AU2001288598A1 - A novel cationic lipopolymer as biocompatible gene delivery agent - Google Patents

A novel cationic lipopolymer as biocompatible gene delivery agent

Info

Publication number
AU2001288598A1
AU2001288598A1 AU2001288598A AU8859801A AU2001288598A1 AU 2001288598 A1 AU2001288598 A1 AU 2001288598A1 AU 2001288598 A AU2001288598 A AU 2001288598A AU 8859801 A AU8859801 A AU 8859801A AU 2001288598 A1 AU2001288598 A1 AU 2001288598A1
Authority
AU
Australia
Prior art keywords
gene delivery
delivery agent
cationic lipopolymer
novel cationic
pei
Prior art date
Legal status (The legal status 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 status listed.)
Abandoned
Application number
AU2001288598A
Inventor
Darin Y. Furgeson
Sang-Oh Han
Ram I. Mahato
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Expression Genetics Inc
Original Assignee
Expression Genetics Inc
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 Expression Genetics Inc filed Critical Expression Genetics Inc
Publication of AU2001288598A1 publication Critical patent/AU2001288598A1/en
Abandoned legal-status Critical Current

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
    • 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
    • A61K47/62—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 a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents

Abstract

A biodegradable non-toxic cationic lipopolymer comprising a branched polyethylenimine (PEI), a lipid anchor, biocompatible hydrophilic polymer spacer, and a biodegradable linker which covalently links the branched PEI, the spacer and the cholesterol derived lipid anchor. The cationic lipopolymers in the present invention can be used in drug delivery and are especially useful for delivery of a nucleic acid or any anionic bioactive agent to various organs and tissues after local or systemic administration.
AU2001288598A 2000-09-14 2001-08-31 A novel cationic lipopolymer as biocompatible gene delivery agent Abandoned AU2001288598A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US09/662,511 2000-09-14
US09/662,511 US6696038B1 (en) 2000-09-14 2000-09-14 Cationic lipopolymer as biocompatible gene delivery agent
PCT/US2001/027181 WO2002022174A1 (en) 2000-09-14 2001-08-31 A novel cationic lipopolymer as biocompatible gene delivery agent

Publications (1)

Publication Number Publication Date
AU2001288598A1 true AU2001288598A1 (en) 2002-03-26

Family

ID=24658024

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2001288598A Abandoned AU2001288598A1 (en) 2000-09-14 2001-08-31 A novel cationic lipopolymer as biocompatible gene delivery agent

Country Status (9)

Country Link
US (2) US6696038B1 (en)
EP (1) EP1320386B1 (en)
JP (1) JP2004522809A (en)
AT (1) ATE502652T1 (en)
AU (1) AU2001288598A1 (en)
CA (1) CA2420495C (en)
DE (1) DE60144281D1 (en)
ES (1) ES2364006T3 (en)
WO (1) WO2002022174A1 (en)

Families Citing this family (156)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070173465A9 (en) * 1995-10-11 2007-07-26 Monahan Sean D Expression of zeta negative and zeta positive nucleic acids using a dystrophin gene
ES2617692T3 (en) * 2000-07-21 2017-06-19 Revance Therapeutics, Inc. Multi-component biological agent transport systems
US20040220100A1 (en) * 2000-07-21 2004-11-04 Essentia Biosystems, Inc. Multi-component biological transport systems
US20040142474A1 (en) * 2000-09-14 2004-07-22 Expression Genetics, Inc. Novel cationic lipopolymer as a biocompatible gene delivery agent
DE10065710A1 (en) * 2000-12-29 2002-07-04 Bayer Ag Medicament containing a polyamine as an active substance
US6875611B2 (en) * 2001-07-03 2005-04-05 University Of Utah Research Foundation Soluble steroidal peptides for nucleic acid delivery
WO2003015757A1 (en) * 2001-08-16 2003-02-27 The Trustees Of The University Of Pennsylvania Synthesis and use of reagents for improved dna lipofection and/or slow release prodrug and drug therapies
US20030215395A1 (en) * 2002-05-14 2003-11-20 Lei Yu Controllably degradable polymeric biomolecule or drug carrier and method of synthesizing said carrier
EA200401565A1 (en) * 2002-05-24 2005-04-28 Неофарм, Инк. METHOD FOR OBTAINING CARDIOLYPINE OR ANALOGUE OF CARDIOLIPINE (OPTIONS), METHOD FOR OBTAINING LIPOSOME AND COMPOSITION OF CARDIOLIPINE FOR TREATING DISEASES (OPTIONS)
JP2006518701A (en) * 2002-05-24 2006-08-17 ネオファーム、インコーポレイティッド Cardiolipin composition, process for its production and use
GB0221942D0 (en) * 2002-09-20 2002-10-30 Univ Strathclyde Drug delivery
US20050277611A1 (en) * 2002-10-16 2005-12-15 Neopharm, Inc. Cationic cardiolipin analoges and its use thereof
US20050026859A1 (en) * 2002-11-12 2005-02-03 John Hilfinger Methods and compositions of gene delivery agents for systemic and local therapy
US20040138154A1 (en) 2003-01-13 2004-07-15 Lei Yu Solid surface for biomolecule delivery and high-throughput assay
US6878374B2 (en) * 2003-02-25 2005-04-12 Nitto Denko Corporation Biodegradable polyacetals
WO2005007819A2 (en) 2003-07-09 2005-01-27 Wisconsin Alumni Research Foundation Charge-dynamic polymers and delivery of anionic compounds
US8133733B2 (en) 2003-10-24 2012-03-13 Gencia Corporation Nonviral vectors for delivering polynucleotides to target tissues
US20090123468A1 (en) 2003-10-24 2009-05-14 Gencia Corporation Transducible polypeptides for modifying metabolism
AU2004297533B2 (en) 2003-10-24 2010-04-29 Gencia Corporation Methods and compositions for delivering polynucleotides
US8062891B2 (en) 2003-10-24 2011-11-22 Gencia Corporation Nonviral vectors for delivering polynucleotides to plants
US8507277B2 (en) 2003-10-24 2013-08-13 Gencia Corporation Nonviral vectors for delivering polynucleotides
US7163677B2 (en) 2003-10-24 2007-01-16 Nitto Denko Corporation Cationic polymers having degradable crosslinks
US20090208478A1 (en) * 2003-10-24 2009-08-20 Gencia Corporation Transducible polypeptides for modifying metabolism
CA2558676C (en) * 2004-03-03 2019-04-16 Essentia Biosystems, Inc. Compositions and methods for topical diagnostic and therapeutic transport
US9211248B2 (en) 2004-03-03 2015-12-15 Revance Therapeutics, Inc. Compositions and methods for topical application and transdermal delivery of botulinum toxins
TR201815364T4 (en) 2004-03-03 2018-11-21 Revance Therapeutics Inc Topical application and transdermal distribution of botulinum toxins.
US8821859B2 (en) * 2004-05-19 2014-09-02 Agency For Science, Technology And Research Methods and articles for the delivery of therapeutic agents
FR2870741B1 (en) * 2004-05-25 2008-03-14 Coletica Sa HYDRATED LAMELLAR OR LIPOSOME PHASE CONTAINING A FATTY MONOAMINE OR A CATIONIC POLYMER PROMOTING INTERCELLULAR PENETRATION AND A COSMETIC OR PHARMACEUTICAL COMPOSITION CONTAINING THE SAME.
WO2006033679A2 (en) * 2004-05-25 2006-03-30 Chimeracore, Inc. Self-assembling nanoparticle drug delivery system
WO2006012625A2 (en) * 2004-07-22 2006-02-02 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Stat3 decoy oligonucleotides and uses therefor
WO2006027787A1 (en) * 2004-09-09 2006-03-16 Yissum Research Development Company Of The Hebrew University Of Jerusalem Liposomal compositions of glucocorticoid and glucocorticoid derivatives
US7358223B2 (en) * 2004-10-04 2008-04-15 Nitto Denko Corporation Biodegradable cationic polymers
US8057821B2 (en) * 2004-11-03 2011-11-15 Egen, Inc. Biodegradable cross-linked cationic multi-block copolymers for gene delivery and methods of making thereof
US20060105052A1 (en) * 2004-11-15 2006-05-18 Acar Havva Y Cationic nanoparticle having an inorganic core
US7964571B2 (en) * 2004-12-09 2011-06-21 Egen, Inc. Combination of immuno gene therapy and chemotherapy for treatment of cancer and hyperproliferative diseases
US20120269886A1 (en) 2004-12-22 2012-10-25 Nitto Denko Corporation Therapeutic agent for pulmonary fibrosis
EP1842557B1 (en) 2004-12-22 2013-10-23 Nitto Denko Corporation Drug carrier and drug carrier kit for inhibiting fibrosis
US8022179B2 (en) * 2005-03-03 2011-09-20 Revance Therapeutics, Inc. Compositions and methods for topical application and transdermal delivery of an oligopeptide
WO2006094263A2 (en) * 2005-03-03 2006-09-08 Revance Therapeutics, Inc. Compositions and methods for topical application and transdermal delivery of botulinum toxins
US8734851B2 (en) * 2005-04-29 2014-05-27 Wisconsin Alumni Research Foundation Localized delivery of nucleic acid by polyelectrolyte assemblies
JP2007043972A (en) * 2005-08-10 2007-02-22 Tokyo Institute Of Technology Apatite particle and method for producing the same, gene-apatite particle complex, method for producing the same, and method for gene transfer and kit for gene transfer
CN101355962A (en) * 2005-11-17 2009-01-28 雷文斯治疗公司 Compositions and methods of topical application and transdermal delivery of botulinum toxins with reduced non-toxin proteins
US9572886B2 (en) 2005-12-22 2017-02-21 Nitto Denko Corporation Agent for treating myelofibrosis
US7700541B2 (en) * 2006-04-06 2010-04-20 Nitto Denko Corporation Biodegradable cationic polymers
EP2018156B1 (en) * 2006-04-07 2010-03-17 Chimeros, Inc. Compositions and methods for treating b- cell malignancies
US8946200B2 (en) * 2006-11-02 2015-02-03 Southwest Research Institute Pharmaceutically active nanosuspensions
NZ598039A (en) * 2006-12-29 2013-08-30 Revance Therapeutics Inc Compositions and methods of topical application and transdermal delivery of botulinum toxins stabilized with polypeptide fragments derived from HIV-TAT
NZ598159A (en) * 2006-12-29 2013-08-30 Revance Therapeutics Inc Transport molecules using reverse sequence hiv-tat polypeptides
US8834918B2 (en) * 2007-01-22 2014-09-16 Wisconsin Alumni Research Foundation Modified multilayered film
TWI407971B (en) 2007-03-30 2013-09-11 Nitto Denko Corp Cancer cells and tumor-related fibroblasts
AU2008236566A1 (en) * 2007-04-09 2008-10-16 Chimeros, Inc. Self-assembling nanoparticle drug delivery system
US20080312174A1 (en) * 2007-06-05 2008-12-18 Nitto Denko Corporation Water soluble crosslinked polymers
BR122019000266B1 (en) * 2007-07-26 2021-09-14 Revance Therapeutics, Inc PHARMACEUTICAL OR COSMETIC COMPOSITION INCLUDING A CATIONIC PEPTIDE AND KIT FOR ADMINISTRATION
US20090042825A1 (en) * 2007-08-06 2009-02-12 Majed Matar Composition, method of preparation & application of concentrated formulations of condensed nucleic acids with a cationic lipopolymer
US9144546B2 (en) 2007-08-06 2015-09-29 Clsn Laboratories, Inc. Nucleic acid-lipopolymer compositions
US8487079B2 (en) * 2007-08-08 2013-07-16 University of Pittsburgh—of the Commonwealth System of Higher Education Use of mitochondria-targeted electron scavengers as anti-inflammatory agents
US8003621B2 (en) 2007-09-14 2011-08-23 Nitto Denko Corporation Drug carriers
US20090105375A1 (en) * 2007-10-09 2009-04-23 Lynn David M Ultrathin Multilayered Films for Controlled Release of Anionic Reagents
US8404850B2 (en) * 2008-03-13 2013-03-26 Southwest Research Institute Bis-quaternary pyridinium-aldoxime salts and treatment of exposure to cholinesterase inhibitors
CA2717370A1 (en) * 2008-03-14 2009-09-17 Egen, Inc. Biodegradable cross-linked branched poly (alkylene imines)
AU2015270888B2 (en) * 2008-05-28 2017-11-16 Wayne State University Method and composition for a protein transduction technology and its applications
US8722348B2 (en) 2008-05-28 2014-05-13 Wayne State University Method and composition for a protein transduction technology and its applications
US8324333B2 (en) * 2008-06-05 2012-12-04 Wisconsin Alumni Research Foundation Anionic charge-dynamic polymers for release of cationic agents
US8722706B2 (en) * 2008-08-15 2014-05-13 Southwest Research Institute Two phase bioactive formulations of bis-quaternary pyridinium oxime sulfonate salts
US8309134B2 (en) * 2008-10-03 2012-11-13 Southwest Research Institute Modified calcium phosphate nanoparticle formation
TWI373368B (en) * 2008-11-11 2012-10-01 Univ Nat Chiao Tung Liposome and method for producing the same
WO2010120874A2 (en) 2009-04-14 2010-10-21 Chimeros, Inc. Chimeric therapeutics, compositions, and methods for using same
WO2011084694A1 (en) 2009-12-17 2011-07-14 University Of Pittsburgh-Of The Commonwealth System Of Higher Education Stabilized stat3 decoy oligonucleotides and uses therefor
US9028873B2 (en) * 2010-02-08 2015-05-12 Southwest Research Institute Nanoparticles for drug delivery to the central nervous system
JP5510808B2 (en) * 2010-03-10 2014-06-04 独立行政法人産業技術総合研究所 Nucleic acid introduction agent comprising organic nanotubes
CN101864078B (en) * 2010-06-01 2011-12-07 浙江大学 Polyethyleneimine-chitosan-octadecanoic acid grafting, preparation and application
CA2807552A1 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
ES2862955T3 (en) 2010-10-01 2021-10-08 Modernatx Inc Manipulated nucleic acids and methods of using them
KR20130108655A (en) * 2010-12-29 2013-10-04 에프. 호프만-라 로슈 아게 Small molecule conjugates for intracellular delivery of nucleic acids
US8710200B2 (en) 2011-03-31 2014-04-29 Moderna Therapeutics, Inc. Engineered nucleic acids encoding a modified erythropoietin and their expression
CA2847888A1 (en) 2011-09-09 2013-03-14 Biomed Realty, L.P. Methods and compositions for controlling assembly of viral proteins
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
WO2013052523A1 (en) 2011-10-03 2013-04-11 modeRNA Therapeutics Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
GB201117538D0 (en) 2011-10-11 2011-11-23 Royal Veterinary College The Methods
CN104114572A (en) 2011-12-16 2014-10-22 现代治疗公司 Modified nucleoside, nucleotide, and nucleic acid compositions
JP6126075B2 (en) 2012-03-02 2017-05-10 国立研究開発法人科学技術振興機構 Method for constructing functional nucleic acid molecule, and nucleic acid combination used in the method
EP2833920A2 (en) 2012-04-02 2015-02-11 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
AU2013243949A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
US9878056B2 (en) 2012-04-02 2018-01-30 Modernatx, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
US8703197B2 (en) * 2012-09-13 2014-04-22 International Business Machines Corporation Branched polyamines for delivery of biologically active materials
US9109087B2 (en) * 2012-09-13 2015-08-18 International Business Machines Corporation Low molecular weight branched polyamines for delivery of biologically active materials
PL2922554T3 (en) 2012-11-26 2022-06-20 Modernatx, Inc. Terminally modified rna
EP2946014A2 (en) 2013-01-17 2015-11-25 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
US20160024181A1 (en) 2013-03-13 2016-01-28 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
US10258698B2 (en) 2013-03-14 2019-04-16 Modernatx, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
AU2014287009B2 (en) 2013-07-11 2020-10-29 Modernatx, Inc. Compositions comprising synthetic polynucleotides encoding CRISPR related proteins and synthetic sgRNAs and methods of use
AU2014315287A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
US20160194368A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Circular polynucleotides
WO2015048744A2 (en) 2013-09-30 2015-04-02 Moderna Therapeutics, Inc. Polynucleotides encoding immune modulating polypeptides
EP3052521A1 (en) 2013-10-03 2016-08-10 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
JP6415582B2 (en) 2013-10-25 2018-10-31 ウェイン ステート ユニバーシティー Methods, systems, and compositions for cell transformation by protein-induced in vivo cell reprogramming
WO2015095351A1 (en) 2013-12-19 2015-06-25 Novartis Ag LEPTIN mRNA COMPOSITIONS AND FORMULATIONS
SG11201608798YA (en) 2014-04-23 2016-11-29 Modernatx Inc Nucleic acid vaccines
EP4159741A1 (en) 2014-07-16 2023-04-05 ModernaTX, Inc. Method for producing a chimeric polynucleotide encoding a polypeptide having a triazole-containing internucleotide linkage
US20170210788A1 (en) 2014-07-23 2017-07-27 Modernatx, Inc. Modified polynucleotides for the production of intrabodies
CN104224718B (en) * 2014-10-09 2017-07-11 辽宁中医药大学 A kind of multifunctional targeted vinorelbine liposome and preparation method thereof
CA2978885A1 (en) * 2015-03-06 2016-09-15 The University Of North Carolina At Chapel Hill Polymeric metformin and its use as a therapeutic agent and as a delivery vehicle
HRP20220156T1 (en) 2015-09-17 2022-04-15 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
HRP20220652T1 (en) 2015-12-10 2022-06-24 Modernatx, Inc. Compositions and methods for delivery of therapeutic agents
DK3394030T3 (en) 2015-12-22 2022-03-28 Modernatx Inc COMPOUNDS AND COMPOSITIONS FOR INTRACELLULAR RELEASE OF FUNDS
US11090389B2 (en) 2016-10-13 2021-08-17 University of Pittsburgh—of the Commonwealth System of Higher Education Mitochondrially targeted PARP inhibitor, and uses thereof
WO2018089540A1 (en) 2016-11-08 2018-05-17 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
RS63953B1 (en) 2017-03-15 2023-02-28 Modernatx Inc Compound and compositions for intracellular delivery of therapeutic agents
ES2911186T3 (en) 2017-03-15 2022-05-18 Modernatx Inc Crystalline forms of aminolipids
WO2018187590A1 (en) 2017-04-05 2018-10-11 Modernatx, Inc. Reduction or elimination of immune responses to non-intravenous, e.g., subcutaneously administered therapeutic proteins
JP7285220B2 (en) 2017-05-18 2023-06-01 モデルナティエックス インコーポレイテッド Lipid nanoparticles comprising linked interleukin-12 (IL12) polypeptide-encoding polynucleotides
EP4253544A3 (en) 2017-05-18 2023-12-20 ModernaTX, Inc. Modified messenger rna comprising functional rna elements
CN107184552B (en) * 2017-06-07 2021-03-30 东华大学 Preparation method of drug-loaded ethosome modified by galactosylated polyethyleneimine
MA49395A (en) 2017-06-14 2020-04-22 Modernatx Inc POLYNUCLEOTIDES COAGULATION FACTOR VIII CODING
WO2018232357A1 (en) 2017-06-15 2018-12-20 Modernatx, Inc. Rna formulations
EP3675817A1 (en) 2017-08-31 2020-07-08 Modernatx, Inc. Methods of making lipid nanoparticles
EP4219715A3 (en) 2017-09-08 2023-09-06 MiNA Therapeutics Limited Stabilized cebpa sarna compositions and methods of use
WO2019104195A1 (en) 2017-11-22 2019-05-31 Modernatx, Inc. Polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits for the treatment of propionic acidemia
JP7423522B2 (en) 2017-11-22 2024-01-29 モダーナティエックス・インコーポレイテッド Polynucleotide encoding ornithine transcarbamylase for the treatment of urea cycle disorders
JP7423521B2 (en) 2017-11-22 2024-01-29 モダーナティエックス・インコーポレイテッド Polynucleotide encoding phenylalanine hydroxylase for the treatment of phenylketonuria
WO2019136241A1 (en) 2018-01-05 2019-07-11 Modernatx, Inc. Polynucleotides encoding anti-chikungunya virus antibodies
US11566246B2 (en) 2018-04-12 2023-01-31 Mina Therapeutics Limited SIRT1-saRNA compositions and methods of use
WO2019226650A1 (en) 2018-05-23 2019-11-28 Modernatx, Inc. Delivery of dna
WO2020023390A1 (en) 2018-07-25 2020-01-30 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
WO2020033791A1 (en) 2018-08-09 2020-02-13 Verseau Therapeutics, Inc. Oligonucleotide compositions for targeting ccr2 and csf1r and uses thereof
US20220110966A1 (en) 2018-09-02 2022-04-14 Modernatx, Inc. Polynucleotides encoding very long-chain acyl-coa dehydrogenase for the treatment of very long-chain acyl-coa dehydrogenase deficiency
US20220243182A1 (en) 2018-09-13 2022-08-04 Modernatx, Inc. Polynucleotides encoding branched-chain alpha-ketoacid dehydrogenase complex e1-alpha, e1-beta, and e2 subunits for the treatment of maple syrup urine disease
MA53609A (en) 2018-09-13 2021-07-21 Modernatx Inc POLYNUCLEOTIDES ENCODED GLUCOSE-6-PHOSPHATASE FOR THE TREATMENT OF GLYCOGENOSIS
EP3850102A1 (en) 2018-09-14 2021-07-21 ModernaTX, Inc. Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome
US20220152225A1 (en) 2018-09-27 2022-05-19 Modernatx, Inc. Polynucleotides encoding arginase 1 for the treatment of arginase deficiency
US20220211740A1 (en) 2019-04-12 2022-07-07 Mina Therapeutics Limited Sirt1-sarna compositions and methods of use
JP2022532078A (en) 2019-05-08 2022-07-13 アストラゼネカ アクチボラグ Compositions for skin and wounds and methods of their use
CN112516328B (en) * 2019-07-26 2022-06-10 浙江大学 Boron-containing preparation with cell nucleus targeting property and preparation method and application thereof
WO2021055833A1 (en) 2019-09-19 2021-03-25 Modernatx, Inc. Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents
CN111154002B (en) * 2020-01-10 2022-11-22 上海海洋大学 Bifunctional peptide K14, gene vector and co-drug delivery system
JP2023527875A (en) 2020-06-01 2023-06-30 モダーナティエックス・インコーポレイテッド Phenylalanine hydroxylase variants and uses thereof
US20230406895A1 (en) 2020-11-13 2023-12-21 Modernatx, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
US11524023B2 (en) 2021-02-19 2022-12-13 Modernatx, Inc. Lipid nanoparticle compositions and methods of formulating the same
EP4314260A1 (en) 2021-03-24 2024-02-07 Modernatx, Inc. Lipid nanoparticles and polynucleotides encoding ornithine transcarbamylase for the treatment of ornithine transcarbamylase deficiency
WO2022204371A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding glucose-6-phosphatase and uses thereof
WO2022204390A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding phenylalanine hydroxylase and uses thereof
WO2022204380A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits and uses thereof
WO2022204369A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia
CA3214137A1 (en) 2021-03-26 2022-09-29 Mina Therapeutics Limited Tmem173 sarna compositions and methods of use
EP4355882A2 (en) 2021-06-15 2024-04-24 Modernatx, Inc. Engineered polynucleotides for cell-type or microenvironment-specific expression
WO2022271776A1 (en) 2021-06-22 2022-12-29 Modernatx, Inc. Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome
WO2023056044A1 (en) 2021-10-01 2023-04-06 Modernatx, Inc. Polynucleotides encoding relaxin for the treatment of fibrosis and/or cardiovascular disease
WO2023099884A1 (en) 2021-12-01 2023-06-08 Mina Therapeutics Limited Pax6 sarna compositions and methods of use
GB202117758D0 (en) 2021-12-09 2022-01-26 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders
WO2023170435A1 (en) 2022-03-07 2023-09-14 Mina Therapeutics Limited Il10 sarna compositions and methods of use
WO2023183909A2 (en) 2022-03-25 2023-09-28 Modernatx, Inc. Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia
WO2024026254A1 (en) 2022-07-26 2024-02-01 Modernatx, Inc. Engineered polynucleotides for temporal control of expression

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4121982A (en) * 1978-02-03 1978-10-24 American Chemical & Refining Company Incorporated Gold alloy plating bath and method
US5283185A (en) 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
JP3078107B2 (en) * 1992-04-07 2000-08-21 日本原子力研究所 Radioactive waste volume reduction method
US5393335A (en) * 1993-04-23 1995-02-28 Ppg Industries, Inc. Starch-oil sizing for glass fibers
FR2730637B1 (en) 1995-02-17 1997-03-28 Rhone Poulenc Rorer Sa PHARMACEUTICAL COMPOSITION CONTAINING NUCLEIC ACIDS, AND USES THEREOF
EP0833613A1 (en) * 1995-05-26 1998-04-08 Somatix Therapy Corporation Delivery vehicles comprising stable lipid/nucleic acid complexes
US5753262A (en) 1995-06-07 1998-05-19 Aronex Pharmaceuticals, Inc. Cationic lipid acid salt of 3beta N- (N', N'-dimethylaminoethane) - carbamoyl!cholestrol and halogenated solvent-free preliposomal lyophilate thereof
WO1997045069A1 (en) * 1996-05-29 1997-12-04 Cell Genesys, Inc. Cationic polymer/lipid nucleic acid delivery vehicles
EP0979311A1 (en) * 1997-04-30 2000-02-16 Of The University Of Minnesota Regents $i(IN VIVO) USE OF RECOMBINAGENIC OLIGONUCLEOBASES TO CORRECT GENETIC LESIONS IN HEPATOCYTES
US5955415A (en) * 1997-08-04 1999-09-21 Lever Brothers Company, Division Of Conopco, Inc. Detergent compositions containing polyethyleneimines for enhanced peroxygen bleach stability
DE19743135A1 (en) * 1997-09-30 1999-04-01 Hoechst Marion Roussel De Gmbh Biologically compatible low molecular weight polyethyleneimines
CA2329147A1 (en) * 1998-05-20 1999-11-25 Feng Liu A hepatocyte targeting polyethylene glyco-grafted poly-l-lysine polymeric gene carrier

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