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ES2897565T8 - Lípidos catiónicos de amina y uso de los mismos - Google Patents

Lípidos catiónicos de amina y uso de los mismos

Info

Publication number
ES2897565T8
ES2897565T8 ES19191168T ES19191168T ES2897565T8 ES 2897565 T8 ES2897565 T8 ES 2897565T8 ES 19191168 T ES19191168 T ES 19191168T ES 19191168 T ES19191168 T ES 19191168T ES 2897565 T8 ES2897565 T8 ES 2897565T8
Authority
ES
Spain
Prior art keywords
cationic amine
amine lipids
lipids
cationic
amine
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
ES19191168T
Other languages
English (en)
Other versions
ES2897565T3 (es
Inventor
Bob Dale Brown
Sujit Kumar Basu
David Schwartz
Allister Fraser
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.)
Dicerna Pharmaceuticals Inc
Original Assignee
Dicerna Pharmaceuticals 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 Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Application granted granted Critical
Publication of ES2897565T3 publication Critical patent/ES2897565T3/es
Publication of ES2897565T8 publication Critical patent/ES2897565T8/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D403/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
    • C07D403/02—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings
    • C07D403/06—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings linked by a carbon chain containing only aliphatic carbon atoms
    • 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
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • 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
    • A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • 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
    • A61K9/107—Emulsions ; Emulsion preconcentrates; Micelles
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    • 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
    • A—HUMAN NECESSITIES
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    • 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
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    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/141—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers
    • A61K9/145—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers with organic compounds
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    • A61K9/141—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers
    • A61K9/146—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers with organic macromolecular compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C—CHEMISTRY; METALLURGY
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    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C211/00—Compounds containing amino groups bound to a carbon skeleton
    • C07C211/01—Compounds containing amino groups bound to a carbon skeleton having amino groups bound to acyclic carbon atoms
    • C07C211/20—Compounds containing amino groups bound to a carbon skeleton having amino groups bound to acyclic carbon atoms of an acyclic unsaturated carbon skeleton
    • C07C211/21—Monoamines
    • C—CHEMISTRY; METALLURGY
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    • C07C237/00—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups
    • C07C237/02—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton
    • C07C237/04—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C237/06—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated having the nitrogen atoms of the carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms
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    • C07D211/00—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
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    • C07D211/06—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
    • C07D211/36—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
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    • C07D211/00—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
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    • C07D211/06—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
    • C07D211/36—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D211/56—Nitrogen atoms
    • C07D211/58—Nitrogen atoms attached in position 4
    • C—CHEMISTRY; METALLURGY
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    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D213/00—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members
    • C07D213/02—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members
    • C07D213/04—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D213/60—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen or carbon atoms directly attached to the ring nitrogen atom with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D213/72—Nitrogen atoms
    • C07D213/74—Amino or imino radicals substituted by hydrocarbon or substituted hydrocarbon radicals
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    • C07—ORGANIC CHEMISTRY
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    • C07D233/00—Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings
    • C07D233/54—Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings having two double bonds between ring members or between ring members and non-ring members
    • C07D233/56—Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings having two double bonds between ring members or between ring members and non-ring members with only hydrogen atoms or radicals containing only hydrogen and carbon atoms, attached to ring carbon atoms
    • C07D233/61—Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings having two double bonds between ring members or between ring members and non-ring members with only hydrogen atoms or radicals containing only hydrogen and carbon atoms, attached to ring carbon atoms with hydrocarbon radicals, substituted by nitrogen atoms not forming part of a nitro radical, attached to ring nitrogen atoms
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    • C07D233/64—Heterocyclic compounds containing 1,3-diazole or hydrogenated 1,3-diazole rings, not condensed with other rings having two double bonds between ring members or between ring members and non-ring members with substituted hydrocarbon radicals attached to ring carbon atoms, e.g. histidine
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    • C07D239/02—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings
    • C07D239/24—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings having three or more double bonds between ring members or between ring members and non-ring members
    • C07D239/28—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings having three or more double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, directly attached to ring carbon atoms
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    • C—CHEMISTRY; METALLURGY
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    • C07D241/04—Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings not condensed with other rings having no double bonds between ring members or between ring members and non-ring members
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    • C07D243/00—Heterocyclic compounds containing seven-membered rings having two nitrogen atoms as the only ring hetero atoms
    • C07D243/06—Heterocyclic compounds containing seven-membered rings having two nitrogen atoms as the only ring hetero atoms having the nitrogen atoms in positions 1 and 4
    • C07D243/08—Heterocyclic compounds containing seven-membered rings having two nitrogen atoms as the only ring hetero atoms having the nitrogen atoms in positions 1 and 4 not condensed with other rings
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    • C07D295/00—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
    • C07D295/02—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms containing only hydrogen and carbon atoms in addition to the ring hetero elements
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    • C07D295/02—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms containing only hydrogen and carbon atoms in addition to the ring hetero elements
    • C07D295/027—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms containing only hydrogen and carbon atoms in addition to the ring hetero elements containing only one hetero ring
    • C07D295/03—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms containing only hydrogen and carbon atoms in addition to the ring hetero elements containing only one hetero ring with the ring nitrogen atoms directly attached to acyclic carbon atoms
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    • C07D295/04—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms
    • C07D295/12—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms
    • C07D295/125—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms with the ring nitrogen atoms and the substituent nitrogen atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings
    • C07D295/13—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly or doubly bound nitrogen atoms with the ring nitrogen atoms and the substituent nitrogen atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings to an acyclic saturated chain
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    • C07D295/04—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms
    • C07D295/14—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals
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Families Citing this family (138)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3067046B1 (en) 2005-12-08 2020-03-25 Insmed Incorporated Lipid-based compositions of antiinfectives for treating pulmonary infections
US9119783B2 (en) 2007-05-07 2015-09-01 Insmed Incorporated Method of treating pulmonary disorders with liposomal amikacin formulations
EP3578205A1 (en) 2010-08-06 2019-12-11 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof
CN103429606A (zh) 2010-10-01 2013-12-04 现代治疗公司 设计核酸及其使用方法
WO2012083005A2 (en) 2010-12-15 2012-06-21 Miragen Therapeutics Microrna inhibitors comprising locked nucleotides
CA2831613A1 (en) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
KR20190099538A (ko) 2011-10-03 2019-08-27 모더나 세라퓨틱스, 인코포레이티드 변형된 뉴클레오사이드, 뉴클레오타이드, 및 핵산, 및 이들의 용도
KR20140091688A (ko) 2011-10-06 2014-07-22 미라젠 세러퓨틱스 인코포레이티드 마이크로rna 조절에 의한 전신 에너지 항상성의 제어
EP2768958B1 (en) * 2011-10-18 2019-08-14 Dicerna Pharmaceuticals, Inc. Amine cationic lipids and uses thereof
US9061063B2 (en) 2011-12-07 2015-06-23 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
EP4144378A1 (en) 2011-12-16 2023-03-08 ModernaTX, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
AU2013243954A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
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
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
CA2876180C (en) 2012-06-21 2019-11-19 MiRagen Therapeutics, Inc. Oligonucleotide-based inhibitors comprising locked nucleic acid motif
DK2922554T3 (en) 2012-11-26 2022-05-23 Modernatx Inc Terminalt modificeret rna
US10124066B2 (en) 2012-11-29 2018-11-13 Insmed Incorporated Stabilized vancomycin formulations
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
US9567641B2 (en) 2013-07-03 2017-02-14 Samsung Electronics Co., Ltd. Combination therapy for the treatment of cancer using an anti-C-met antibody
US10030243B2 (en) 2013-07-05 2018-07-24 Bioneer Corporation Nanoparticle type oligonucleotide structure having high efficiency and method for preparing same
JP2016525346A (ja) * 2013-07-05 2016-08-25 バイオニア コーポレーションＢｉｏｎｅｅｒ Ｃｏｒｐｏｒａｔｉｏｎ 呼吸器疾患関連遺伝子特異的ｓｉＲＮＡ、そのようなｓｉＲＮＡを含む二重らせんオリゴＲＮＡ構造体およびこれを含む呼吸器疾患予防または治療用組成物
SG11201507571TA (en) * 2013-07-05 2015-10-29 Bioneer Corp Dengue virus-specific sirna, double helix oligo-rna structure comprising sirna, and composition for suppressing proliferation of dengue virus comprising rna structure
US20160194368A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Circular polynucleotides
AU2014315287A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
BR112016007255A2 (pt) 2013-10-03 2017-09-12 Moderna Therapeutics Inc polinucleotídeos que codificam receptor de lipoproteína de baixa densidade
JP6389477B2 (ja) * 2014-01-09 2018-09-12 エーザイ・アール・アンド・ディー・マネジメント株式会社 カチオン性脂質
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
PT3981437T (pt) 2014-04-23 2025-01-15 Modernatx Inc Vacinas de ácidos nucleicos
CN106535877A (zh) 2014-05-15 2017-03-22 英斯梅德股份有限公司 用于治疗肺部非结核性分枝杆菌感染的方法
CN105085437B (zh) * 2014-05-23 2018-07-03 上海交通大学 3-(1-叔丁氧羰酰哌嗪-4-yl)丙酸的两亲性衍生物及其用途
HUE060907T2 (hu) 2014-06-25 2023-04-28 Acuitas Therapeutics Inc Új lipidek és lipid nanorészecske formulációk nukleinsavak bevitelére
WO2016011226A1 (en) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Chimeric polynucleotides
US20170210788A1 (en) 2014-07-23 2017-07-27 Modernatx, Inc. Modified polynucleotides for the production of intrabodies
SG11201705907XA (en) 2015-01-20 2017-08-30 Miragen Therapeutics Inc Mir-92 inhibitors and uses thereof
CA2984125A1 (en) 2015-04-27 2016-11-03 The Trustees Of The University Of Pennsylvania Nucleoside-modified rna for inducing an adaptive immune response
CN104873976B (zh) * 2015-05-08 2016-04-20 厦门成坤生物技术有限公司 一类阳离子类脂化合物的应用
US10221127B2 (en) * 2015-06-29 2019-03-05 Acuitas Therapeutics, Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
US11364292B2 (en) 2015-07-21 2022-06-21 Modernatx, Inc. CHIKV RNA vaccines
TW201718638A (zh) 2015-07-21 2017-06-01 現代治療公司 傳染病疫苗
US11564893B2 (en) 2015-08-17 2023-01-31 Modernatx, Inc. Methods for preparing particles and related compositions
WO2017044623A1 (en) * 2015-09-09 2017-03-16 Lau Warren C Methods, compositions, and uses of novel fyn kinase inhibitors
SMT202200178T1 (it) 2015-09-17 2022-05-12 Modernatx Inc Composti e composizioni per il rilascio intracellulare di agenti terapeutici
CN105205747A (zh) * 2015-10-08 2015-12-30 张姬娟 送餐信息的处理方法
KR20180096592A (ko) 2015-10-22 2018-08-29 모더나티엑스, 인크. 호흡기 세포융합 바이러스 백신
JP6921833B2 (ja) 2015-10-22 2021-08-18 モデルナティーエックス， インコーポレイテッド ヒトサイトメガロウイルスワクチン
US11643441B1 (en) 2015-10-22 2023-05-09 Modernatx, Inc. Nucleic acid vaccines for varicella zoster virus (VZV)
MA45209A (fr) 2015-10-22 2019-04-17 Modernatx Inc Vaccins contre les maladies sexuellement transmissibles
EP3718565B1 (en) 2015-10-22 2022-04-27 ModernaTX, Inc. Respiratory virus vaccines
WO2017070624A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Tropical disease vaccines
CN118846026A (zh) 2015-10-22 2024-10-29 摩登纳特斯有限公司 广谱流感病毒疫苗
CA3002822A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Herpes simplex virus vaccine
AU2016344384A1 (en) 2015-10-26 2018-05-17 Translate Bio Ma, Inc. Nanoparticle formulations for delivery of nucleic acid complexes
IL307179A (en) 2015-10-28 2023-11-01 Acuitas Therapeutics Inc Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
JP2019500318A (ja) * 2015-11-18 2019-01-10 インスメッド インコーポレイテッド 細菌感染症を治療するための組成物及び方法
PL3386484T3 (pl) 2015-12-10 2022-07-25 Modernatx, Inc. Kompozycje i sposoby dostarczania środków terapeutycznych
RU2609107C1 (ru) * 2015-12-16 2017-01-30 Федеральное Государственное Бюджетное Учреждение Науки Институт Молекулярной Биологии Им. В.А. Энгельгардта Российской Академии Наук (Имб Ран) Подавление экспрессии гена C-KIT в клетках нейробластом и лентивирусные конструкции, направляющие синтез shPHK, специфичных в отношении данного гена
PL3394030T3 (pl) 2015-12-22 2022-04-11 Modernatx, Inc. Związki i kompozycje do wewnątrzkomórkowego dostarczania środków
SI3394093T1 (sl) 2015-12-23 2022-05-31 Modernatx, Inc. Metode uporabe liganda OX40, ki kodira polinukleotid
MA43587A (fr) 2016-01-10 2018-11-14 Modernatx Inc Arnm thérapeutiques codant pour des anticorps anti-ctla-4
WO2017174417A1 (en) * 2016-04-08 2017-10-12 Rhodia Operations Diamines surfactants suitable as emulsifier
IL263079B2 (en) 2016-05-18 2024-05-01 Modernatx Inc Polynucleotides encoding relaxin
US10501416B2 (en) 2016-06-24 2019-12-10 Eisai R&D Management Co., Ltd. Cationic lipid
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
US11583504B2 (en) 2016-11-08 2023-02-21 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
BR112019008550B1 (pt) * 2016-11-08 2023-02-07 Rhodia Operations Composto, processo para preparar um composto, uso de um composto, composição adjuvante, e, método para controlar a flutuação de pulverização de uma composição aquosa de pesticida
EP3538146A4 (en) 2016-11-11 2020-07-15 ModernaTX, Inc. INFLUENZA VACCINE
CN110177544A (zh) 2016-11-29 2019-08-27 普尔泰克健康有限公司 用于递送治疗剂的外泌体
US11103578B2 (en) 2016-12-08 2021-08-31 Modernatx, Inc. Respiratory virus nucleic acid vaccines
JP7317715B2 (ja) 2017-01-11 2023-07-31 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ジカウイルスに対する免疫応答を誘導するためのヌクレオシド改変ｒｎａ
US11464848B2 (en) 2017-03-15 2022-10-11 Modernatx, Inc. Respiratory syncytial virus vaccine
WO2018170256A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Herpes simplex virus vaccine
AU2018234828A1 (en) 2017-03-15 2019-09-19 Modernatx, Inc. Lipid nanoparticle formulation
WO2018170245A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Broad spectrum influenza virus vaccine
LT3596041T (lt) 2017-03-15 2023-01-25 Modernatx, Inc. Terapinių medžiagų, skirtų intraląsteliniam tiekimui, junginys ir sudėtys
AU2018234814B2 (en) 2017-03-15 2022-06-30 Modernatx, Inc. Crystal forms of amino lipids
WO2018170270A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Varicella zoster virus (vzv) vaccine
US11357856B2 (en) 2017-04-13 2022-06-14 Acuitas Therapeutics, Inc. Lipids for delivery of active agents
CN116693411A (zh) 2017-04-28 2023-09-05 爱康泰生治疗公司 用于递送核酸的新型羰基脂质和脂质纳米颗粒制剂
EP3638678B1 (en) 2017-06-14 2025-12-03 ModernaTX, Inc. Compounds and compositions for intracellular delivery of agents
EP3638215A4 (en) 2017-06-15 2021-03-24 Modernatx, Inc. RNA FORMULATIONS
EP3662913A4 (en) 2017-08-04 2021-06-30 Kyowa Kirin Co., Ltd. Nucleic-acid-containing lipid nanoparticles
US11639329B2 (en) 2017-08-16 2023-05-02 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
US12065396B2 (en) 2017-08-17 2024-08-20 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019036028A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICULAR FORMULATIONS
US11524932B2 (en) 2017-08-17 2022-12-13 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
CN111315359A (zh) 2017-08-31 2020-06-19 摩登纳特斯有限公司 制备脂质纳米颗粒的方法
US10653767B2 (en) 2017-09-14 2020-05-19 Modernatx, Inc. Zika virus MRNA vaccines
EP3728181B1 (en) * 2017-12-20 2024-05-08 Specialty Operations France New cationic quaternary ammonium compounds and compositions comprising same and processes for their manufacture
EP3733647B1 (en) 2017-12-27 2022-06-15 Eisai R&D Management Co., Ltd. Cationic lipid
WO2019148101A1 (en) 2018-01-29 2019-08-01 Modernatx, Inc. Rsv rna vaccines
CA3089897A1 (en) * 2018-02-21 2019-08-29 Rhodia Operations Gelling fluids and related methods of use
CN110283223B (zh) * 2018-03-19 2022-01-04 广州市锐博生物科技有限公司 一种阳离子脂质分子及其在核酸递送中的应用
WO2019191627A1 (en) 2018-03-30 2019-10-03 Insmed Incorporated Methods for continuous manufacture of liposomal drug products
CN112533909B (zh) * 2018-05-30 2024-11-15 川斯勒佰尔公司 维生素阳离子脂质
CN116172975B (zh) 2018-06-08 2025-03-28 富士胶片株式会社 脂质颗粒、包含该脂质颗粒的核酸药物以及核酸递送用组合物
US12357580B2 (en) 2018-06-19 2025-07-15 The Board Of Regents Of The University Of Texas System Lipid nanoparticle compositions for delivery of mRNA and long nucleic acids
WO2020051220A1 (en) * 2018-09-04 2020-03-12 The Board of the Regents of the University of Texas System Compositions and methods for organ specific delivery of nucleic acids
AU2019336679B2 (en) 2018-09-04 2025-05-29 The Board Of Regents Of The University Of Texas System Compositions and methods for organ specific delivery of nucleic acids
EP3852732A1 (en) 2018-09-19 2021-07-28 ModernaTX, Inc. Peg lipids and uses thereof
CA3113436A1 (en) 2018-09-19 2020-03-26 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
WO2020061457A1 (en) 2018-09-20 2020-03-26 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
US12329857B2 (en) 2018-09-21 2025-06-17 Acuitas Therapeutics, Inc. Systems and methods for manufacturing lipid nanoparticles and liposomes
AU2020205717B2 (en) 2019-01-11 2025-09-11 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
US11351242B1 (en) 2019-02-12 2022-06-07 Modernatx, Inc. HMPV/hPIV3 mRNA vaccine composition
WO2020190750A1 (en) 2019-03-15 2020-09-24 Modernatx, Inc. Hiv rna vaccines
KR20250152113A (ko) * 2019-04-25 2025-10-22 인텔리아 테라퓨틱스, 인크. 이온화 가능한 아민 지질 및 지질 나노입자
BR112022004759A2 (pt) 2019-09-19 2022-06-21 Modernatx Inc Composições e compostos lipídicos com cauda ramificada para entrega intracelular de agentes terapêuticos
EP4087619A4 (en) * 2020-01-07 2024-02-21 Translate Bio MA, Inc. FORMULATIONS FOR THE DELIVERY OF OLIGONUCLEOTIDES TO LUNG CELLS
IL296133A (en) 2020-03-04 2022-11-01 Verve Therapeutics Inc Preparations and methods for administering targeted RNA
AU2021308681A1 (en) 2020-07-16 2023-03-09 Acuitas Therapeutics, Inc. Cationic lipids for use in lipid nanoparticles
JP2023533864A (ja) * 2020-07-17 2023-08-04 サノフィ・パスツール 式－ｎｈ－ｃｘ－ａまたは－ｎｈ－ｃｘ－ｎｈ－ａの少なくとも１つの末端ラジカルを含む脂質化合物、それらを含有する組成物およびそれらの使用
WO2022081750A1 (en) 2020-10-14 2022-04-21 George Mason Research Foundation, Inc. Ionizable lipids and methods of manufacture and use thereof
CN113185421B (zh) * 2020-11-27 2022-01-25 广州市锐博生物科技有限公司 脂质化合物及其组合物
EP4255888A4 (en) * 2020-12-02 2024-12-04 Merck Sharp & Dohme LLC LIPID DNANOPARTICLE COMPOSITIONS WITH CATIONIC MONOESTER LIPIDS
CN114904002B (zh) * 2021-02-09 2024-04-26 广州立得生物医药科技有限公司 可电离的阳离子脂质类似物材料及其在作为药物递送载体中的应用
US11524023B2 (en) 2021-02-19 2022-12-13 Modernatx, Inc. Lipid nanoparticle compositions and methods of formulating the same
CN112979483B (zh) * 2021-05-14 2021-08-06 苏州艾博生物科技有限公司 一种阳离子脂质化合物、包含其的组合物及应用
JP7781448B2 (ja) * 2021-05-28 2025-12-08 北京啓辰生生物科技有限公司 脂質化合物及び核酸送達におけるその応用
WO2022266385A1 (en) * 2021-06-17 2022-12-22 The Regents Of The University Of California Vesicle formulations for delivery of antifungal nucleic acids
KR20240123832A (ko) 2021-12-16 2024-08-14 아퀴타스 테라퓨틱스 인크. 지질 나노입자 제형에 사용하기 위한 지질
US20250327041A1 (en) 2022-02-24 2025-10-23 Io Biotech Aps Nucleotide delivery of cancer therapy
KR102465349B1 (ko) * 2022-02-25 2022-11-11 주식회사 테르나테라퓨틱스 신규 이온화 지질 및 이를 포함하는 지질 나노입자
WO2023166511A1 (en) * 2022-03-02 2023-09-07 Barcode Nanotech Ltd. Ionizable lipids and compositions comprising same
CN116813493A (zh) * 2022-03-21 2023-09-29 苏州科锐迈德生物医药科技有限公司 一种脂质化合物及基于其的脂质载体、核酸脂质纳米粒组合物和药物制剂
WO2024006960A1 (en) 2022-06-29 2024-01-04 Juno Therapeutics, Inc. Lipid nanoparticles for delivery of nucleic acids
CN115232128B (zh) * 2022-08-15 2024-03-29 中国科学技术大学 一种咪唑并吡啶基脂质化合物及其制备方法与应用
WO2024177424A1 (ko) * 2023-02-24 2024-08-29 에스티팜 주식회사 신규 이온화 가능한 지질 및 이를 포함하는 지질나노입자
KR20250141799A (ko) * 2023-03-13 2025-09-29 후지필름 가부시키가이샤 화합물 또는 그 염, 지질 조성물, 의약 조성물 및 송달 캐리어
WO2024249954A1 (en) 2023-05-31 2024-12-05 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
EP4520345A1 (en) 2023-09-06 2025-03-12 Myneo Nv Product
CN117482066B (zh) * 2023-11-02 2024-08-13 深圳市易瑞生物技术股份有限公司 脂质组合物和用于脂质组合物的化合物
WO2025147545A1 (en) 2024-01-03 2025-07-10 Juno Therapeutics, Inc. Lipid nanoparticles for delivery of nucleic acids and related methods and uses
WO2025157978A1 (en) * 2024-01-26 2025-07-31 Merck Patent Gmbh Ionizable lipids for use in lipid nanoparticles

Family Cites Families (67)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3288774A (en) * 1963-12-03 1966-11-29 Universal Oil Prod Co Reaction product of substituted-aminoalkyl alkanolamine and polycarboxylic acid or the like
SE467861B (sv) 1987-12-18 1992-09-28 Kabi Pharmacia Ab Emulsion foer parenteral administrering
US5157309A (en) 1990-09-13 1992-10-20 Motorola Inc. Cold-cathode field emission device employing a current source means
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US6469158B1 (en) 1992-05-14 2002-10-22 Ribozyme Pharmaceuticals, Incorporated Synthesis, deprotection, analysis and purification of RNA and ribozymes
US5804683A (en) 1992-05-14 1998-09-08 Ribozyme Pharmaceuticals, Inc. Deprotection of RNA with alkylamine
US5977343A (en) 1992-05-14 1999-11-02 Ribozyme Pharmaceuticals, Inc. Synthesis, deprotection, analysis and purification of RNA and ribozymes
FR2694559B1 (fr) * 1992-08-06 1994-10-28 Atta Nouveaux dérivés amphiphiles d'aminoacides ou de peptides, leur procédé de préparation et leur utilisation dans des préparations à usage biomédical.
DE69431669T2 (de) 1993-09-02 2003-10-23 Ribozyme Pharmaceuticals, Inc. Enzymatische nukleiksaüre die nicht-nukleotide enthaltet
US5534499A (en) 1994-05-19 1996-07-09 The University Of British Columbia Lipophilic drug derivatives for use in liposomes
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5889136A (en) 1995-06-09 1999-03-30 The Regents Of The University Of Colorado Orthoester protecting groups in RNA synthesis
DE19521412A1 (de) * 1995-06-14 1996-12-19 Boehringer Mannheim Gmbh Neue kationische und polykationische Amphiphile, diese enthaltende Reagenzien und deren Verwendung
JPH11510489A (ja) * 1995-07-21 1999-09-14 ジンタ・インコーポレイテッド 新規アミド基本カチオン性脂質
US5744335A (en) * 1995-09-19 1998-04-28 Mirus Corporation Process of transfecting a cell with a polynucleotide mixed with an amphipathic compound and a DNA-binding protein
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
DE19631189A1 (de) * 1996-08-02 1998-02-05 Max Delbrueck Centrum Neuartige kationische Amphiphile für den liposomalen Gentransfer
AR013269A1 (es) 1997-08-04 2000-12-13 Scras Producto que contiene por lo menos un rna de doble filamento combinado con por lo menos un agente anti-viral, para la utilizacion terapeutica en eltratamiento de una enfermedad viral, en especial de la hepatitis viral
US6121457A (en) * 1997-11-14 2000-09-19 Megabios Corporation Compositions and methods using novel substituted imidazolium lipids
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
US6320017B1 (en) 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
US6111086A (en) 1998-02-27 2000-08-29 Scaringe; Stephen A. Orthoester protecting groups
GB9827152D0 (en) 1998-07-03 1999-02-03 Devgen Nv Characterisation of gene function using double stranded rna inhibition
EP2314700A1 (en) 1999-01-28 2011-04-27 Medical College of Georgia Research Institute, Inc Composition and method for in vivo and in vitro attenuation of gene expression using double stranded RNA
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
HK1047109A1 (zh) 1999-10-15 2003-02-07 University Of Massachusetts 作为指定基因干预工具的rna干预轨迹基因
GB9927444D0 (en) 1999-11-19 2000-01-19 Cancer Res Campaign Tech Inhibiting gene expression
AU2001288337B2 (en) * 2000-08-19 2006-02-02 University Of Florida Functionalized polymers
CZ302719B6 (cs) 2000-12-01 2011-09-21 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Izolovaná molekula dvouretezcové RNA, zpusob její výroby a její použití
WO2002087541A1 (en) 2001-04-30 2002-11-07 Protiva Biotherapeutics Inc. Lipid-based formulations for gene transfer
US7407943B2 (en) 2001-08-01 2008-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein B expression
DK1527176T4 (en) 2002-08-05 2017-07-03 Silence Therapeutics Gmbh ADDITIONAL NEW FORMS OF INTERFERRING RNA MOLECULES
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
US8084599B2 (en) 2004-03-15 2011-12-27 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
US7303881B2 (en) * 2004-04-30 2007-12-04 Pds Biotechnology Corporation Antigen delivery compositions and methods of use
US7838685B2 (en) * 2005-04-27 2010-11-23 Shinji Takeoka Cationic amino acid type lipid
US7915230B2 (en) * 2005-05-17 2011-03-29 Molecular Transfer, Inc. Reagents for transfection of eukaryotic cells
CA2628300C (en) 2005-11-02 2018-04-17 Protiva Biotherapeutics, Inc. Modified sirna molecules and uses thereof
US20100184209A1 (en) 2006-02-17 2010-07-22 Dharmacon, Inc. Compositions and methods for inhibiting gene silencing by rna interference
EP1844772A1 (en) * 2006-04-06 2007-10-17 Polyplus-Transfection SA Compositions for transfection of oligonucleotides active for gene silencing and their biological and therapeutical applications
NZ572564A (en) * 2006-05-05 2011-10-28 Molecular Transfer Inc Novel reagents for transfection of eukaryotic cells
WO2009129385A1 (en) * 2008-04-16 2009-10-22 Abbott Laboratories Cationic lipids and uses thereof
US20100055168A1 (en) * 2008-04-16 2010-03-04 Abbott Laboratories Cationic lipids and uses thereof
US20090285881A1 (en) * 2008-04-16 2009-11-19 Abbott Laboratories Cationic lipids and uses thereof
US20100063135A1 (en) * 2008-09-10 2010-03-11 Abbott Laboratories Polyethylene glycol lipid conjugates and uses thereof
WO2010030730A1 (en) * 2008-09-10 2010-03-18 Abbott Laboratories Polyethylene glycol lipid conjugates and uses thereof
US20100331389A1 (en) 2008-09-22 2010-12-30 Bob Dale Brown Compositions and methods for the specific inhibition of gene expression by dsRNA containing modified nucleotides
EA037404B1 (ru) * 2008-11-10 2021-03-24 Арбутус Биофарма Корпорэйшн Липиды и композиции для доставки лекарственных средств
US8513207B2 (en) 2008-12-18 2013-08-20 Dicerna Pharmaceuticals, Inc. Extended dicer substrate agents and methods for the specific inhibition of gene expression
WO2010093788A2 (en) 2009-02-11 2010-08-19 Dicerna Pharmaceuticals, Inc. Multiplex dicer substrate rna interference molecules having joining sequences
EP2756845B1 (en) 2009-04-03 2017-03-15 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of KRAS by asymmetric double-stranded RNA
WO2010115202A2 (en) 2009-04-03 2010-10-07 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of kras by blunt ended double-stranded rna
ES2921573T3 (es) 2009-04-03 2022-08-29 Dicerna Pharmaceuticals Inc Métodos y composiciones para la inhibición específica de KRAS por RNA bicatenario asimétrico
CN102497870A (zh) 2009-06-03 2012-06-13 戴瑟纳制药公司 肽-dicer底物试剂及其特异性抑制基因表达的方法
US20100310623A1 (en) 2009-06-05 2010-12-09 Laurencin Cato T Synergetic functionalized spiral-in-tubular bone scaffolds
WO2010141933A1 (en) 2009-06-05 2010-12-09 Dicerna Pharmaceuticals, Inc. Specific inhibition of gene expression by nucleic acid containing a dicer substrate
US9181295B2 (en) * 2009-08-20 2015-11-10 Sirna Therapeutics, Inc. Cationic lipids with various head groups for oligonucleotide delivery
WO2011072292A2 (en) 2009-12-11 2011-06-16 Dicerna Pharmaceuticals, Inc. Phase changing formulations of rna and rna derivatives
EP3494963A1 (en) * 2009-12-18 2019-06-12 The University of British Columbia Methods and compositions for delivery of nucleic acids
EP2525781A1 (en) * 2010-01-22 2012-11-28 Schering Corporation Novel cationic lipids for oligonucleotide delivery
WO2011109294A1 (en) 2010-03-01 2011-09-09 Dicerna Pharmaceuticals, Inc. Lipid delivery formulations
EP2569276B1 (en) * 2010-05-12 2021-02-24 Arbutus Biopharma Corporation Novel cationic lipids and methods of use thereof
WO2012000104A1 (en) * 2010-06-30 2012-01-05 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
EP2768958B1 (en) * 2011-10-18 2019-08-14 Dicerna Pharmaceuticals, Inc. Amine cationic lipids and uses thereof

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