ZA201008729B - Diblock copolymers and polynucleotide complexes thereof for delivery into cells - Google Patents

Diblock copolymers and polynucleotide complexes thereof for delivery into cells

Info

Publication number
ZA201008729B
ZA201008729B ZA2010/08729A ZA201008729A ZA201008729B ZA 201008729 B ZA201008729 B ZA 201008729B ZA 2010/08729 A ZA2010/08729 A ZA 2010/08729A ZA 201008729 A ZA201008729 A ZA 201008729A ZA 201008729 B ZA201008729 B ZA 201008729B
Authority
ZA
South Africa
Prior art keywords
delivery
cells
diblock copolymers
polynucleotide complexes
polynucleotide
Prior art date
Application number
ZA2010/08729A
Other languages
English (en)
Inventor
Patrick S Stayton
Allan S Hoffman
Anthony J Convertine
Danielle Benoit
Craig L Duvall
Paul Johnson
Anna Gall
Original Assignee
Univ Washington
Phaserx 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 Univ Washington, Phaserx Inc filed Critical Univ Washington
Publication of ZA201008729B publication Critical patent/ZA201008729B/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C08—ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08F—MACROMOLECULAR COMPOUNDS OBTAINED BY REACTIONS ONLY INVOLVING CARBON-TO-CARBON UNSATURATED BONDS
    • C08F293/00—Macromolecular compounds obtained by polymerisation on to a macromolecule having groups capable of inducing the formation of new polymer chains bound exclusively at one or both ends of the starting macromolecule
    • C08F293/005—Macromolecular compounds obtained by polymerisation on to a macromolecule having groups capable of inducing the formation of new polymer chains bound exclusively at one or both ends of the starting macromolecule using free radical "living" or "controlled" polymerisation, e.g. using a complexing agent
    • C—CHEMISTRY; METALLURGY
    • C08—ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08F—MACROMOLECULAR COMPOUNDS OBTAINED BY REACTIONS ONLY INVOLVING CARBON-TO-CARBON UNSATURATED BONDS
    • C08F299/00—Macromolecular compounds obtained by interreacting polymers involving only carbon-to-carbon unsaturated bond reactions, in the absence of non-macromolecular monomers
    • 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
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/32—Macromolecular compounds obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. carbomers, poly(meth)acrylates, or polyvinyl pyrrolidone
    • 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
    • A61K47/58—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 by reactions only involving carbon-to-carbon unsaturated bonds, e.g. poly[meth]acrylate, polyacrylamide, polystyrene, polyvinylpyrrolidone, polyvinylalcohol or polystyrene sulfonic acid resin
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C08—ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08F—MACROMOLECULAR COMPOUNDS OBTAINED BY REACTIONS ONLY INVOLVING CARBON-TO-CARBON UNSATURATED BONDS
    • C08F220/00—Copolymers of compounds having one or more unsaturated aliphatic radicals, each having only one carbon-to-carbon double bond, and only one being terminated by only one carboxyl radical or a salt, anhydride ester, amide, imide or nitrile thereof
    • C08F220/02—Monocarboxylic acids having less than ten carbon atoms; Derivatives thereof
    • C08F220/10—Esters
    • C—CHEMISTRY; METALLURGY
    • C08—ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
    • C08F—MACROMOLECULAR COMPOUNDS OBTAINED BY REACTIONS ONLY INVOLVING CARBON-TO-CARBON UNSATURATED BONDS
    • C08F293/00—Macromolecular compounds obtained by polymerisation on to a macromolecule having groups capable of inducing the formation of new polymer chains bound exclusively at one or both ends of the starting macromolecule
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
  • Polymers & Plastics (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Neurology (AREA)
  • Immunology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Neurosurgery (AREA)
  • Inorganic Chemistry (AREA)
  • Psychiatry (AREA)
  • Transplantation (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
ZA2010/08729A 2008-05-13 2010-12-03 Diblock copolymers and polynucleotide complexes thereof for delivery into cells ZA201008729B (en)

Applications Claiming Priority (10)

Application Number Priority Date Filing Date Title
US5291408P 2008-05-13 2008-05-13
US5290808P 2008-05-13 2008-05-13
US9129408P 2008-08-22 2008-08-22
US11204808P 2008-11-06 2008-11-06
US11205408P 2008-11-06 2008-11-06
US12076908P 2008-12-08 2008-12-08
US14077408P 2008-12-24 2008-12-24
US14077908P 2008-12-24 2008-12-24
US17137709P 2009-04-21 2009-04-21
PCT/US2009/043847 WO2009140427A2 (en) 2008-05-13 2009-05-13 Diblock copolymers and polynucleotide complexes thereof for delivery into cells

Publications (1)

Publication Number Publication Date
ZA201008729B true ZA201008729B (en) 2014-05-28

Family

ID=41319323

Family Applications (1)

Application Number Title Priority Date Filing Date
ZA2010/08729A ZA201008729B (en) 2008-05-13 2010-12-03 Diblock copolymers and polynucleotide complexes thereof for delivery into cells

Country Status (13)

Country Link
US (3) US9476063B2 (enExample)
EP (2) EP2281011B1 (enExample)
JP (2) JP5911723B2 (enExample)
KR (1) KR101661636B1 (enExample)
CN (2) CN103342789B (enExample)
AU (1) AU2009246327B8 (enExample)
BR (1) BRPI0912159B8 (enExample)
CA (3) CA3065577C (enExample)
ES (1) ES2404131T3 (enExample)
IL (1) IL209238A (enExample)
MX (1) MX2010012238A (enExample)
WO (1) WO2009140427A2 (enExample)
ZA (1) ZA201008729B (enExample)

Families Citing this family (70)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2724014C (en) 2008-05-13 2016-09-13 University Of Washington Micellic assemblies
MX2010012238A (es) 2008-05-13 2011-04-11 Univ Washington Copolimeros dibloque y sus complejos con polinucleotidos destinados a introducirlos en celulas.
WO2009140421A2 (en) * 2008-05-13 2009-11-19 University Of Washington Polymeric carrier
CA2734917A1 (en) 2008-08-22 2010-02-25 University Of Washington Heterogeneous polymeric micelles for intracellular delivery
EP2364330B1 (en) * 2008-11-06 2015-03-25 University Of Washington Multiblock copolymers
EP2352522A4 (en) 2008-11-06 2014-03-05 Univ Washington BISPECIFIC AUXILIARIES FOR INTRA-CELLULAR RELEASE
CA2745926A1 (en) 2008-12-08 2010-07-08 Phaserx, Inc. Omega-functionalized polymers, junction-functionalized block copolymers, polymer bioconjugates, and radical chain extension polymerization
WO2011060281A1 (en) * 2009-11-13 2011-05-19 University Of Washington Through Its Center For Commercialization Hydrophobic block conjugated therapeutic agents
WO2011062965A2 (en) 2009-11-18 2011-05-26 University Of Washington Through Its Center For Commercialization Targeting monomers and polymers having targeting blocks
US8349308B2 (en) 2010-03-26 2013-01-08 Mersana Therapeutics, Inc. Modified polymers for delivery of polynucleotides, method of manufacture, and methods of use thereof
CA2807552A1 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP2857499A1 (en) 2010-10-01 2015-04-08 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
CA2831613A1 (en) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
CA2842039A1 (en) 2011-08-26 2013-03-07 Arrowhead Research Corporation Poly(vinyl ester) polymers for in vivo nucleic acid delivery
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
CA2848685A1 (en) * 2011-09-13 2013-03-21 Monsanto Technology Llc Methods and compositions for weed control comprising topical application of a glutamine synthetase polynucleotide
PT3682905T (pt) 2011-10-03 2022-04-07 Modernatx Inc Nucleósidos, nucleótidos e ácidos nucleicos modificados e respetivas utilizações
ES2923757T3 (es) 2011-12-16 2022-09-30 Modernatx Inc Composiciones de ARNm modificado
WO2013151664A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
MX2014001965A (es) * 2012-04-18 2014-03-31 Arrowhead Res Corp Polimeros de poli(acrilato) para suministro de acido nucleico in vivo.
JP6195171B2 (ja) 2012-04-27 2017-09-13 国立大学法人 東京大学 核酸デリバリー用ユニット構造型医薬組成物
CN104619843B (zh) 2012-05-24 2020-03-06 A.B.种子有限公司 用于使基因表达沉默的组合物和方法
WO2014047524A1 (en) 2012-09-21 2014-03-27 Vanderbilt University Poly(thioketal-urethane) scaffolds and methods of use
US10046086B2 (en) 2012-09-21 2018-08-14 Vanderbilt University Poly(thioketal-urethane) scaffolds and methods of use
PT2922554T (pt) 2012-11-26 2022-06-28 Modernatx Inc Arn modificado nas porções terminais
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
AU2014296278C1 (en) 2013-07-30 2023-02-02 Genevant Sciences Gmbh Block copolymers and their conjugates or complexes with oligonucleotides
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
KR20160067219A (ko) 2013-10-03 2016-06-13 모더나 세라퓨틱스, 인코포레이티드 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드
WO2015088990A1 (en) 2013-12-09 2015-06-18 Durect Corporation Pharmaceutically active agent complexes, polymer complexes, and compositions and methods involving the same
BR112016020688B1 (pt) 2014-03-09 2024-01-30 The Trustees Of The University Of Pennsylvania Vetores virais recombinantes, vírus adenoassociado recombinante, composição farmacêutica, bem como uso dos mesmos
EP3125946B1 (en) * 2014-03-28 2022-12-07 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules
US9993563B2 (en) * 2014-03-28 2018-06-12 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules
US11318206B2 (en) 2014-03-28 2022-05-03 Aposense Ltd Compounds and methods for trans-membrane delivery of molecules
US10695288B2 (en) 2014-11-19 2020-06-30 Vanderbilt University Reactive oxygen species (ROS)-responsive compositions and methods thereof
EP3247328B1 (en) * 2015-01-21 2026-01-07 Genevant Sciences Gmbh Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
JP6851319B2 (ja) 2015-04-27 2021-03-31 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ヒト疾患のCRISPR/Cas9媒介性の修正のためのデュアルAAVベクター系
ES3009026T3 (en) 2015-07-22 2025-03-25 Univ Duke High-throughput screening of regulatory element function with epigenome editing technologies
KR20240132120A (ko) 2015-08-25 2024-09-02 듀크 유니버시티 Rna-가이드된 엔도뉴클레아제를 이용하는 게놈 조작에서 특이성을 개선하는 조성물 및 방법
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
US10947338B2 (en) * 2016-03-29 2021-03-16 Lg Chem, Ltd. Block polymer and polymer electrolyte membrane comprising same
EP3443081A4 (en) 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Cpf1に基づくゲノム編集の治療適用
WO2018045317A1 (en) 2016-09-02 2018-03-08 Dicerna Pharmaceuticals, Inc. 4'-phosphate analogs and oligonucleotides comprising the same
WO2018126084A1 (en) 2016-12-30 2018-07-05 Phaserx, Inc. Branched peg molecules and related compositions and methods
CN110402152B (zh) 2017-01-09 2024-03-19 艾博森斯有限公司 用于分子的跨膜递送的化合物和方法
MA49421A (fr) 2017-06-15 2020-04-22 Modernatx Inc Formulations d'arn
MX2020002348A (es) 2017-08-31 2020-10-08 Modernatx Inc Métodos de elaboración de nanopartículas lipídicas.
EP3677268A4 (en) 2017-08-31 2021-04-21 The University Of Tokyo UNIFORM POLYIONAL COMPLEX LOADED WITH NUCLEIC ACID
EP3735276A4 (en) 2018-01-01 2021-10-13 Aposense Ltd. COMPOUNDS AND METHODS FOR THE TRANSMEMBRANARY ADMINISTRATION OF MOLECULES
US12090235B2 (en) 2018-09-20 2024-09-17 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
US12508233B2 (en) 2018-10-30 2025-12-30 Vanderbilt University Graft copolymers, methods of forming graft copolymers, and methods of use thereof
JP7389055B2 (ja) * 2019-01-09 2023-11-29 デンカ株式会社 トリブロック共重合体及びトリブロック共重合体の製造方法
CN113939282A (zh) 2019-01-31 2022-01-14 摩登纳特斯有限公司 制备脂质纳米颗粒的方法
WO2020205473A1 (en) 2019-03-29 2020-10-08 Decerna Pharmaceuticals, Inc. Compositions and methods for the treatment of kras associated diseases or disorders
TWI887234B (zh) 2019-04-14 2025-06-21 美國公爵大學 治療杜興氏(duchenne)肌肉失養症之大型突變熱點之aav載體介導之刪除
WO2020226960A1 (en) 2019-05-03 2020-11-12 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules with shortened sense strands
TW202140043A (zh) 2020-01-15 2021-11-01 美商戴瑟納製藥股份有限公司 4’-o-亞甲基膦酸酯核酸及其類似物
WO2021222694A1 (en) * 2020-04-30 2021-11-04 Dow Global Technologies Llc Process for preparing olefin-acrylate block copolymers by raft polyerization
JP2023537499A (ja) 2020-08-04 2023-09-01 ディセルナ ファーマシューティカルズ インコーポレイテッド オリゴヌクレオチドの全身送達
CN113087017A (zh) * 2021-04-06 2021-07-09 中国科学院理化技术研究所 一种偏钒酸钠纳米球及其制备方法和应用
CA3260237A1 (en) 2022-06-24 2023-12-28 Tune Therapeutics, Inc. Compositions, systems and methods for reducing low-density lipoprotein by targeted gene repression
AU2024349841A1 (en) 2023-09-29 2026-04-09 Battelle Memorial Institute Polymer nanoparticle compositions for non-viral gene delivery
WO2025072751A1 (en) 2023-09-29 2025-04-03 Battelle Memorial Institute Polymer nanoparticle compositions for in vivo expression of polypeptides
WO2025250979A1 (en) 2024-05-31 2025-12-04 Battelle Memorial Institute Polymer nanoparticle compositions for non-viral gene delivery
US20260090998A1 (en) 2024-10-02 2026-04-02 Battelle Memorial Institute Methods of using polymer nanoparticle compositions for treating a disease

Family Cites Families (71)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4699784A (en) * 1986-02-25 1987-10-13 Center For Molecular Medicine & Immunology Tumoricidal methotrexate-antibody conjugate
US5057313A (en) * 1986-02-25 1991-10-15 The Center For Molecular Medicine And Immunology Diagnostic and therapeutic antibody conjugates
US4877603A (en) 1987-12-18 1989-10-31 The Procter & Gamble Company Oral compositions
US6359054B1 (en) * 1994-11-18 2002-03-19 Supratek Pharma Inc. Polynucleotide compositions for intramuscular administration
FR2767829B1 (fr) 1997-09-01 1999-12-03 Georges Sturtz Composes phosphonates ou gem-bisphosphonates acryliques ou methacryliques
US6410057B1 (en) * 1997-12-12 2002-06-25 Samyang Corporation Biodegradable mixed polymeric micelles for drug delivery
CN1281355A (zh) 1997-12-12 2001-01-24 三阳株式会社 用于基因递送的生物可降解性混合聚合胶束
US6383811B2 (en) * 1997-12-30 2002-05-07 Mirus Corporation Polyampholytes for delivering polyions to a cell
ES2356342T3 (es) * 1998-01-05 2011-04-07 The University Of Washington Mejora de transporte utilizando agentes de alteración de membrana.
US6306994B1 (en) * 1999-05-14 2001-10-23 E. I. Du Pont De Nemours And Company Inks with enhanced substrate binding characteristics
US7098032B2 (en) * 2001-01-02 2006-08-29 Mirus Bio Corporation Compositions and methods for drug delivery using pH sensitive molecules
US7033607B2 (en) * 1999-12-31 2006-04-25 Mirus Bio Corporation pH-titratable polyampholytes for delivering polyions to a cell
AU2764801A (en) * 2000-01-07 2001-07-24 University Of Washington Enhanced transport of agents using membrane disruptive agents
US20030134420A1 (en) * 2000-02-18 2003-07-17 Lollo Charles Peter Methods and compositions for gene delivery
AU2001276627A1 (en) 2000-05-17 2001-11-26 Labopharm Inc. Drug containing polymeric micelles
SE517421C2 (sv) * 2000-10-06 2002-06-04 Bioglan Ab Mikropartiklar, lämpade för parenteral administration, väsentligen bestående av stärkelse med minst 85 % amylopektin och med reducerad molekylvikt, samt framställning därav
EP1325046B1 (en) 2000-10-06 2007-06-27 Biocompatibles UK Limited Zwitterionic polymers
AU2002367709A1 (en) 2001-04-26 2003-10-27 Nanosphere, Inc. Oligonucleotide-modified romp polymers and co-polymers
US6780428B2 (en) * 2001-06-08 2004-08-24 Labopharm, Inc. Unimolecular polymeric micelles with an ionizable inner core
US7094810B2 (en) * 2001-06-08 2006-08-22 Labopharm, Inc. pH-sensitive block copolymers for pharmaceutical compositions
US6939564B2 (en) * 2001-06-08 2005-09-06 Labopharm, Inc. Water-soluble stabilized self-assembled polyelectrolytes
EP1463518A2 (en) * 2001-12-28 2004-10-06 Supratek Pharma, Inc. Pharmaceutical compositions comprising polyanionic polymers and amphiphilic block copolymers and methods of use thereof to improve gene expression
AU2003214387A1 (en) * 2002-03-07 2003-09-16 Biocompatibles Uk Limited Drug carriers comprising amphiphilic block copolymers
US7229973B2 (en) 2002-05-19 2007-06-12 You Han Bae pH-sensitive polymeric micelles for drug delivery
EP1400555A1 (en) 2002-09-17 2004-03-24 Kawamura Institute Of Chemical Research Water-soluble block copolymer and production method therefor
WO2004044023A1 (fr) * 2002-11-07 2004-05-27 Rhodia Chimie Copolymere a structure controlee presentant une partie amphotere ou zwitterionique.
AU2003293082A1 (en) * 2002-11-27 2004-06-23 Tufts University Antioxidant-functionalized polymers
WO2004060977A1 (en) * 2002-12-30 2004-07-22 Nektar Therapeutics Al, Corporation Multi-arm polypeptide-poly(ethylene glycol) block copolymers as drug delivery vehicles
US7871818B2 (en) * 2003-01-31 2011-01-18 Roche Madison Inc. Membrane active polymers
US7217776B1 (en) * 2003-02-14 2007-05-15 Iowa State University Research Foundation pH-sensitive methacrylic copolymer gels and the production thereof
US20040162235A1 (en) * 2003-02-18 2004-08-19 Trubetskoy Vladimir S. Delivery of siRNA to cells using polyampholytes
JP4535229B2 (ja) * 2003-05-08 2010-09-01 国立大学法人 東京大学 ポリエチレングリコール−ポリカチオンブロック共重合体
FR2868784B1 (fr) * 2004-04-09 2006-05-26 Oreal Copolymere sequence, composition le comprenant et procede de traitement cosmetique
US7632905B2 (en) 2004-04-09 2009-12-15 L'oreal S.A. Block copolymer, composition comprising it and cosmetic treatment process
WO2005108614A2 (en) 2004-04-07 2005-11-17 Northwestern University Reversible and chemically programmable micelle assembly with dna block-copolymer amphiphiles
US8821859B2 (en) * 2004-05-19 2014-09-02 Agency For Science, Technology And Research Methods and articles for the delivery of therapeutic agents
GB0418123D0 (en) * 2004-08-13 2004-09-15 Asahi Chemical Ind Polymers useful as medical materials
US20060134221A1 (en) * 2004-12-03 2006-06-22 Vical Incorporated Methods for producing block copolymer/amphiphilic particles
WO2006071769A1 (en) * 2004-12-28 2006-07-06 The Trustees Of The University Of Pennsylvania Controlled release from block co-polymer worm micelles
US20060171980A1 (en) * 2005-02-01 2006-08-03 Helmus Michael N Implantable or insertable medical devices having optimal surface energy
CN101155844A (zh) * 2005-03-31 2008-04-02 阿帕医药有限公司 聚乙二醇-聚缩醛和聚乙二醇-聚缩醛-聚原酸酯接枝共聚物和药物组合物
US20080171067A1 (en) * 2007-01-17 2008-07-17 Immunomedics, Inc. Polymeric Carriers of Therapeutic Agents and Recognition Moieties for Antibody-Based Targeting of Disease Sites
CN101203250B (zh) * 2005-04-15 2013-03-20 界面生物公司 经由非共价相互作用递送生物活性剂的方法、组合物及成形物件
US9139850B2 (en) * 2005-05-19 2015-09-22 L'oreal Vectorization of dsRNA by cationic particles and topical use
WO2007008300A2 (en) 2005-05-31 2007-01-18 ECOLE POLYTECHNIQUE FéDéRALE DE LAUSANNE Triblock copolymers for cytoplasmic delivery of gene-based drugs
FR2890859B1 (fr) * 2005-09-21 2012-12-21 Oreal Oligonucleotide d'arn double brin inhibant l'expression de la tyrosinase
US7718193B2 (en) * 2006-03-16 2010-05-18 University Of Washington Temperature- and pH-responsive polymer compositions
US8367113B2 (en) * 2006-05-15 2013-02-05 Massachusetts Institute Of Technology Polymers for functional particles
WO2008004978A1 (en) 2006-07-05 2008-01-10 Agency For Science, Technology And Research Micelles for drug delivery
CN101500548A (zh) 2006-08-18 2009-08-05 弗·哈夫曼-拉罗切有限公司 用于体内递送多核苷酸的多缀合物
WO2008085556A2 (en) 2006-09-12 2008-07-17 University Of South Florida Surfactant-free nanoparticles for drug delivery
US20080081075A1 (en) * 2006-10-02 2008-04-03 National Tsing Hua University Multifunctional mixed micelle of graft and block copolymers and preparation thereof
WO2008071009A1 (en) 2006-12-15 2008-06-19 The Governors Of The University Of Alberta Novel ligand guided block copolymers for targeted drug delivery
US20080311045A1 (en) * 2007-06-06 2008-12-18 Biovaluation & Analysis, Inc. Polymersomes for Use in Acoustically Mediated Intracellular Drug Delivery in vivo
US8747830B2 (en) 2007-06-08 2014-06-10 Queensland University Of Technology Wound repair composition and method
US20080312134A1 (en) 2007-06-11 2008-12-18 Thomas Hirt Vesicles for active macromolecule delivery
WO2009009025A1 (en) 2007-07-06 2009-01-15 Northeastern University Mixed micelles including amphipathic conjugates of rna agents, and uses thereof
US20090036625A1 (en) * 2007-08-01 2009-02-05 Chung Yuan Christian University Amphiphilic Polymer, Method for Forming the Same and Application thereof
EP2025348A1 (en) 2007-08-13 2009-02-18 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Targeted block copolymer micelles
WO2009140423A2 (en) 2008-05-13 2009-11-19 University Of Washington Targeted polymer bioconjugates
MX2010012237A (es) 2008-05-13 2011-04-28 Univ Washington Micelas para el suministro intracelular de agentes terapeuticos.
CA2724014C (en) * 2008-05-13 2016-09-13 University Of Washington Micellic assemblies
WO2009140421A2 (en) 2008-05-13 2009-11-19 University Of Washington Polymeric carrier
MX2010012238A (es) * 2008-05-13 2011-04-11 Univ Washington Copolimeros dibloque y sus complejos con polinucleotidos destinados a introducirlos en celulas.
CA2734917A1 (en) 2008-08-22 2010-02-25 University Of Washington Heterogeneous polymeric micelles for intracellular delivery
DK2180004T3 (da) 2008-10-22 2011-09-05 Clariant Finance Bvi Ltd Olefinisk umættede phosphonatforbindelser, polymere fremstillet ud fra disse og deres anvendelse
US20110281934A1 (en) * 2008-11-06 2011-11-17 Phaserx, Inc. Micelles of hydrophilically shielded membrane-destabilizing copolymers
EP2364330B1 (en) * 2008-11-06 2015-03-25 University Of Washington Multiblock copolymers
EP2352522A4 (en) * 2008-11-06 2014-03-05 Univ Washington BISPECIFIC AUXILIARIES FOR INTRA-CELLULAR RELEASE
US20100150952A1 (en) * 2008-11-07 2010-06-17 University Of Washington pH-RESPONSIVE POLYMER CARRIER COMPOSITIONS FOR CYTOSOLIC PROTEIN DELIVERY
CA2745926A1 (en) 2008-12-08 2010-07-08 Phaserx, Inc. Omega-functionalized polymers, junction-functionalized block copolymers, polymer bioconjugates, and radical chain extension polymerization

Also Published As

Publication number Publication date
US9862792B2 (en) 2018-01-09
BRPI0912159A2 (pt) 2015-10-13
EP2281011A4 (en) 2011-10-26
EP2281011A2 (en) 2011-02-09
AU2009246327A1 (en) 2009-11-19
US20180171056A1 (en) 2018-06-21
KR20110033124A (ko) 2011-03-30
US9476063B2 (en) 2016-10-25
CA2724105A1 (en) 2009-11-19
CN103342789A (zh) 2013-10-09
EP2620161A1 (en) 2013-07-31
KR101661636B1 (ko) 2016-09-30
JP6239565B2 (ja) 2017-11-29
CA3065577C (en) 2022-05-31
WO2009140427A3 (en) 2010-03-04
AU2009246327B8 (en) 2013-10-03
US20170096517A1 (en) 2017-04-06
CA2724105C (en) 2017-09-05
US20110143434A1 (en) 2011-06-16
CA3065577A1 (en) 2009-11-19
CN102083878A (zh) 2011-06-01
JP2011521054A (ja) 2011-07-21
AU2009246327A8 (en) 2011-01-13
CN103342789B (zh) 2016-11-23
IL209238A0 (en) 2011-01-31
JP5911723B2 (ja) 2016-04-27
JP2016053169A (ja) 2016-04-14
BRPI0912159B1 (pt) 2019-11-05
CN102083878B (zh) 2013-07-10
BRPI0912159B8 (pt) 2021-05-25
CA2972694A1 (en) 2009-11-19
MX2010012238A (es) 2011-04-11
ES2404131T3 (es) 2013-05-24
CA2972694C (en) 2020-02-11
IL209238A (en) 2014-03-31
EP2281011B1 (en) 2013-02-27
AU2009246327B2 (en) 2013-09-12
WO2009140427A2 (en) 2009-11-19

Similar Documents

Publication Publication Date Title
IL209238A0 (en) Diblock copolymers and polynucleotide complexes thereof for delivery into cells
ZA201103289B (en) Multiblock copolymers
ZA201103515B (en) Composition comprising polymer and enzyme
ZA201009009B (en) Aryl gpr119 agonists and uses thereof
GB2488075B (en) Polymer composition, swellable composition comprising the polymer composition, and articles including the swellable composition
AP3856A (en) Improvements in feedwells.
EP2128196A4 (en) POLYMERIC COMPOSITION AND APPLICATION THEREOF
EP2162493A4 (en) STABLE AND COMPATIBLE POLYMER MIXTURES
IL210506A0 (en) Complement antagonists and uses thereof
GB0902052D0 (en) Polymers,composition and use
IL207569A0 (en) Compositions and methods for drug delivery
GB0909652D0 (en) Improvements in security substrates
EG25972A (en) Polymer composition
GB0808058D0 (en) Polymer composition
IL195764A0 (en) Compositions and methods for drug delivery
SI2260142T1 (sl) Izboljšave v varnostnih substratih
GB0918601D0 (en) Decahydro-1H-Indenoquinolinone and decahydro-3H-cyclopenaphenanthridinone CYP17 inhibitors
EP2343759A4 (en) BATTERY COMPONENT AND BATTERY
PL2276347T3 (pl) Toltrazuril o ulepszonych właściwościach rozpuszczania
IL205237A0 (en) Nucleic acid complex and nucleic acid delivery composition
EP2498606A4 (en) TIVOZANIB AND TEMSIROLIMUS IN COMBINATION
PL2467410T3 (pl) Statystycznie rozgałęzione kopolimery, ich wytwarzanie i zastosowanie jako środka poprawiającego rozlewność w materiałach powłokowych
EP2185685A4 (en) NEW PROCESSES AND NEW CELL LINES
EP2247427A4 (en) RECEIVED GRAPHICS FILMS AND MANUFACTURING METHOD THEREFOR
IL212459A0 (en) Multiblock copolymers