JP5782602B2 - ポリアミン誘導体 - Google Patents

ポリアミン誘導体 Download PDF

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Publication number
JP5782602B2
JP5782602B2 JP2012500998A JP2012500998A JP5782602B2 JP 5782602 B2 JP5782602 B2 JP 5782602B2 JP 2012500998 A JP2012500998 A JP 2012500998A JP 2012500998 A JP2012500998 A JP 2012500998A JP 5782602 B2 JP5782602 B2 JP 5782602B2
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Japan
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alkylene
sirna
compound
formula
provides
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JP2012500998A
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Japanese (ja)
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JP2012521358A5 (enExample
JP2012521358A (ja
Inventor
スロボドキン、グレゴリー
コンゴ、リチャード
マター、マジェド
フェウェル、ジェイソン
アンワー、クルシェード
スパークス、ブライアン、ジェフリー
Original Assignee
シーエルエスエヌ ラボラトリーズ、インコーポレイテッド
シーエルエスエヌ ラボラトリーズ、インコーポレイテッド
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    • 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
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • 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
    • A61K47/54—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 compound
    • A61K47/542—Carboxylic acids, e.g. a fatty acid or an amino acid
    • 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—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • A61P31/12—Antivirals
    • 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
    • A61P35/00—Antineoplastic agents
    • A61P35/04—Antineoplastic agents specific for metastasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C233/00—Carboxylic acid amides
    • C07C233/01—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms
    • C07C233/34—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by amino groups
    • C07C233/35—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by amino groups with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom
    • C07C233/38—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by amino groups with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom having the carbon atom of the carboxamide group bound to a carbon atom of an acyclic unsaturated carbon skeleton
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C235/00—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms
    • C07C235/02—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to acyclic carbon atoms and singly-bound oxygen atoms bound to the same carbon skeleton
    • C07C235/04—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to acyclic carbon atoms and singly-bound oxygen atoms bound to the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C235/10—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to acyclic carbon atoms and singly-bound oxygen atoms bound to the same carbon skeleton the carbon skeleton being acyclic and saturated having the nitrogen atom of at least one of the carboxamide groups bound to an acyclic carbon atom of a hydrocarbon radical substituted by nitrogen atoms not being part of nitro or nitroso groups
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C235/00—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms
    • C07C235/42—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to carbon atoms of six-membered aromatic rings and singly-bound oxygen atoms bound to the same carbon skeleton
    • C07C235/44—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to carbon atoms of six-membered aromatic rings and singly-bound oxygen atoms bound to the same carbon skeleton with carbon atoms of carboxamide groups and singly-bound oxygen atoms bound to carbon atoms of the same non-condensed six-membered aromatic ring
    • C07C235/58—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to carbon atoms of six-membered aromatic rings and singly-bound oxygen atoms bound to the same carbon skeleton with carbon atoms of carboxamide groups and singly-bound oxygen atoms bound to carbon atoms of the same non-condensed six-membered aromatic ring with carbon atoms of carboxamide groups and singly-bound oxygen atoms, bound in ortho-position to carbon atoms of the same non-condensed six-membered aromatic ring
    • C07C235/60—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to carbon atoms of six-membered aromatic rings and singly-bound oxygen atoms bound to the same carbon skeleton with carbon atoms of carboxamide groups and singly-bound oxygen atoms bound to carbon atoms of the same non-condensed six-membered aromatic ring with carbon atoms of carboxamide groups and singly-bound oxygen atoms, bound in ortho-position to carbon atoms of the same non-condensed six-membered aromatic ring having the nitrogen atoms of the carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D475/00—Heterocyclic compounds containing pteridine ring systems
    • C07D475/02—Heterocyclic compounds containing pteridine ring systems with an oxygen atom directly attached in position 4
    • C07D475/04—Heterocyclic compounds containing pteridine ring systems with an oxygen atom directly attached in position 4 with a nitrogen atom directly attached in position 2
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/02—Acyclic radicals, not substituted by cyclic structures
    • C07H15/04—Acyclic radicals, not substituted by cyclic structures attached to an oxygen atom of the saccharide radical
    • C07H15/10—Acyclic radicals, not substituted by cyclic structures attached to an oxygen atom of the saccharide radical containing unsaturated carbon-to-carbon bonds
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/23—Luteinising hormone-releasing hormone [LHRH]; Related peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/64—Cyclic peptides containing only normal peptide links

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Genetics & Genomics (AREA)
  • Dispersion Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Biotechnology (AREA)
  • Oncology (AREA)
  • Endocrinology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Ophthalmology & Optometry (AREA)
  • Urology & Nephrology (AREA)
  • Pulmonology (AREA)
  • Communicable Diseases (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Polyethers (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Compositions Of Macromolecular Compounds (AREA)
  • Macromolecular Compounds Obtained By Forming Nitrogen-Containing Linkages In General (AREA)
JP2012500998A 2009-03-20 2010-03-19 ポリアミン誘導体 Active JP5782602B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US16182809P 2009-03-20 2009-03-20
US61/161,828 2009-03-20
PCT/US2010/028000 WO2010108108A2 (en) 2009-03-20 2010-03-19 Polyamine derivatives

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2015018143A Division JP6190401B2 (ja) 2009-03-20 2015-02-02 ポリアミン誘導体

Publications (3)

Publication Number Publication Date
JP2012521358A JP2012521358A (ja) 2012-09-13
JP2012521358A5 JP2012521358A5 (enExample) 2014-10-16
JP5782602B2 true JP5782602B2 (ja) 2015-09-24

Family

ID=42359538

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JP2012500998A Active JP5782602B2 (ja) 2009-03-20 2010-03-19 ポリアミン誘導体
JP2015018143A Active JP6190401B2 (ja) 2009-03-20 2015-02-02 ポリアミン誘導体

Family Applications After (1)

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JP2015018143A Active JP6190401B2 (ja) 2009-03-20 2015-02-02 ポリアミン誘導体

Country Status (10)

Country Link
US (4) US8460696B2 (enExample)
EP (1) EP2414322B1 (enExample)
JP (2) JP5782602B2 (enExample)
KR (2) KR101800833B1 (enExample)
CN (2) CN104922676B (enExample)
AU (1) AU2010226434A1 (enExample)
CA (1) CA2755963C (enExample)
ES (1) ES2931174T3 (enExample)
PT (1) PT2414322T (enExample)
WO (1) WO2010108108A2 (enExample)

Families Citing this family (100)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2941152B1 (fr) * 2009-01-20 2013-10-18 Centre Nat Rech Scient Vecteurs comprenant une macromolecule anionique et un lipide cationique pour l'administration de petits acides nucleiques.
US8460696B2 (en) 2009-03-20 2013-06-11 Egen, Inc. Polyamine derivatives
US8822663B2 (en) 2010-08-06 2014-09-02 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
CA2821992A1 (en) 2010-10-01 2012-04-05 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
CN113214102A (zh) 2010-11-15 2021-08-06 生命技术公司 含胺的转染试剂及其制备和使用方法
US20120202890A1 (en) * 2011-02-08 2012-08-09 Nian Wu Polymer-carbohydrate-lipid conjugates
DE12722942T1 (de) 2011-03-31 2021-09-30 Modernatx, Inc. Freisetzung und formulierung von manipulierten nukleinsäuren
EP2696678B1 (en) 2011-04-15 2019-11-20 Molecular Transfer, Inc. Agents for improved delivery of nucleic acids to eukaryotic cells
US8883177B2 (en) * 2011-06-28 2014-11-11 Nian Wu Pharmaceutical compositions for parenteral administration
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
HRP20220250T1 (hr) 2011-10-03 2022-04-29 Modernatx, Inc. Modificirani nukleozidi, nukleotidi i nukleinske kiseline, te njihove uporabe
SI2791160T1 (sl) 2011-12-16 2022-07-29 Modernatx, Inc. Sestave modificirane MRNA
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
AU2013243952A1 (en) 2012-04-02 2014-10-30 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
HK1206612A1 (en) 2012-04-02 2016-01-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of secreted proteins
SMT202200337T1 (it) 2012-11-26 2022-09-14 Modernatx Inc Rna modificato al livello del terminale
AU2013374345A1 (en) 2013-01-17 2015-08-06 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
EP2968391A1 (en) 2013-03-13 2016-01-20 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
EP2971010B1 (en) 2013-03-14 2020-06-10 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
JP6358661B2 (ja) * 2013-03-19 2018-07-18 公立大学法人首都大学東京 界面活性剤様化合物
BR112016000160B1 (pt) 2013-07-05 2021-06-08 Bioneer Corporation estrutura de oligonucleotídeo tipo nanopartícula melhorada tendo alta eficiência e método para a preparação da mesma
MX2016000021A (es) 2013-07-05 2017-04-06 Bioneer Corp Sirna virus especifico del dengue, estructura arn oligo helicoidal, y composicion para la supresion de proliferacion del virus del dengue que comprende la estructura arn.
MX382210B (es) * 2013-07-05 2025-03-13 Bioneer Corp (arnip) gen especifico relacionado con las enfermedades respiratorias, (arnip) que contiene la estructura arn oligo helicoidal, composicion que contiene la misma para prevencion o tratamiento de las enfermedades respiratorias.
HUE056760T2 (hu) 2013-07-11 2022-03-28 Modernatx Inc A CRISPR-hez kapcsolódó fehérjéket és a szintetikus SGRNS-ket kódoló szintetikus polinukleotidokat tartalmazó készítmények és felhasználási módjaik
AU2014315287A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
EP3041938A1 (en) 2013-09-03 2016-07-13 Moderna Therapeutics, Inc. Circular polynucleotides
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
US10323076B2 (en) 2013-10-03 2019-06-18 Modernatx, Inc. Polynucleotides encoding low density lipoprotein receptor
KR101647178B1 (ko) * 2013-11-28 2016-08-23 충남대학교산학협력단 효소 절단성 링커 또는 올리고 라이신을 포함하는 폴리에틸렌글리콜-리피드를 이용한 안정화된 플라스미드-지질 입자
WO2015095351A1 (en) 2013-12-19 2015-06-25 Novartis Ag LEPTIN mRNA COMPOSITIONS AND FORMULATIONS
CN112159807A (zh) 2014-04-04 2021-01-01 柏业公司 新颖的双链寡rna和包含它的用于预防或治疗纤维化或呼吸系统疾病的药物组合物
SG10201912038TA (en) 2014-04-23 2020-02-27 Modernatx Inc Nucleic acid vaccines
KR102511554B1 (ko) * 2014-06-24 2023-03-16 샤이어 휴먼 지네틱 테라피즈 인크. 핵산의 전달용 입체화학적으로 풍부한 조성물
WO2016011226A1 (en) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Chimeric polynucleotides
WO2016014846A1 (en) 2014-07-23 2016-01-28 Moderna Therapeutics, Inc. Modified polynucleotides for the production of intrabodies
US20180008727A1 (en) * 2015-01-30 2018-01-11 The Regents Of The University Of California Spinal subpial gene delivery system
CA2998810A1 (en) 2015-09-17 2017-03-23 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
ES2924407T3 (es) 2015-12-10 2022-10-06 Modernatx Inc Composiciones y procedimientos para el suministro de agentes terapéuticos
SMT202200252T1 (it) 2015-12-22 2022-07-21 Modernatx Inc Composti e composizioni per il rilascio intracellulare di agenti
US11583504B2 (en) 2016-11-08 2023-02-21 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
AU2018234828A1 (en) 2017-03-15 2019-09-19 Modernatx, Inc. Lipid nanoparticle formulation
EP3596041B1 (en) 2017-03-15 2022-11-02 ModernaTX, Inc. Compound and compositions for intracellular delivery of therapeutic agents
AU2018234814B2 (en) 2017-03-15 2022-06-30 Modernatx, Inc. Crystal forms of amino lipids
CA3063723A1 (en) 2017-05-18 2018-11-22 Modernatx, Inc. Polynucleotides encoding tethered interleukin-12 (il12) polypeptides and uses thereof
EP3625345B1 (en) 2017-05-18 2023-05-24 ModernaTX, Inc. Modified messenger rna comprising functional rna elements
WO2018232120A1 (en) 2017-06-14 2018-12-20 Modernatx, Inc. Compounds and compositions for intracellular delivery of agents
MA49395A (fr) 2017-06-14 2020-04-22 Modernatx Inc Polynucléotides codant pour le facteur viii de coagulation
WO2018232357A1 (en) 2017-06-15 2018-12-20 Modernatx, Inc. Rna formulations
US11744801B2 (en) 2017-08-31 2023-09-05 Modernatx, Inc. Methods of making lipid nanoparticles
US20200208152A1 (en) 2017-09-08 2020-07-02 Mina Therapeutics Limited Stabilized 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
WO2019104160A2 (en) 2017-11-22 2019-05-31 Modernatx, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
JP7423522B2 (ja) 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 尿素サイクル異常症の治療のためのオルニチントランスカルバミラーゼをコードするポリヌクレオチド
US11802146B2 (en) 2018-01-05 2023-10-31 Modernatx, Inc. Polynucleotides encoding anti-chikungunya virus antibodies
KR102056144B1 (ko) 2018-03-08 2019-12-16 주식회사 파마리서치프로덕트 Dna 단편 혼합물이 고농도로 함유된 유동성을 갖는 액제 조성물 및 이의 제조방법
US11566246B2 (en) 2018-04-12 2023-01-31 Mina Therapeutics Limited SIRT1-saRNA compositions and methods of use
MA52709A (fr) 2018-05-23 2021-03-31 Modernatx Inc Administration d'adn
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
EP3833762A4 (en) 2018-08-09 2022-09-28 Verseau Therapeutics, Inc. Oligonucleotide compositions for targeting ccr2 and csf1r and uses thereof
WO2020047201A1 (en) 2018-09-02 2020-03-05 Modernatx, Inc. Polynucleotides encoding very long-chain acyl-coa dehydrogenase for the treatment of very long-chain acyl-coa dehydrogenase deficiency
MA53608A (fr) 2018-09-13 2021-07-21 Modernatx Inc Polynucléotides codant pour les sous-unités e1-alpha, e1-beta et e2 du complexe alpha-cétoacide déshydrogénase à chaîne ramifiée pour le traitement de la leucinose
MA53609A (fr) 2018-09-13 2021-07-21 Modernatx Inc Polynucléotides codant la glucose-6-phosphatase pour le traitement de la glycogénose
US20220401584A1 (en) 2018-09-14 2022-12-22 Modernatx, Inc. Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome
JP7640452B2 (ja) 2018-09-19 2025-03-05 モデルナティエックス インコーポレイテッド 高純度ｐｅｇ脂質及びそれらの使用
WO2020061367A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
WO2020061284A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. Peg lipids and uses thereof
WO2020061457A1 (en) 2018-09-20 2020-03-26 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
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
EP3965797A1 (en) 2019-05-08 2022-03-16 AstraZeneca AB Compositions for skin and wounds and methods of use thereof
US11066355B2 (en) 2019-09-19 2021-07-20 Modernatx, Inc. Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents
JP2023527875A (ja) 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 フェニルアラニンヒドロキシラーゼバリアント及びその使用
US20230365966A1 (en) * 2020-10-09 2023-11-16 Napajen Pharma, Inc. Homogeneous beta-1,3-glucan/nucleic acid complexes and use thereof
CA3199784A1 (en) 2020-11-13 2022-05-19 Modernatx, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
US11524023B2 (en) 2021-02-19 2022-12-13 Modernatx, Inc. Lipid nanoparticle compositions and methods of formulating the same
WO2022204371A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding glucose-6-phosphatase 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
WO2022204370A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles and polynucleotides encoding ornithine transcarbamylase for the treatment of ornithine transcarbamylase deficiency
WO2022204390A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding phenylalanine hydroxylase and uses thereof
US20240226025A1 (en) 2021-03-24 2024-07-11 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia
JP2024511092A (ja) 2021-03-26 2024-03-12 ミナ セラピューティクス リミテッド ＴＭＥＭ１７３ｓａＲＮＡ組成物及び使用方法
US20240384277A1 (en) 2021-06-15 2024-11-21 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
EP4408871A1 (en) 2021-10-01 2024-08-07 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
KR20230096311A (ko) * 2021-12-23 2023-06-30 주식회사 삼양홀딩스 약물의 폐 전달용 나노입자 조성물
KR102852074B1 (ko) * 2021-12-23 2025-08-28 주식회사 삼양홀딩스 약물 전달용 지질 및 이를 포함하는 나노입자, 및 이 나노입자를 포함하는 약물 전달용 조성물
WO2023170435A1 (en) 2022-03-07 2023-09-14 Mina Therapeutics Limited Il10 sarna compositions and methods of use
US20250221931A1 (en) 2022-03-25 2025-07-10 Modernatx, Inc. Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia
TW202412818A (zh) 2022-07-26 2024-04-01 美商現代公司 用於暫時控制表現之經工程化多核苷酸
WO2024044728A1 (en) * 2022-08-26 2024-02-29 Renagade Therapeutics Management Inc. Pegylated lipid compounds and methods of use thereof
EP4634388A1 (en) 2022-12-14 2025-10-22 Providence Therapeutics Holdings Inc. Compositions and methods for infectious diseases
WO2024134199A1 (en) 2022-12-22 2024-06-27 Mina Therapeutics Limited Chemically modified sarna compositions and methods of use
WO2024197033A1 (en) 2023-03-21 2024-09-26 Modernatx, Inc. Polynucleotides encoding relaxin for the treatment of heart failure
WO2024229321A1 (en) 2023-05-03 2024-11-07 Modernatx, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
WO2025072482A1 (en) 2023-09-27 2025-04-03 Modernatx, Inc. Immunoglobulin a protease polypeptides, polynucleotides, and uses thereof
WO2025255199A1 (en) 2024-06-05 2025-12-11 Modernatx, Inc. Argininosuccinate synthase 1 and argininosuccinate lyase polypeptides and polynucleotides and uses thereof

Family Cites Families (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US2580880A (en) * 1942-03-27 1952-01-01 Nat Aluminate Corp Prevention of foaming in steam generation
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
WO1989008098A2 (en) * 1988-02-19 1989-09-08 The Upjohn Company Lipophilic polyamines useful for treating hypercholesterolemia
JP2610752B2 (ja) * 1992-07-02 1997-05-14 花王株式会社 新規ジアミドアミン及びその製造法
JPH06345704A (ja) * 1993-06-04 1994-12-20 Lion Corp 新規なジアミドアミン化合物、その中間体及びその製造方法と柔軟剤
ATE301201T1 (de) 1993-06-07 2005-08-15 Vical Inc Für die gentherapie verwendbare plasmide
JPH0718571A (ja) * 1993-06-29 1995-01-20 Lion Corp 柔軟剤組成物
JPH0718569A (ja) * 1993-06-29 1995-01-20 Lion Corp 柔軟剤組成物
DE4425857A1 (de) * 1994-07-07 1996-01-11 Schering Ag Kaskaden-Polymer-Komplexe, Verfahren zur ihrer Herstellung und diese enthaltende pharmazeutische Mittel
US5962428A (en) 1995-03-30 1999-10-05 Apollon, Inc. Compositions and methods for delivery of genetic material
JPH101869A (ja) * 1996-06-12 1998-01-06 Lion Corp 液体柔軟剤組成物
PT957929E (pt) * 1996-10-25 2006-06-30 Gilead Sciences Inc Complexos de ligandos de acido nucleico do factor de crescimento endotelial vascular
ZA9811379B (en) * 1997-12-12 1999-08-27 Macromed Inc Hetero-functionalized star-shaped poly(ethylene glycols) for protein modification.
AUPP249298A0 (en) 1998-03-20 1998-04-23 Ag-Gene Australia Limited Synthetic genes and genetic constructs comprising same I
AU1830200A (en) * 1998-11-25 2000-06-13 Vanderbilt University Cationic liposomes for gene transfer
US20030235916A1 (en) * 2002-06-14 2003-12-25 Monahan Sean D. Novel methods for the delivery of polynucleotides to cells
US20050064595A1 (en) * 2003-07-16 2005-03-24 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering RNA
AU2004272646B2 (en) * 2003-09-15 2011-11-24 Arbutus Biopharma Corporation Polyethyleneglycol-modified lipid compounds and uses thereof
US20050095280A1 (en) * 2003-10-15 2005-05-05 Michalakis Savva Single-component pH-sensitive liposomes of reduced solid-to-liquid phase transition temperatures
JP4884231B2 (ja) * 2004-11-02 2012-02-29 公益財団法人新産業創造研究機構 コネキシン２６阻害活性を有する多量体型オレアミド誘導体と、その癌治療等への利用
DE602006017985D1 (de) * 2005-04-15 2010-12-16 Procter & Gamble Reinigungsmittel mit alkoxylierten polyalkyleniminen
ATE536854T1 (de) * 2005-10-20 2011-12-15 Dentsply Detrey Gmbh Dentales haftmittel
WO2008042973A2 (en) * 2006-10-03 2008-04-10 Alnylam Pharmaceuticals, Inc. Lipid containing formulations
EP1955712A1 (en) * 2007-02-09 2008-08-13 Scil proteins GmbH Multimeric conjugate
US8460696B2 (en) 2009-03-20 2013-06-11 Egen, Inc. Polyamine derivatives

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US20130251809A1 (en) 2013-09-26
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US8460696B2 (en) 2013-06-11
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US8900621B2 (en) 2014-12-02
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