The present disclosure relates to the field of medicine, specifically to a long-acting spleen-targeting cationic lipid compound comprising a benzene ring structure, a composition comprising the same, and a use thereof. More specifically, the present disclosure provides a compound of formula (I), or an N-oxide thereof, a solvate thereof, a pharmaceutically acceptable salt thereof, or a stereoisomer thereof. The present disclosure further provides a composition comprising the aforementioned compound and a use thereof in delivering therapeutic or prophylactic agents.
The present disclosure relates to the field of medicine, specifically to a long-acting spleen-targeting cationic lipid compound comprising a benzene ring structure, a composition comprising the same, and a use thereof. More specifically, the present disclosure provides a compound of formula (I), or an N-oxide thereof, a solvate thereof, a pharmaceutically acceptable salt thereof, or a stereoisomer thereof. The present disclosure further provides a composition comprising the aforementioned compound and a use thereof in delivering therapeutic or prophylactic agents.
C07C 229/14 - 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 carbon skeletons containing rings
A61K 39/385 - Haptens or antigens, bound to carriers
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
2.
LIPID COMPOSITION TARGETING ANTIGEN-PRESENTING CELLS AND USE THEREOF
The present disclosure relates to the technical field of molecular biology and provides a lipid composition targeting antigen-presenting cells and a use thereof. The composition provided by the present disclosure demonstrates good organ and cell targeting specificity, significantly enhancing the protein expression level of antigens in the spleen. Additionally, the composition significantly increases the percentage of cells that express the antigen among antigen-presenting cells (e.g., B cells, pDC cells, cDC cells, and macrophages) in the spleen, indicating that the composition can be used in the immunotherapy of diseases.
A61K 39/39 - Medicinal preparations containing antigens or antibodies characterised by the immunostimulating additives, e.g. chemical adjuvants
A61K 39/00 - Medicinal preparations containing antigens or antibodies
A61K 47/54 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
3.
EXTRAHEPATIC TARGETED CATIONIC LIPID COMPOUND WITH HIGH EFFICIENCY AND LOW TOXICITY AND ITS COMPOSITION
Provided in the present disclosure is a compound of formula (I), or an N-oxide, solvate, pharmaceutically acceptable salt or stereoisomer thereof, which is an extrahepatic targeted cationic lipid compound with high efficiency and low toxicity. Further provided are a composition containing the aforementioned compound, and the use thereof in the delivery of a therapeutic or prophylactic agent.
Provided in the present disclosure is a compound of formula (I), or an N-oxide, solvate, pharmaceutically acceptable salt or stereoisomer thereof, which is an extrahepatic targeted cationic lipid compound with high efficiency and low toxicity. Further provided are a composition containing the aforementioned compound, and the use thereof in the delivery of a therapeutic or prophylactic agent.
C07C 229/16 - 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 hydrocarbon radicals substituted by amino or carboxyl groups, e.g. ethylenediamine-tetra-acetic acid, iminodiacetic acids
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
A61K 47/18 - AminesAmidesUreasQuaternary ammonium compoundsAmino acidsOligopeptides having up to five amino acids
4.
EXTRAHEPATIC TARGETED HIGH-EFFICIENCY AND LOW-TOXICITY CATIONIC LIPID COMPOUND AND COMPOSITION THEREOF
The present disclosure provides a compound of formula (I) or an N-oxide thereof, a solvate thereof, a pharmaceutically acceptable salt thereof, or a stereoisomer thereof, which is an extrahepatic targeted high-efficiency and low-toxicity cationic lipid compound. Further provided are compositions comprising the described compound and use thereof for the delivery of therapeutic or prophylactic agents.
C07C 229/16 - 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 hydrocarbon radicals substituted by amino or carboxyl groups, e.g. ethylenediamine-tetra-acetic acid, iminodiacetic acids
C07C 219/06 - 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 acyclic and saturated having the hydroxy groups esterified by carboxylic acids having the esterifying carboxyl groups bound to hydrogen atoms or to acyclic carbon atoms of an acyclic saturated carbon skeleton
A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection
A61P 3/10 - Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
A61P 9/00 - Drugs for disorders of the cardiovascular system
A61P 13/12 - Drugs for disorders of the urinary system of the kidneys
A61P 11/00 - Drugs for disorders of the respiratory system
A61P 31/22 - Antivirals for DNA viruses for herpes viruses
A61K 47/18 - AminesAmidesUreasQuaternary ammonium compoundsAmino acidsOligopeptides having up to five amino acids
A61K 47/24 - Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
A61K 47/28 - Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
Provided in the present invention are long-acting low-toxicity novel cationic lipid compounds, which are compounds shown as formula (I), or N-oxides, solvates, pharmaceutically acceptable salts or stereoisomers thereof. Further provided are a composition comprising the compounds and the use thereof for the delivery of therapeutic or prophylactic agents.
C07C 229/16 - 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 hydrocarbon radicals substituted by amino or carboxyl groups, e.g. ethylenediamine-tetra-acetic acid, iminodiacetic acids
A61P 31/00 - Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
A61P 37/00 - Drugs for immunological or allergic disorders
A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection
A61P 3/10 - Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
A61P 9/00 - Drugs for disorders of the cardiovascular system
A61P 13/12 - Drugs for disorders of the urinary system of the kidneys
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
Provided in the present invention is a compound of formula (I) or an N-oxide, a solvate, a pharmaceutically acceptable salt or a stereoisomer thereof, which compound is an ionizable cationic lipid compound. Further provided are a composition comprising the aforementioned compound and the use thereof in the delivery of a therapeutic agent or a prophylactic agent.
C07C 229/16 - 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 hydrocarbon radicals substituted by amino or carboxyl groups, e.g. ethylenediamine-tetra-acetic acid, iminodiacetic acids
C07C 227/08 - Formation of amino groups in compounds containing carboxyl groups by addition or substitution reactions, without increasing the number of carbon atoms in the carbon skeleton of the acid by reaction of ammonia or amines with acids containing functional groups
C07C 227/16 - Preparation of compounds containing amino and carboxyl groups bound to the same carbon skeleton from compounds containing already amino and carboxyl groups or derivatives thereof by reactions not involving the amino or carboxyl groups
C07C 329/06 - Esters of monothiocarbonic acids having sulfur atoms of thiocarbonic groups bound to acyclic carbon atoms
C07C 333/04 - Monothiocarbamic acidsDerivatives thereof having nitrogen atoms of thiocarbamic groups bound to hydrogen atoms or to acyclic carbon atoms
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
A61K 45/00 - Medicinal preparations containing active ingredients not provided for in groups
A61P 31/00 - Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
A61P 37/00 - Drugs for immunological or allergic disorders
A61P 43/00 - Drugs for specific purposes, not provided for in groups
A61P 3/10 - Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
A61P 9/00 - Drugs for disorders of the cardiovascular system
A61P 13/12 - Drugs for disorders of the urinary system of the kidneys
The present invention provides a cationic lipid compound with high efficiency, low toxicity and stable expression, which is a compound of formula (I) or an N-oxide, a solvate, a pharmaceutically acceptable salt or a stereoisomer thereof. Further provided are a composition comprising the described compound and a use thereof in delivering a therapeutic or prophylactic agent.
C07C 229/16 - 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 hydrocarbon radicals substituted by amino or carboxyl groups, e.g. ethylenediamine-tetra-acetic acid, iminodiacetic acids
C07C 229/12 - 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
C07C 323/12 - Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and singly-bound oxygen atoms bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated
C07C 67/08 - Preparation of carboxylic acid esters by reacting carboxylic acids or symmetrical anhydrides with the hydroxy or O-metal group of organic compounds
C07C 67/11 - Preparation of carboxylic acid esters by reacting carboxylic acids or symmetrical anhydrides with ester groups or with a carbon-halogen bond being mineral ester groups
C07C 69/63 - Halogen-containing esters of saturated acids
C07C 227/08 - Formation of amino groups in compounds containing carboxyl groups by addition or substitution reactions, without increasing the number of carbon atoms in the carbon skeleton of the acid by reaction of ammonia or amines with acids containing functional groups
C07C 213/02 - Preparation of compounds containing amino and hydroxy, amino and etherified hydroxy or amino and esterified hydroxy groups bound to the same carbon skeleton by reactions involving the formation of amino groups from compounds containing hydroxy groups or etherified or esterified hydroxy groups
C07C 219/06 - 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 acyclic and saturated having the hydroxy groups esterified by carboxylic acids having the esterifying carboxyl groups bound to hydrogen atoms or to acyclic carbon atoms of an acyclic saturated carbon skeleton
C07C 319/24 - Preparation of thiols, sulfides, hydropolysulfides or polysulfides of hydropolysulfides or polysulfides by reactions involving the formation of sulfur-to-sulfur bonds
C07C 319/22 - Preparation of thiols, sulfides, hydropolysulfides or polysulfides of hydropolysulfides or polysulfides
C07C 319/20 - Preparation of thiols, sulfides, hydropolysulfides or polysulfides of sulfides by reactions not involving the formation of sulfide groups