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—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated 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—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
    • 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 N.A.
    • 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)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • General Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Dispersion Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (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 (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012006369A2 (en) 2010-07-06 2012-01-12 Novartis Ag Immunisation of large mammals with low doses of rna
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
HUE047796T2 (hu) 2010-07-06 2020-05-28 Glaxosmithkline Biologicals Sa RNS bevitele több immunútvonal bekapcsolására
DK4066855T3 (da) 2010-08-31 2023-02-20 Glaxosmithkline Biologicals Sa Pegylerede liposomer til forsyning af RNA, der koder for immunogen
TR201903651T4 (tr) 2010-10-11 2019-04-22 Glaxosmithkline Biologicals Sa Antijen uygulama platformları.
US11896636B2 (en) * 2011-07-06 2024-02-13 Glaxosmithkline Biologicals Sa Immunogenic combination compositions and uses thereof
MX2015008847A (es) * 2013-01-10 2015-10-30 Novartis Ag Composiciones inmunogenicas de virus de influenza y usos de las mismas.
SG10201706960TA (en) 2013-02-28 2017-10-30 Arrowhead Res Corp Organic compositions to treat epas1-related diseases
JP6352950B2 (ja) 2013-03-08 2018-07-04 ノバルティス アーゲー 活性薬物の送達のための脂質と脂質組成物
US9988627B2 (en) 2013-10-04 2018-06-05 Novartis Ag Formats for organic compounds for use in RNA interference
CN105792832B (zh) 2013-10-04 2021-03-23 诺华股份有限公司 用于治疗乙肝病毒的有机化合物
EP3052464B1 (en) 2013-10-04 2020-04-15 Novartis AG 3'end caps for rna-interferring agents for use in rna interference
EP3169309B1 (en) 2014-07-16 2023-05-10 Novartis AG Method of encapsulating a nucleic acid in a lipid nanoparticle host
WO2016037053A1 (en) * 2014-09-05 2016-03-10 Novartis Ag Lipids and lipid compositions for the delivery of active agents
JP6116782B1 (ja) 2015-08-28 2017-04-19 積水メディカル株式会社 ベンジル化合物
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
US11878060B2 (en) * 2016-08-07 2024-01-23 Novartis Ag mRNA-mediated immunization methods
WO2018112282A1 (en) 2016-12-14 2018-06-21 Ligandal, Inc. Compositions and methods for nucleic acid and/or protein payload delivery

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
ES2546808T3 (es) 2006-03-24 2015-09-28 Jitsubo Co., Ltd. Reactivo para síntesis orgánica y método de reacción de síntesis orgánica con dicho reactivo
AU2008219165A1 (en) 2007-02-16 2008-08-28 Merck Sharp & Dohme Corp. Compositions and methods for potentiated activity of biologicaly active molecules
EP2326331A4 (en) * 2008-08-18 2013-05-15 Merck Sharp & Dohme NOVEL LIPID NANOPARTICLES AND NEW COMPONENTS FOR NUCLEIC ACID ADMINISTRATION
WO2011150347A2 (en) 2010-05-28 2011-12-01 Pharmatrophix Non-peptide bdnf neurotrophin mimetics

Also Published As

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

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
AR120080A1 (es) Compuestos lipídicos de cola ramificada y composiciones para la administración intracelular de agentes terapéuticos
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
AR106865A1 (es) Piridinas sustituidas y métodos de uso
AR090292A1 (es) Compuestos basados en pirazolo[1,5-a]pirimidina, composiciones que los comprenden y metodos que los utilizan
AR110139A1 (es) COMPUESTOS MONO Y ESPIROCÍCLICOS QUE CONTIENEN CICLOBUTANO Y AZETIDINA COMO INHIBIDORES DE LA INTEGRINA aV
EA201591662A1 (ru) Липиды и липидные композиции для доставки активных агентов
AR094410A1 (es) Compuestos terapéuticos
AR090847A1 (es) Compuestos moduladores del receptor huerfano retinoideo (ror)
AR091156A1 (es) Nucleosidos de espirooxetano de uracilo
AR088175A1 (es) 3-pirimidin-4-il-oxazolidin-2-onas utiles para tratar cancer y composiciones farmaceuticas que las contienen
AR091981A1 (es) Dihidropiridona p1 como inhibidores del factor xia
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
AR092538A1 (es) Inhibidores de girasa triciclica
AR103251A1 (es) Compuestos policíclicos de carbamoilpiridona y su uso farmacéutico
AR093532A1 (es) Compuestos y composiciones para el tratamiento de enfermedades parasitarias
AR091093A1 (es) Tienopirimidinas
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.
AR088810A1 (es) Moduladores del receptor mas acoplado a la proteina g utiles en el tratamiento de trastornos cardiovasculares y composiciones farmaceuticas que los contienen
AR091939A1 (es) 4-metil-2,3,5,9,9b-pentaaza-ciclopenta[a]naftalenos
AR097545A1 (es) COMPUESTOS A BASE DE PIRAZOLO[1,5-a]PIRIMIDINA, COMPOSICIONES QUE LOS COMPRENDEN Y SUS MÉTODOS DE USO
AR098522A1 (es) Compuesto de triazolo-piridina
AR107054A1 (es) DERIVADOS DE ISOINDOL Y COMPOSICIONES FARMACÉUTICAS QUE LOS COMPRENDEN PARA EL TRATAMIENTO DE ENFERMEDADES MEDIADAS POR RORg Y RORgt

Legal Events

Date Code Title Description
FB Suspension of granting procedure