[go: up one dir, main page]

AR095077A1 - Lípidos y composiciones lípidas para la entrega de agentes activos - Google Patents

Lípidos y composiciones lípidas para la entrega de agentes activos

Info

Publication number
AR095077A1
AR095077A1 ARP140100778A ARP140100778A AR095077A1 AR 095077 A1 AR095077 A1 AR 095077A1 AR P140100778 A ARP140100778 A AR P140100778A AR P140100778 A ARP140100778 A AR P140100778A AR 095077 A1 AR095077 A1 AR 095077A1
Authority
AR
Argentina
Prior art keywords
optionally substituted
alkyl
independently
alkylene
alkynyl
Prior art date
Application number
ARP140100778A
Other languages
English (en)
Inventor
Byers Crystal
Lee Baryza Jeremy
Zhao Junping
Zabawa Thomas
Chuck Lee Cameron
Munn - Fazal Tanzina
Grant Gamber Gabriel
Eric John Beckwith Rohan
A Bowman Keith
Wang Shuangxi
West Laura
Vargeese Chandra
Bhanudasji Tichkule Ritesh
Original Assignee
Novartis Ag
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 Novartis Ag filed Critical Novartis Ag
Publication of AR095077A1 publication Critical patent/AR095077A1/es

Links

Classifications

    • 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
    • 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
    • A61K47/16—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
    • A61K47/183—Amino acids, e.g. glycine, EDTA or aspartame
    • 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
    • A61K9/127—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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C217/00—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton
    • C07C217/54—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups bound to carbon atoms of at least one six-membered aromatic ring and amino groups bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings of the same carbon skeleton
    • C07C217/56—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups bound to carbon atoms of at least one six-membered aromatic ring and amino groups bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings of the same carbon skeleton with amino groups linked to the six-membered aromatic ring, or to the condensed ring system containing that ring, by carbon chains not further substituted by singly-bound oxygen atoms
    • C07C217/58—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups bound to carbon atoms of at least one six-membered aromatic ring and amino groups bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings of the same carbon skeleton with amino groups linked to the six-membered aromatic ring, or to the condensed ring system containing that ring, by carbon chains not further substituted by singly-bound oxygen atoms with amino groups and the six-membered aromatic ring, or the condensed ring system containing that ring, bound to the same carbon atom of the carbon chain
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C217/00—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton
    • C07C217/54—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups bound to carbon atoms of at least one six-membered aromatic ring and amino groups bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings of the same carbon skeleton
    • C07C217/56—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups bound to carbon atoms of at least one six-membered aromatic ring and amino groups bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings of the same carbon skeleton with amino groups linked to the six-membered aromatic ring, or to the condensed ring system containing that ring, by carbon chains not further substituted by singly-bound oxygen atoms
    • C07C217/60—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups bound to carbon atoms of at least one six-membered aromatic ring and amino groups bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings of the same carbon skeleton with amino groups linked to the six-membered aromatic ring, or to the condensed ring system containing that ring, by carbon chains not further substituted by singly-bound oxygen atoms linked by carbon chains having two carbon atoms between the amino groups and the six-membered aromatic ring or the condensed ring system containing that ring
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C217/00—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton
    • C07C217/54—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups bound to carbon atoms of at least one six-membered aromatic ring and amino groups bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings of the same carbon skeleton
    • C07C217/56—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups bound to carbon atoms of at least one six-membered aromatic ring and amino groups bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings of the same carbon skeleton with amino groups linked to the six-membered aromatic ring, or to the condensed ring system containing that ring, by carbon chains not further substituted by singly-bound oxygen atoms
    • C07C217/62—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups bound to carbon atoms of at least one six-membered aromatic ring and amino groups bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings of the same carbon skeleton with amino groups linked to the six-membered aromatic ring, or to the condensed ring system containing that ring, by carbon chains not further substituted by singly-bound oxygen atoms linked by carbon chains having at least three carbon atoms between the amino groups and the six-membered aromatic ring or the condensed ring system containing that ring
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C219/00—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton
    • C07C219/02—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C219/20—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being unsaturated
    • C07C219/22—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being unsaturated and containing six-membered aromatic rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C219/00—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton
    • C07C219/26—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups bound to carbon atoms of at least one six-membered aromatic ring and amino groups bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings of the same carbon skeleton
    • C07C219/28—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups bound to carbon atoms of at least one six-membered aromatic ring and amino groups bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings of the same carbon skeleton having amino groups bound to acyclic carbon atoms of the carbon skeleton
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C229/00—Compounds containing amino and carboxyl groups bound to the same carbon skeleton
    • C07C229/02—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C229/04—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C229/06—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton
    • C07C229/10—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings
    • C07C229/12—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings to carbon atoms of acyclic carbon skeletons
    • 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
    • C07C235/46—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 having the nitrogen atoms of the carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • 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
    • C07C237/08—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 atom of at least one of the carboxamide groups bound to an acyclic carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D205/00—Heterocyclic compounds containing four-membered rings with one nitrogen atom as the only ring hetero atom
    • C07D205/02—Heterocyclic compounds containing four-membered rings with one nitrogen atom as the only ring hetero atom not condensed with other rings
    • C07D205/04—Heterocyclic compounds containing four-membered rings with one nitrogen atom as the only ring hetero atom not condensed with other rings having no double bonds between ring members or between ring members and non-ring members
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • 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
    • C07D213/62—Oxygen or sulfur atoms
    • C07D213/69—Two or more oxygen atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • 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
    • C07D213/78—Carbon atoms having three bonds to hetero atoms, with at the most one bond to halogen, e.g. ester or nitrile radicals
    • C07D213/79—Acids; Esters
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • 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
    • C07D295/04—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
    • C07D295/08—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 bound oxygen or sulfur atoms
    • C07D295/096—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 bound oxygen or sulfur atoms with the ring nitrogen atoms and the oxygen or sulfur atoms separated by carbocyclic rings or by carbon chains interrupted by carbocyclic rings
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D317/00—Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms
    • C07D317/08—Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms having the hetero atoms in positions 1 and 3
    • C07D317/10—Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms having the hetero atoms in positions 1 and 3 not condensed with other rings
    • C07D317/14—Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms having the hetero atoms in positions 1 and 3 not condensed with other rings with substituted hydrocarbon radicals attached to ring carbon atoms
    • C07D317/18—Radicals substituted by singly bound oxygen or sulfur atoms
    • C07D317/24—Radicals substituted by singly bound oxygen or sulfur atoms esterified
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D317/00—Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms
    • C07D317/08—Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms having the hetero atoms in positions 1 and 3
    • C07D317/10—Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms having the hetero atoms in positions 1 and 3 not condensed with other rings
    • C07D317/14—Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms having the hetero atoms in positions 1 and 3 not condensed with other rings with substituted hydrocarbon radicals attached to ring carbon atoms
    • C07D317/28—Radicals substituted by nitrogen atoms
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Dispersion Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)

Abstract

Los compuestos de la fórmula (1) y sales farmacéuticamente aceptables de los mismos son los lípidos catiónicos útiles en la administración de agentes biológicamente activos a las células y tejidos. Reivindicación 1: Un compuesto según la fórmula (1), en donde: L es alquileno C₁₋₆, alquenileno C₂₋₆, alquinileno C₂₋₆, -(CH₂)ʳ-cicloalquileno C₃₋₇-(CH₂)ₛ-, -(CH₂)ₛ-cicloalquenileno C₃₋₇-(CH₂)ₛ-, -(CH₂)ₛ-cicloalquinileno C₃₋₇-(CH₂)ₛ-, *-alquileno C₁₋₄-L²-, *-alquileno C₁₋₄-L²-alquileno C₁₋₄-, un resto de fórmula (2) o un resto de fórmula (3), en donde el * denota unión de la fracción al grupo NR¹R²; L², fijado en cualquier dirección, es -O-, -S-, -C(O)-, -C(O)O-, -OC(O)O-, -CONH-, S(O)₂NH-, NHCONH- o -NHCSNH-; cada s es independientemente 0,1 ó 2; cada t es independientemente 0, 1, 2, 3, ó 4; u es 0, 1, 2, 3, 4, 5 ó 6; R¹ y R² son cada uno independientemente alquilo C₁₋₆ opcionalmente sustituido, alquenilo C₂₋₆ opcionalmente sustituido, alquinilo C₂₋₆ opcionalmente sustituido, cicloalquilo C₃₋₇-(CH₂)ₛ- opcionalmente sustituido, cicloalquenilo C₃₋₇-(CH₂)ₛ- opcionalmente sustituido, cicloalquinilo C₃₋₇-(CH₂)ₛ- opcionalmente sustituido, o fenilo-(CH₂)ₛ- opcionalmente sustituido; en donde dicho alquilo C₁₋₆, alquenilo C₂₋₆, alquinilo C₂₋₆, cicloalquilo C₃₋₇, cicloalquenilo C₃₋₇, cicloalquinilo C₃₋₇, y fenilo son opcionalmente sustituidos por uno o dos sustituyentes cada independientemente seleccionado del grupo que consta de: OH, alcoxilo C₁₋₃, COOH, y COO-alquilo C₁₋₄, o R¹ y R² se juntan formando un anillo heterocíclico opcionalmente sustituido de 4 - 12 miembros, dicho anillo heterocíclico es opcionalmente sustituido por uno a tres sustituyentes cada independientemente seleccionado del grupo que consta de: OH, halo, alquilo C₁₋₃, alcoxilo C₁₋₃, dimetilamino, -COO-alquilo C₁₋₄, fenilo, piperidinilo, y morfolinilo; R³ y R⁴ son cada uno independientemente: (a) -Z¹-Rᵃ, (b) -Z¹-Rᵇ-Z²-Rᵃ, (c) -Z¹-Rᵇ-Z²-Rᵇ-Z³-Rᵃ, (d) -Z¹-Rᵇ-Z²-Rᵇ-Z³-Rᵇ-Z⁴-Rᵃ, (e) -Rᵇ-Z¹-Rᵃ, (f) -Rᵇ-Z¹-Rᵇ-Z²-Rᵃ, (g) -Rᵇ-Z¹-Rᵇ-Z²-Rᵇ-Z³-Rᵃ, (h) -Rᵇ-Z¹-Rᵇ-Z²-Rᵇ-Z³-Rᵇ-Z⁴-Rᵃ, (i) -Rᶜ, (j) -Z¹-Rᵇ-Rᶜ, o (k) -Rᵇ-Z¹-Rᵇ-Rᶜ; en donde Z¹, Z², Z³, y Z⁴, fijado en cualquier dirección, son cada uno independientemente -O-, -C(O)O-, -OC(O)O-, o -CONH-; Rᵃ es alquilo C₂₋₂₂, alquenilo C₂₋₂₂, o alquinilo C₂₋₂₂; cada Rᵇ es independientemente alquileno C₁₋₂₀, alquenileno C₂₋₂₀, o alquinileno C₂₋₂₀; Rᶜ es un compuesto del grupo de formulas (4); R es alquilo C₅₋₂₂, alquenilo C₅₋₂₂, o alquinilo C₅₋₂₂; n es 0 - 12; m, p, y q son cada uno independientemente 0, 1, 2, 3 ó 4; siempre que las cadenas (a) - (h) tengan 12 - 30 átomos de carbono y las cadenas (i) - (k) tenga 12 - 70 átomos de carbono; X es CR⁶ o N; y R⁶ es H, halo, alquilo C₁₋₆, o R⁴; o una sal farmacéuticamente aceptable del mismo.
ARP140100778A 2013-03-08 2014-03-10 Lípidos y composiciones lípidas para la entrega de agentes activos AR095077A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201361774759P 2013-03-08 2013-03-08

Publications (1)

Publication Number Publication Date
AR095077A1 true AR095077A1 (es) 2015-09-16

Family

ID=51654891

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP140100778A AR095077A1 (es) 2013-03-08 2014-03-10 Lípidos y composiciones lípidas para la entrega de agentes activos

Country Status (2)

Country Link
US (3) US9504747B2 (es)
AR (1) AR095077A1 (es)

Families Citing this family (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2557382T3 (es) 2010-07-06 2016-01-25 Glaxosmithkline Biologicals Sa Liposomas con lípidos que tienen un valor de pKa ventajoso para el suministro de ARN
CA2804492A1 (en) 2010-07-06 2012-01-12 Novartis Ag Immunisation of large mammals with low doses of rna
US9801897B2 (en) 2010-07-06 2017-10-31 Glaxosmithkline Biologicals Sa Delivery of RNA to trigger multiple immune pathways
EP4226941B1 (en) 2010-08-31 2024-10-23 GlaxoSmithKline Biologicals SA Pegylated liposomes for delivery of immunogen-encoding rna
KR102162111B1 (ko) 2010-10-11 2020-10-07 노파르티스 아게 항원 전달 플랫폼
EP3332802A1 (en) * 2011-07-06 2018-06-13 GlaxoSmithKline Biologicals SA Immunogenic combination compositions and uses thereof
CA2897752A1 (en) * 2013-01-10 2014-07-17 Novartis Ag Influenza virus immunogenic compositions and uses thereof
JP2016511256A (ja) 2013-02-28 2016-04-14 アローヘッド リサーチ コーポレイション Ｅｐａｓ１関連疾患を治療するための有機組成物
AU2014224205C1 (en) 2013-03-08 2019-04-04 Novartis Ag Lipids and lipid compositions for the delivery of active agents
EP3052627B1 (en) 2013-10-04 2018-08-22 Novartis AG Novel formats for organic compounds for use in rna interference
EP3052107B1 (en) 2013-10-04 2018-05-02 Novartis AG Organic compounds to treat hepatitis b virus
US10227588B2 (en) 2013-10-04 2019-03-12 Novartis Ag 3′end caps for RNAi agents for use in RNA interference
EP4223285A3 (en) 2014-07-16 2023-11-22 Novartis AG Method of encapsulating a nucleic acid in a lipid nanoparticle host
ES2969956T3 (es) * 2014-09-05 2024-05-23 Novartis Ag Lípidos y composiciones lipídicas para el suministro de agentes activos
KR102630720B1 (ko) 2015-08-28 2024-01-29 세키스이 메디칼 가부시키가이샤 벤질 화합물
JP2019511255A (ja) 2016-01-11 2019-04-25 バーンダリ，インク． マイクロニードル組成物およびそれを使用する方法
EP3419959A4 (en) * 2016-02-23 2019-10-23 Carnot, LLC COMBINATION THERAPY
JP2019528312A (ja) * 2016-08-07 2019-10-10 ノバルティス アーゲー ｍＲＮＡ媒介性の免疫化方法
IL267034B2 (en) 2016-12-14 2025-03-01 Ligandal Inc Methods and compositions for nucleic acid and protein payload delivery
US12221610B2 (en) 2019-01-09 2025-02-11 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of HIF-2 alpha (EPAS1), compositions thereof, and methods of use
CA3143327A1 (en) 2019-06-13 2020-12-17 J. Keith Joung Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells
JP2023535726A (ja) 2020-07-24 2023-08-21 ザ ジェネラル ホスピタル コーポレイション 増強ウイルス様粒子および細胞への送達のためのその使用方法
JP2025519070A (ja) 2022-05-17 2025-06-24 エンヴェロップ セラピューティクス， インコーポレイテッド 効率的ｉｎ ｖｉｖｏ送達のための組成物および方法
CN121311221A (zh) * 2023-04-07 2026-01-09 旗舰实验室114股份有限公司 用于治疗性脂质纳米颗粒 (lnp)递送的脂质和脂质样化合物
US12311033B2 (en) 2023-05-31 2025-05-27 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE3144150A1 (de) 1981-04-10 1982-12-09 Basf Ag, 6700 Ludwigshafen (omega)-cyan-1,(omega)-diphenyl-azaalkan-derivate, ihre herstellung und diese enthaltende arzneimittel
DE3542994A1 (de) 1985-12-05 1987-06-11 Basf Ag Basisch substituierte phenylacetaldehyde, ihre herstellung und diese enthaltende arzneimittel
US20040006061A1 (en) 2001-10-04 2004-01-08 Wolfgang Haap Alkoxybenzylamine
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
CN101405240B (zh) 2006-03-24 2012-07-25 日商·Jitsubo株式会社 有机合成用试剂、以及使用该试剂的有机合成反应方法
AU2008219165A1 (en) 2007-02-16 2008-08-28 Merck Sharp & Dohme Corp. Compositions and methods for potentiated activity of biologicaly active molecules
WO2010021865A1 (en) * 2008-08-18 2010-02-25 Merck Sharp & Dohme Corp. Novel lipid nanoparticles and novel components for delivery of nucleic acids
US20120004310A1 (en) 2010-05-28 2012-01-05 Longo Frank M Non-peptide bdnf neurotrophin mimetics

Also Published As

Publication number Publication date
US20170196979A1 (en) 2017-07-13
US10729775B2 (en) 2020-08-04
US20140303232A1 (en) 2014-10-09
US9504747B2 (en) 2016-11-29
US20180296677A1 (en) 2018-10-18

Similar Documents

Publication Publication Date Title
AR095077A1 (es) Lípidos y composiciones lípidas para la entrega de agentes activos
AR121016A2 (es) Sales de fenotiazindiaminio y su uso
AR093038A1 (es) Conjugados de suministro de farmacos que contienen aminoacidos no naturales y metodos para usarlos, composicion y uso
AR094668A1 (es) Amidas como moduladores de canales de sodio
PE20190437A1 (es) Procesos para la preparacion de (s)-n-(5-((r)-2-(2,5-difluorofenil)pirrolidin-1-il)-pirazolo[1,5-a]pirimidin-3-il)-3-hidroxipirrolidin-1-carboxamida y sales del mismo
MX377255B (es) Lipidos y composiciones de lipidos para el suministro de agentes activos.
AR090292A1 (es) Compuestos basados en pirazolo[1,5-a]pirimidina, composiciones que los comprenden y metodos que los utilizan
AR102361A1 (es) Compuestos de metil-quinolina útiles para inhibir la sintasa-1 de prostaglandina microsomal e2
AR094990A1 (es) Derivados de amida y su uso para el tratamiento de infección por vih
AR090590A1 (es) COMPUESTOS 1,3,4-OXADIAZOL Y 1,3,4-TIADIAZOL COMO INHIBIDORES DE b-LACTAMASA
AR092538A1 (es) Inhibidores de girasa triciclica
AR094410A1 (es) Compuestos terapéuticos
AR091156A1 (es) Nucleosidos de espirooxetano de uracilo
AR090847A1 (es) Compuestos moduladores del receptor huerfano retinoideo (ror)
AR088175A1 (es) 3-pirimidin-4-il-oxazolidin-2-onas utiles para tratar cancer y composiciones farmaceuticas que las contienen
AR110139A1 (es) COMPUESTOS MONO Y ESPIROCÍCLICOS QUE CONTIENEN CICLOBUTANO Y AZETIDINA COMO INHIBIDORES DE LA INTEGRINA aV
CY1118739T1 (el) Ενωσεις αμφι(φθoρoαλκυλ)-1,4-βενζoδιαζεπινoνης ως αναστολεις toy notch
AR078278A1 (es) Antagonistas de la tiazol y oxazol hepcidina, composiciones farmaceuticas que los contienen y uso de los mismos para el tratamiento de anemias y enfermedades asociadas a deficiencias de hierro.
UY33500A (es) Derivados de acil sulfonamida y sus sales farmacéuticamente aceptables para uso en medicina
UY33501A (es) Derivados de acil sulfonamida y sus sales farmacéuticamente aceptables para uso en medicina
AR093532A1 (es) Compuestos y composiciones para el tratamiento de enfermedades parasitarias
AR088748A1 (es) Macrociclos como inhibidores del factor xia
AR103251A1 (es) Compuestos policíclicos de carbamoilpiridona y su uso farmacéutico
AR088226A1 (es) Derivados piperidinicos heterociclicos, composiciones farmaceuticas que los contienen y uso de los mismos para el tratamiento de enfermedades neurodegenerativas
AR088810A1 (es) Moduladores del receptor mas acoplado a la proteina g utiles en el tratamiento de trastornos cardiovasculares y composiciones farmaceuticas que los contienen

Legal Events

Date Code Title Description
FB Suspension of granting procedure