CA2755963C - Polyamine derivatives - Google Patents

Polyamine derivatives Download PDF

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Publication number
CA2755963C
CA2755963C CA2755963A CA2755963A CA2755963C CA 2755963 C CA2755963 C CA 2755963C CA 2755963 A CA2755963 A CA 2755963A CA 2755963 A CA2755963 A CA 2755963A CA 2755963 C CA2755963 C CA 2755963C
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Canada
Prior art keywords
alkylene
nr3c
formula
alkyleneoxy
independently
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CA2755963A
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English (en)
French (fr)
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CA2755963A1 (en
Inventor
Gregory Slobodkin
Richard Congo
Majed Matar
Jason Fewell
Khursheed Anwer
Brian Jeffery Sparks
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CLSN LABORATORIES Inc
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CLSN LABORATORIES Inc
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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
    • 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
    • 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
    • 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

Landscapes

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Genetics & Genomics (AREA)
  • Dispersion Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biotechnology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Oncology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Endocrinology (AREA)
  • Virology (AREA)
  • Urology & Nephrology (AREA)
  • Communicable Diseases (AREA)
  • Ophthalmology & Optometry (AREA)
  • Pulmonology (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)
CA2755963A 2009-03-20 2010-03-19 Polyamine derivatives Active CA2755963C (en)

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

Publications (2)

Publication Number Publication Date
CA2755963A1 CA2755963A1 (en) 2010-09-23
CA2755963C true CA2755963C (en) 2019-04-02

Family

ID=42359538

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2755963A Active CA2755963C (en) 2009-03-20 2010-03-19 Polyamine derivatives

Country Status (10)

Country Link
US (4) US8460696B2 (https=)
EP (1) EP2414322B1 (https=)
JP (2) JP5782602B2 (https=)
KR (2) KR101906392B1 (https=)
CN (2) CN104922676B (https=)
AU (1) AU2010226434A1 (https=)
CA (1) CA2755963C (https=)
ES (1) ES2931174T3 (https=)
PT (1) PT2414322T (https=)
WO (1) WO2010108108A2 (https=)

Families Citing this family (102)

* 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
WO2012019168A2 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP2625189B1 (en) 2010-10-01 2018-06-27 ModernaTX, Inc. Engineered nucleic acids and methods of use thereof
CN103380113B (zh) 2010-11-15 2018-03-30 生命科技公司 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
AU2012236099A1 (en) 2011-03-31 2013-10-03 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
WO2012142622A1 (en) 2011-04-15 2012-10-18 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
DE19216461T1 (de) 2011-10-03 2021-10-07 Modernatx, Inc. Modifizierte nukleoside, nukleotide und nukleinsäuren und verwendungen davon
CA3018046A1 (en) 2011-12-16 2013-06-20 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
WO2013151665A2 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
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
CN104411338A (zh) 2012-04-02 2015-03-11 现代治疗公司 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
PL2922554T3 (pl) 2012-11-26 2022-06-20 Modernatx, Inc. Na zmodyfikowany na końcach
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
WO2014152211A1 (en) 2013-03-14 2014-09-25 Moderna Therapeutics, 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
CN105705639A (zh) 2013-07-05 2016-06-22 柏业公司 登革病毒相关的基因特异性siRNA、包含该siRNA的双链寡RNA分子、和包含它们的用于抑制登革病毒复制的组合物
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
CN110592082A (zh) * 2013-07-05 2019-12-20 柏业公司 呼吸疾病相关基因特异性siRNA、含有siRNA的双螺旋寡RNA结构及其用途
CA2917348A1 (en) 2013-07-11 2015-01-15 Moderna Therapeutics, Inc. Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use
WO2015034925A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Circular polynucleotides
EP3041934A1 (en) 2013-09-03 2016-07-13 Moderna Therapeutics, Inc. Chimeric 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
KR101647178B1 (ko) * 2013-11-28 2016-08-23 충남대학교산학협력단 효소 절단성 링커 또는 올리고 라이신을 포함하는 폴리에틸렌글리콜-리피드를 이용한 안정화된 플라스미드-지질 입자
JP2017500865A (ja) 2013-12-19 2017-01-12 ノバルティス アーゲー レプチンｍＲＮＡの組成物および製剤
EP3128008B1 (en) 2014-04-04 2024-05-29 Bioneer Corporation Double-stranded oligo rna and pharmaceutical composition comprising same for preventing or treating fibrosis or respiratory diseases
PL4023249T3 (pl) 2014-04-23 2025-03-10 Modernatx, Inc. Szczepionki z kwasem nukleinowym
KR102559979B1 (ko) * 2014-06-24 2023-07-25 샤이어 휴먼 지네틱 테라피즈 인크. 핵산의 전달용 입체화학적으로 풍부한 조성물
US20170204152A1 (en) 2014-07-16 2017-07-20 Moderna Therapeutics, Inc. Chimeric polynucleotides
EP3171895A1 (en) 2014-07-23 2017-05-31 Modernatx, 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
JP6948313B6 (ja) 2015-09-17 2022-01-14 モデルナティエックス インコーポレイテッド 治療剤の細胞内送達のための化合物および組成物
PL3386484T3 (pl) 2015-12-10 2022-07-25 Modernatx, Inc. Kompozycje i sposoby dostarczania środków terapeutycznych
LT3394030T (lt) 2015-12-22 2022-04-11 Modernatx, Inc. Junginiai ir kompozicijos terapinei medžiagai teikti intraceliuliniu būdu
US11583504B2 (en) 2016-11-08 2023-02-21 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
CA3055653A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Lipid nanoparticle formulation
WO2018170322A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Crystal forms of amino lipids
EP4186888B1 (en) 2017-03-15 2025-11-26 ModernaTX, Inc. Compound and compositions for intracellular delivery of therapeutic agents
JP7285220B2 (ja) 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 連結したインターロイキン－１２（ｉｌ１２）ポリペプチドをコードするポリヌクレオチドを含む脂質ナノ粒子
US11485972B2 (en) 2017-05-18 2022-11-01 Modernatx, Inc. Modified messenger RNA comprising functional RNA elements
EP3638292A1 (en) 2017-06-14 2020-04-22 ModernaTX, Inc. Polynucleotides encoding coagulation factor viii
WO2018232120A1 (en) 2017-06-14 2018-12-20 Modernatx, Inc. Compounds and compositions for intracellular delivery of agents
MA49421A (fr) 2017-06-15 2020-04-22 Modernatx Inc Formulations d'arn
EP3675817A1 (en) 2017-08-31 2020-07-08 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
US11939601B2 (en) 2017-11-22 2024-03-26 Modernatx, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
EP3714045A1 (en) 2017-11-22 2020-09-30 Modernatx, Inc. Polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits for the treatment of propionic acidemia
CA3079428A1 (en) 2017-11-22 2019-05-31 Modernatx, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders
MA51523A (fr) 2018-01-05 2020-11-11 Modernatx Inc Polynucléotides codant pour des anticorps anti-virus du chikungunya
KR102056144B1 (ko) 2018-03-08 2019-12-16 주식회사 파마리서치프로덕트 Dna 단편 혼합물이 고농도로 함유된 유동성을 갖는 액제 조성물 및 이의 제조방법
EP3775211B1 (en) 2018-04-12 2023-04-05 MiNA Therapeutics Limited Sirt1-sarna compositions and methods of use
WO2019226650A1 (en) 2018-05-23 2019-11-28 Modernatx, Inc. Delivery of dna
US20220184185A1 (en) 2018-07-25 2022-06-16 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
WO2020056155A2 (en) 2018-09-13 2020-03-19 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
MX2021003015A (es) 2018-09-13 2021-07-15 Modernatx Inc Polinucleotidos que codifican glucosa-6-fosfatasa para el tratamiento de la glucogenosis.
JP2022500444A (ja) 2018-09-14 2022-01-04 モダーナティエックス・インコーポレイテッドModernaTX, Inc. クリグラー−ナジャー症候群の治療のためのウリジン二リン酸グリコシルトランスフェラーゼ１ファミリー、ポリペプチドａ１をコードするポリヌクレオチド
WO2020061295A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. High-purity peg lipids and uses thereof
CA3113025A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. Peg lipids and uses thereof
WO2020061367A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
WO2020061457A1 (en) 2018-09-20 2020-03-26 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
EP3856233A1 (en) 2018-09-27 2021-08-04 Modernatx, Inc. Polynucleotides encoding arginase 1 for the treatment of arginase deficiency
EP3953473A1 (en) 2019-04-12 2022-02-16 MiNA Therapeutics Limited Sirt1-sarna compositions and methods of use
JP2022532078A (ja) 2019-05-08 2022-07-13 アストラゼネカ アクチボラグ 皮膚及び創傷のための組成物並びにその使用の方法
AU2020350759A1 (en) 2019-09-19 2022-03-31 Modernatx, Inc. Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents
CA3155015A1 (en) 2019-09-19 2021-03-25 Modernatx, Inc. Carbonate containing lipid compounds and compositions for intracellular delivery of therapeutic agents
EP4158005A1 (en) 2020-06-01 2023-04-05 ModernaTX, Inc. Phenylalanine hydroxylase variants and uses thereof
US20230365966A1 (en) * 2020-10-09 2023-11-16 Napajen Pharma, Inc. Homogeneous beta-1,3-glucan/nucleic acid complexes and use thereof
WO2022104131A1 (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
WO2022204390A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding phenylalanine hydroxylase and uses thereof
US20240207374A1 (en) 2021-03-24 2024-06-27 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding glucose-6-phosphatase and uses thereof
US20240226025A1 (en) 2021-03-24 2024-07-11 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia
US20240216288A1 (en) 2021-03-24 2024-07-04 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
TW202305133A (zh) 2021-03-26 2023-02-01 英商米納治療有限公司 Tmem173 sarna組合物及其使用方法
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
KR102852074B1 (ko) * 2021-12-23 2025-08-28 주식회사 삼양홀딩스 약물 전달용 지질 및 이를 포함하는 나노입자, 및 이 나노입자를 포함하는 약물 전달용 조성물
KR102927154B1 (ko) * 2021-12-23 2026-02-12 (주)삼양바이오팜 약물의 폐 전달용 나노입자 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
WO2024026254A1 (en) 2022-07-26 2024-02-01 Modernatx, Inc. Engineered polynucleotides for temporal control of expression
WO2024044728A1 (en) * 2022-08-26 2024-02-29 Renagade Therapeutics Management Inc. Pegylated lipid compounds and methods of use thereof
WO2024125597A1 (en) 2022-12-14 2024-06-20 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
EP4704816A1 (en) 2023-05-03 2026-03-11 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
WO2026027887A2 (en) 2024-08-02 2026-02-05 Mina Therapeutics Limited Hbg1/2-sarna compositions and methods of use

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
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
AU3203489A (en) 1988-02-19 1989-09-22 Upjohn Company, The 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
JP3626187B2 (ja) 1993-06-07 2005-03-02 バイカル インコーポレイテッド 遺伝子治療に適するプラスミド
JPH0718569A (ja) * 1993-06-29 1995-01-20 Lion Corp 柔軟剤組成物
JPH0718571A (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
RU2177950C2 (ru) * 1996-10-25 2002-01-10 Нексстар Фармасьютикалз, Инк. Комплексы нуклеиновых кислот - лигандов сосудистого эндотелиального фактора роста (vegf)
KR20010015856A (ko) * 1997-12-12 2001-02-26 마크로메드 인코퍼레이션 단백질 변성용으로 헤테로 작용기를 가진 별 모양의폴리(에틸렌글리콜)
AUPP249298A0 (en) 1998-03-20 1998-04-23 Ag-Gene Australia Limited Synthetic genes and genetic constructs comprising same I
WO2000030444A1 (en) * 1998-11-25 2000-06-02 Vanderbilt University Cationic liposomes for gene transfer
WO2003106636A2 (en) 2002-06-14 2003-12-24 Mirus Corporation Novel methods for the delivery of polynucleotides to cells
CA2532228C (en) * 2003-07-16 2017-02-14 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
EP1664316B1 (en) * 2003-09-15 2012-08-29 Protiva Biotherapeutics Inc. 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
WO2006049157A1 (ja) * 2004-11-02 2006-05-11 The New Industry Research Organization 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
US7999035B2 (en) * 2005-04-15 2011-08-16 Basf Aktiengesellschaft Amphiphilic water-soluble alkoxylated polyalkylenimines with an internal polyethylene oxide block and an external polypropylene oxide block
EP1776943B1 (en) * 2005-10-20 2011-12-14 Dentsply DeTrey GmbH Dental adhesive
CA2927045A1 (en) * 2006-10-03 2008-04-10 Muthiah Manoharan 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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