTE OF PHYSICS, MELVILLE, NY, US, vol. 96, no. 18, 5 May 2010 (2010-05-05), pages 183702 - 183702, XP012131040, ISSN: 0003-6951, DOI: 10.1063/1.3427354 *
SARDER NASIR UDDIN: "Cationic lipids used in non-viral gene delivery systems", BIOTECHNOLOGY AND MOLECULAR BIOLOGY REVIEW, vol. 2, no. 3, 1 August 2007 (2007-08-01), pages 58 - 67, XP055069508 *
SIMONA MOTTA: "Nanoparticles systems for gene delivery interacting with model cell membranes: a structural study", FISMAT2013, 1 July 2013 (2013-07-01), XP055108194, Retrieved from the Internet <URL:http://www.fisi.polimi.it/it/fismat2013/submission/view/409> [retrieved on 20140317] *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107106703A (en) * 2014-11-25 2017-08-29 纳米生物技术公司 Pharmaceutical composition, its preparation and use

Also Published As

Publication number Publication date
WO2014057432A9 (en) 2014-05-30
WO2014057432A2 (en) 2014-04-17
ITRM20120480A1 (en) 2014-04-10

Similar Documents

Publication Publication Date Title
WO2014057432A3 (en) Multicomponent lipid nanoparticles and processes for the preparation thereof
IL289934B (en) Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
IL286515A (en) Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
IL283545A (en) Lipids and lipid nanoparticle formulations for delivery of nucleic acids
HK1255336A1 (en) Pharmaceutical formulations and processes of preparation thereof
MX2019005287A (en) Cationic lipids for nucleic acid delivery and preparation thereof.
WO2013170170A3 (en) Compositions and methods for gene therapy
WO2013028942A8 (en) Targeting microbubbles
EP3303598A4 (en) Targeted lipid particles for systemic delivery of nucleic acid molecules to leukocytes
PH12015500758A1 (en) Pyrrolo[3,2-d]pyrimidine derivatives for the treatment of viral infections and other diseases
EP3204032A4 (en) Efficient delivery of therapeutic molecules in vitro and in vivo
WO2012116073A3 (en) Amphiphilic dendron-coils, micelles thereof and uses
IL231536A (en) Indazole-3-carboxamides , pharmaceutical compositions comprising them and their use in the treatment of diseases
HK1243718A1 (en) Fgfr3 fusion gene and pharmaceutical drug targeting same
EP3033113A4 (en) A novel plga-modified polyethylenimine self-assembly nanotechnology for nucleic acid and drug delivery
WO2014145159A3 (en) Charge-engineered antibodies or compositions of penetration-enhanced targeting proteins and methods of use
EP2849770A4 (en) Pharmaceutical compositions for the delivery of substantially water-insoluble drugs
IL226205B (en) Fusion proteins, pharmaceutical compositions comprising them and their use in the manufacture of medicaments for cancer therapy
EP2964770A4 (en) In vitro and in vivo delivery of genes and proteins using the bacteriophage t4 dna packaging machine
IN2014MN01886A (en)
GB201222455D0 (en) Novel pharmaceutical formulations and their use in the treatment of periodontaldisease
WO2013190509A3 (en) Preparation of intermediates of boceprevir
EP3291838A4 (en) Anionic nanoparticles for use in the delivery of anionic small molecule drugs
WO2013109213A3 (en) Pharmaceutical formulations comprising tiotropium

Legal Events

Date Code Title Description
121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 13812096

Country of ref document: EP

Kind code of ref document: A2

122 Ep: pct application non-entry in european phase

Ref document number: 13812096

Country of ref document: EP

Kind code of ref document: A2