ES2931174T3 - Derivados de poliamina - Google Patents

Derivados de poliamina Download PDF

Info

Publication number
ES2931174T3
ES2931174T3 ES10709947T ES10709947T ES2931174T3 ES 2931174 T3 ES2931174 T3 ES 2931174T3 ES 10709947 T ES10709947 T ES 10709947T ES 10709947 T ES10709947 T ES 10709947T ES 2931174 T3 ES2931174 T3 ES 2931174T3
Authority
ES
Spain
Prior art keywords
alkylene
sirna
nr3c
compounds
formulation
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
ES10709947T
Other languages
English (en)
Spanish (es)
Inventor
Gregory Slobodkin
Richard Congo
Majed Matar
Jason Fewell
Khursheed Anwer
Brian Sparks
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Egen Inc
Original Assignee
Egen 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 Egen Inc filed Critical Egen Inc
Application granted granted Critical
Publication of ES2931174T3 publication Critical patent/ES2931174T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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)
  • Communicable Diseases (AREA)
  • Virology (AREA)
  • Urology & Nephrology (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)
ES10709947T 2009-03-20 2010-03-19 Derivados de poliamina Active ES2931174T3 (es)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
ES2931174T3 true ES2931174T3 (es) 2022-12-27

Family

ID=42359538

Family Applications (1)

Application Number Title Priority Date Filing Date
ES10709947T Active ES2931174T3 (es) 2009-03-20 2010-03-19 Derivados de poliamina

Country Status (10)

Country Link
US (4) US8460696B2 (https=)
EP (1) EP2414322B1 (https=)
JP (2) JP5782602B2 (https=)
KR (2) KR101800833B1 (https=)
CN (2) CN102438978B (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.
PT2414322T (pt) 2009-03-20 2022-12-07 Egen Inc Derivados de poliamina
WO2012019168A2 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
RU2013120302A (ru) 2010-10-01 2014-11-20 Модерна Терапьютикс, Инк. Сконструированные нуклеиновые кислоты и способы их применения
US20120136073A1 (en) 2010-11-15 2012-05-31 Life Technologies Corporation Amine-Containing Transfection Reagents and methods for making and using same
US20120202890A1 (en) * 2011-02-08 2012-08-09 Nian Wu Polymer-carbohydrate-lipid conjugates
EP2691101A2 (en) 2011-03-31 2014-02-05 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
AU2012242455A1 (en) 2011-04-15 2013-11-28 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
RU2648950C2 (ru) 2011-10-03 2018-04-02 Модерна Терапьютикс, Инк. Модифицированные нуклеозиды, нуклеотиды и нуклеиновые кислоты и их применение
CN104114572A (zh) 2011-12-16 2014-10-22 现代治疗公司 经修饰的核苷、核苷酸和核酸组合物
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
CA2868391A1 (en) 2012-04-02 2013-10-10 Stephane Bancel Polynucleotides comprising n1-methyl-pseudouridine and methods for preparing the same
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
AU2013243948A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
WO2014081507A1 (en) 2012-11-26 2014-05-30 Moderna Therapeutics, Inc. Terminally modified rna
US20140200261A1 (en) 2013-01-17 2014-07-17 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
WO2015002513A2 (ko) * 2013-07-05 2015-01-08 (주)바이오니아 호흡기 질환 연관 유전자 특이적 siRNA, 그러한 siRNA를 포함하는 이중나선 올리고 RNA 구조체 및 이를 포함하는 호흡기 질환 예방 또는 치료용 조성물
US9695421B2 (en) 2013-07-05 2017-07-04 Bioneer Corporation Dengue virus-specific siRNA, double helix oligo-RNA structure comprising siRNA, and composition for suppressing proliferation of dengue virus comprising RNA structure
CN105705638B (zh) 2013-07-05 2019-11-26 柏业公司 改善的具有高效能的纳米颗粒型寡核苷酸结构及其制备方法
SMT202100691T1 (it) 2013-07-11 2022-01-10 Modernatx Inc Composizioni 5 comprendenti polinucleotidi sintetici che codificano proteine correlate a crispr e sgrna sintetici e metodi d'uso
US20160194368A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Circular polynucleotides
US20160194625A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Chimeric polynucleotides
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
JP2016538829A (ja) 2013-10-03 2016-12-15 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 低密度リポタンパク質受容体をコードするポリヌクレオチド
KR101647178B1 (ko) * 2013-11-28 2016-08-23 충남대학교산학협력단 효소 절단성 링커 또는 올리고 라이신을 포함하는 폴리에틸렌글리콜-리피드를 이용한 안정화된 플라스미드-지질 입자
CN106061466A (zh) 2013-12-19 2016-10-26 诺华股份有限公司 瘦蛋白mRNA组合物和制剂
CN106459974A (zh) 2014-04-04 2017-02-22 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
EP3160959B1 (en) * 2014-06-24 2023-08-30 Translate Bio, Inc. Stereochemically enriched compositions for delivery of nucleic acids
EP3169693B1 (en) 2014-07-16 2022-03-09 ModernaTX, Inc. Chimeric polynucleotides
WO2016014846A1 (en) 2014-07-23 2016-01-28 Moderna Therapeutics, Inc. Modified polynucleotides for the production of intrabodies
CA2975447C (en) * 2015-01-30 2021-02-23 The Regents Of The University Of California Spinal subpial gene delivery system
EP4286012A3 (en) 2015-09-17 2024-05-29 ModernaTX, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
HRP20220652T1 (hr) 2015-12-10 2022-06-24 Modernatx, Inc. Pripravci i postupci unosa terapijskih sredstava
HUE057877T2 (hu) 2015-12-22 2022-06-28 Modernatx Inc Vegyületek és készítmények terápiás szerek intracelluláris bejuttatására
US11583504B2 (en) 2016-11-08 2023-02-21 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
WO2018170322A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Crystal forms of amino lipids
SMT202300097T1 (it) 2017-03-15 2023-05-12 Modernatx Inc Composto e composizioni per il rilascio intracellulare di agenti terapeutici
US11969506B2 (en) 2017-03-15 2024-04-30 Modernatx, Inc. Lipid nanoparticle formulation
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
WO2018213789A1 (en) 2017-05-18 2018-11-22 Modernatx, Inc. Modified messenger rna comprising functional rna elements
EP3638292A1 (en) 2017-06-14 2020-04-22 ModernaTX, Inc. Polynucleotides encoding coagulation factor viii
US12077501B2 (en) 2017-06-14 2024-09-03 Modernatx, Inc. Compounds and compositions for intracellular delivery of agents
US11786607B2 (en) 2017-06-15 2023-10-17 Modernatx, Inc. RNA formulations
WO2019046809A1 (en) 2017-08-31 2019-03-07 Modernatx, Inc. METHODS OF MANUFACTURING LIPID NANOPARTICLES
WO2019048645A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE
JP7423521B2 (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 フェニルケトン尿症の治療用のフェニルアラニンヒドロキシラーゼをコードするポリヌクレオチド
US20220265856A1 (en) 2017-11-22 2022-08-25 Modernatx, Inc. Polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits for the treatment of propionic acidemia
EP3714048B1 (en) 2017-11-22 2025-04-09 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
WO2020056147A2 (en) 2018-09-13 2020-03-19 Modernatx, Inc. Polynucleotides encoding glucose-6-phosphatase for the treatment of glycogen storage disease
US20220243182A1 (en) 2018-09-13 2022-08-04 Modernatx, Inc. Polynucleotides encoding branched-chain alpha-ketoacid dehydrogenase complex e1-alpha, e1-beta, and e2 subunits for the treatment of maple syrup urine disease
WO2020056239A1 (en) 2018-09-14 2020-03-19 Modernatx, Inc. Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome
US12263248B2 (en) 2018-09-19 2025-04-01 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
AU2019345067A1 (en) 2018-09-19 2021-04-08 Modernatx, Inc. High-purity peg lipids and uses thereof
MA53650A (fr) 2018-09-19 2021-07-28 Modernatx Inc Lipides peg et leurs utilisations
CN113271926A (zh) 2018-09-20 2021-08-17 摩登纳特斯有限公司 脂质纳米颗粒的制备及其施用方法
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
BR112022004936A2 (pt) 2019-09-19 2022-06-14 Modernatx Inc Compostos lipídicos contendo carbonato e composições para distribuição intracelular de agentes terapêuticos
US20230235298A1 (en) 2020-06-01 2023-07-27 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
AU2021377895A1 (en) 2020-11-13 2023-06-15 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
WO2022204369A1 (en) 2021-03-24 2022-09-29 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
US20240207444A1 (en) 2021-03-24 2024-06-27 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding phenylalanine hydroxylase and uses thereof
EP4314292A1 (en) 2021-03-26 2024-02-07 MiNA Therapeutics Limited Tmem173 sarna compositions and methods of use
EP4355882A2 (en) 2021-06-15 2024-04-24 Modernatx, Inc. Engineered polynucleotides for cell-type or microenvironment-specific expression
WO2022271776A1 (en) 2021-06-22 2022-12-29 Modernatx, Inc. Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome
US20250017867A1 (en) 2021-10-01 2025-01-16 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
KR102927154B1 (ko) * 2021-12-23 2026-02-12 (주)삼양바이오팜 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
WO2023183909A2 (en) 2022-03-25 2023-09-28 Modernatx, Inc. Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia
US20260035707A1 (en) 2022-07-26 2026-02-05 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
CA2164088C (en) 1993-06-07 2005-06-14 Gary J. Nabel Plasmids suitable for gene therapy
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
DE69735292T2 (de) 1996-10-25 2006-10-19 Gilead Sciences, Inc., Foster City Gefaesszellen wachstumsfaktor (vegf) nukleinsäureligand-komplexe
AR014621A1 (es) * 1997-12-12 2001-03-28 Macromed Inc Un poli (etilenglicol) con forma de estrella, heterofuncional, biocompatible, metodo para su obtencion y metodo para conjugarlo con una proteina
AUPP249298A0 (en) 1998-03-20 1998-04-23 Ag-Gene Australia Limited Synthetic genes and genetic constructs comprising same I
US6656498B1 (en) * 1998-11-25 2003-12-02 Vanderbilt University Cationic liposomes for gene transfer
JP2005529959A (ja) * 2002-06-14 2005-10-06 マイラス コーポレイション 細胞へのポリヌクレオチドの伝達をするための新規な方法
ES2559828T3 (es) * 2003-07-16 2016-02-16 Protiva Biotherapeutics Inc. ARN de interferencia encapsulado en lípidos
NZ592917A (en) * 2003-09-15 2012-12-21 Protiva Biotherapeutics Inc Stable polyethyleneglycol (PEG) dialkyloxypropyl (DAA) lipid conjugates
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
DE502006008083D1 (de) * 2005-04-15 2010-11-25 Basf Se Amphiphile wasserlösliche alkoxylierte polyalkylenimine mit einem inneren polyethylenoxidblock und einem äusseren polypropylenoxidblock
ATE536854T1 (de) * 2005-10-20 2011-12-15 Dentsply Detrey Gmbh Dentales haftmittel
EP3192788A1 (en) * 2006-10-03 2017-07-19 Arbutus Biopharma Corporation Lipid containing formulations
EP1955712A1 (en) * 2007-02-09 2008-08-13 Scil proteins GmbH Multimeric conjugate
PT2414322T (pt) 2009-03-20 2022-12-07 Egen Inc Derivados de poliamina

Also Published As

Publication number Publication date
KR101906392B1 (ko) 2018-10-10
EP2414322B1 (en) 2022-09-14
US20130251809A1 (en) 2013-09-26
KR101800833B1 (ko) 2017-11-23
CN102438978B (zh) 2017-02-22
US20100260817A1 (en) 2010-10-14
WO2010108108A2 (en) 2010-09-23
US8900621B2 (en) 2014-12-02
US8932638B2 (en) 2015-01-13
JP6190401B2 (ja) 2017-08-30
CA2755963C (en) 2019-04-02
CN102438978A (zh) 2012-05-02
JP2015147927A (ja) 2015-08-20
US8460696B2 (en) 2013-06-11
CA2755963A1 (en) 2010-09-23
US9254334B2 (en) 2016-02-09
AU2010226434A1 (en) 2011-10-13
US20150099000A1 (en) 2015-04-09
US20140050775A1 (en) 2014-02-20
EP2414322A2 (en) 2012-02-08
PT2414322T (pt) 2022-12-07
KR20110129969A (ko) 2011-12-02
JP5782602B2 (ja) 2015-09-24
WO2010108108A3 (en) 2011-04-07
JP2012521358A (ja) 2012-09-13
CN104922676B (zh) 2019-03-12
KR20170130626A (ko) 2017-11-28
CN104922676A (zh) 2015-09-23

Similar Documents

Publication Publication Date Title
ES2931174T3 (es) Derivados de poliamina
US20240246898A1 (en) Cationic lipids for nucleic acid delivery and preparation thereof
KR20230144574A (ko) 폴리옥사졸린-지질 접합체, 및 이를 포함하는 지질 나노입자 및 제약 조성물
US9320798B2 (en) Lipid delivery formulations
KR102560772B1 (ko) 신규한 이온화지질 및 이를 이용한 지질나노입자 조성물
JP2009513151A (ja) 修飾ｓｉＲＮＡ分子およびその使用法
WO2013032643A2 (en) Lipids capable of conformational change and their use in formulations to deliver therapeutic agents to cells
ES2542864T3 (es) Composición portadora de administración rápida de ácidos nucleicos
KR20240159310A (ko) 신규한 이온화지질을 이용한 지질나노입자 조성물
JP2025518221A (ja) 核酸の送達のための複合体
KR102917928B1 (ko) 신규한 이온화 지질 및 이를 이용한 지질 나노입자 조성물
CA3270619C (en) Long-acting spleen-targeting cationic lipid compound comprising benzene ring structure, composition comprising same, and use thereof
WO2010101226A1 (ja) 核酸複合体、及び核酸送達用組成物
CA3039137C (en) Cationic lipids for nucleic acid delivery and preparation thereof
CN121135600A (zh) 可电离脂质化合物及其应用