US2008075763A1PendingUtilityA1

Polycationic sterol derivatives as transfection agents

56
Assignee: IMPERIAL COLLEGEPriority: May 24, 1996Filed: Oct 5, 2007Published: Mar 27, 2008
Est. expiryMay 24, 2016(expired)· nominal 20-yr term from priority
Inventors:Andrew MillerRobert CooperChristopher Etheridge
A01K 2207/05A01K 2267/0393A61K 9/1272C07J 9/00A61K 31/711A61K 31/56C07J 41/0055A61P 43/00A01K 2227/105
56
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Claims

Abstract

A method for treating genetic disorders or conditions or diseases in patients in need of such treatments. The method includes administering an effective amount of a compound comprising a cholesterol group or derivative thereof having linked thereto a head group. The head group is more positive than the heat group of DC-Chol. The heat group may be a straight chain polymeric group having two or more amine groups separated by an ethylene group.

Claims

exact text as granted — not AI-modified
1 . A compound capable of acting as a cationic lipid, the compound comprising a cholesterol group having linked thereto a head group; and wherein the head troup is more positive than the head group of DC-Chol; but wherein the compound is not synthesised by reacting spermidine and cholesterol chloroformate in CH 2 Cl 2  in the presence of N,N-diisoprophylethylamine.  
     
     
         2 - 22 . (canceled)  
     
     
         23 . A method for treating a genetic disorder, or condition or disease in a patient in need of treatment, comprising: 
 administering an effective amount of a compound comprising a cholesterol group or derivative thereof having linked thereto a head group; wherein the head group is more positive than the head group of DC-Chol; further wherein the head group is a straight chain polyamine; further wherein two or more of the amine groups of the polyamine group are separated by an ethylene group.    
     
     
         24 . The method according to  claim 23  wherein the cholesterol group or derivative thereof is cholesterol.  
     
     
         25 . The method according to  claim 23  wherein the cholesterol group is linked to the head group via a carbamoyl linkage.  
     
     
         26 . The method according to  claim 23  wherein the compound is selected from compounds of the formula  
       
         
           
           
               
               
           
         
         where Chol denotes a group of the formula  
         
           
             
             
                 
                 
             
           
         
       
     
     
         27 . The method according to  claim 23  wherein the compound is selected from compounds of the formula  
       
         
           
           
               
               
           
         
       
     
     
         28 . The method according to  claim 23  wherein the compound is selected from compounds of the formula  
       
         
           
           
               
               
           
         
       
     
     
         29 . The method according to  claim 23  wherein the compound is of the formula  
       
         
           
           
               
               
           
         
       
     
     
         30 . The method according to  claim 23  wherein the compound is of the formula  
       
         
           
           
               
               
           
         
       
     
     
         31 . The method according to  claim 23  wherein the compound is of the formula  
       
         
           
           
               
               
           
         
       
     
     
         32 . The method according to  claim 23 , wherein the compound is a cationic lipid compound.  
     
     
         33 . The method according to  claim 32 , wherein the cationic lipid compound is in admixture with or associated with a nucleotide sequence.  
     
     
         34 . The method according to  claim 23 , wherein the compound is a cationic liposome formed from a cationic lipid compound.  
     
     
         35 . The method according to  claim 34 , wherein the cationic liposome is in admixture with or associated with a nucleotide sequence.  
     
     
         36 . A method for treating a genetic disorder, or condition or disease in a patient in need of treatment, comprising: 
 administering an effective amount of a compound selected from the group consisting of cationic lipid compounds, cationic liposomes formed from cationic lipid compounds, cationic lipid compounds in admixture with or associated with a nucleotide sequence, cationic liposomes, formed from a cationic lipid compound, in admixture with or associated with a nucleotide sequence, and combinations thereof,    the compound comprising a cholesterol group or derivative thereof having linked thereto a head group; wherein the head group is more positive than the head group of DC-Choi; further wherein the head group is a straight chain polyamine; further wherein two or more of the amine groups of the polyamine group are separated by an ethylene group.    
     
     
         37 . The method according to  claim 36  wherein the cholesterol group or derivative thereof is cholesterol.  
     
     
         38 . The method according to  claim 36  wherein the cholesterol group is linked to the head group via a carbamoyl linkage.  
     
     
         39 . The method according to  claim 36  wherein the compound is selected from compounds of the formula  
       
         
           
           
               
               
           
         
         where Chol denotes a group of the formula  
         
           
             
             
                 
                 
             
           
         
       
     
     
         40 . The method according to  claim 36  wherein the compound is selected from compounds of the formula  
       
         
           
           
               
               
           
         
       
     
     
         41 . The method according to  claim 36  wherein the compound is selected from compounds of the formula  
       
         
           
           
               
               
           
         
       
     
     
         42 . The method according to  claim 36  wherein the compound is of the formula  
       
         
           
           
               
               
           
         
       
     
     
         43 . The method according to  claim 36  wherein the compound is of the formula  
       
         
           
           
               
               
           
         
       
     
     
         44 . The method according to  claim 36  wherein the compound is of the formula  
       
         
           
           
               
               
           
         
       
     
     
         45 . A method for treating a genetic disorder, or condition or disease in a patient in need of treatment, comprising: 
 administering an effective amount of a composition, the composition comprising: 
 i. a compound selected from the group consisting of cationic lipid compounds, cationic liposomes formed from a cationic lipid compound, and combinations thereof, the compound comprising a cholesterol group or derivative thereof having linked thereto a head group; wherein the head group is more positive than the head group of DC-Chol; further wherein the head group is a straight chain polyamine; further wherein two or more of the amine groups of the polyamine group are separated by an ethylene group, and  
 ii. a pharmaceutical, and optionally a pharmaceutically acceptable diluent, carrier or excipient.  
   
     
     
         46 . The method according to  claim 45  wherein the cholesterol group or derivative thereof is cholesterol.  
     
     
         47 . The method according to  claim 45  wherein the cholesterol group is linked to the head group via a carbamoyl linkage.  
     
     
         48 . The method according to  claim 45  wherein the compound is selected from compounds of the formula  
       
         
           
           
               
               
           
         
         where Chol denotes a group of the formula  
         
           
             
             
                 
                 
             
           
         
       
     
     
         49 . The method according to  claim 45  wherein the compound is selected from compounds of the formula  
       
         
           
           
               
               
           
         
       
     
     
         50 . The method according to  claim 45  wherein the compound is selected from compounds of the formula  
       
         
           
           
               
               
           
         
       
     
     
         51 . The method according to  claim 45  wherein the compound is of the formula  
       
         
           
           
               
               
           
         
       
     
     
         52 . The method according to  claim 45  wherein the compound is of the formula  
       
         
           
           
               
               
           
         
       
     
     
         53 . The method according to  claim 45  wherein the compound is of the formula  
       
         
           
           
               
               
           
         
       
     
     
         54 . A method for treating a genetic disorder or condition or disease in a patient in need of treatment, comprising: 
 administering an effective amount of a composition comprising a compound selected from the group consisting of cationic lipid compounds, cationic lipid compounds in admixture with or associated with a nucleotide sequence, cationic liposomes (formed from a cationic lipid compound) in admixture with or associated with a nucleotide sequence, and combinations thereof;    the compound comprising a cholesterol group or derivative thereof having linked thereto a head group; wherein the head group is more positive than the head group of DC-Chol; further wherein the head group is a straight chain polyamine; further wherein two or more of the amine groups of the polyamine group are separated by an ethylene group.    
     
     
         55 . The method according to  claim 54  wherein the cholesterol group or derivative thereof is cholesterol.  
     
     
         56 . The method according to  claim 54  wherein the cholesterol group is linked to the head group via a carbamoyl linkage.  
     
     
         57 . The method according to  claim 54  wherein the compound is selected from compounds of the formula  
       
         
           
           
               
               
           
         
         where Chol denotes a group of the formula  
         
           
             
             
                 
                 
             
           
         
       
     
     
         58 . The method according to  claim 54  wherein the compound is selected from compounds of the formula  
       
         
           
           
               
               
           
         
       
     
     
         59 . The method according to  claim 54  wherein the compound is selected from compounds of the formula  
       
         
           
           
               
               
           
         
       
     
     
         60 . The method according to  claim 54  wherein the compound is of the formula  
       
         
           
           
               
               
           
         
       
     
     
         61 . The method according to  claim 54  wherein the compound is of the formula  
       
         
           
           
               
               
           
         
       
     
     
         62 . The method according to  claim 54  wherein the compound is of the formula  
       
         
           
           
               
               
           
         
       
     
     
         63 . The method according to  claim 54  wherein the composition further comprises a pharmaceutical.  
     
     
         64 . The method according to  claim 63 , wherein the composition further comprises a pharmaceutically acceptable diluent, carrier or excipient.  
     
     
         65 . A method for the treatment of a genetic disorder or condition or disease in a patient in need thereof, comprising administering a cationic lipid compound, the compound comprising a cholesterol group or derivative thereof having linked thereto a head group, wherein the head group is more positive than the head group of DC-Chol; further wherein the head group is a polyamine group which is a straight chain polyamine group; further wherein two or more of the amine groups of the polyamine group are separated by an ethylene group.  
     
     
         66 . A method for the treatment of a genetic disorder or condition or disease in a patient in need thereof, comprising administering a cationic liposome formed from a cationic lipid compound, the compound comprising a cholesterol group having linked thereto a head group; wherein the head group is more positive than the head group of DC-Chol; further wherein the head group is a polyamine group which is a straight chain polyamine group; further wherein two or more of the amine groups of the polyamine group are separated by an ethylene group.  
     
     
         67 . A method for the treatment of a genetic disorder or condition or disease in a patient in need thereof, comprising administering a cationic lipid compound in admixture with or associated with a nucleotide sequence, the compound comprising a cholesterol group or derivative thereof having linked thereto a head group; wherein the head group is more positive than the head group of DC-Chol; further wherein the head group is a polyamine group which is a straight chain polyamine group; further wherein two or more of the amine groups of the polyamine group are separated by an ethylene group.  
     
     
         68 . A method for the treatment of a genetic disorder or condition or disease in a patient in need thereof, comprising administering a cationic liposome in admixture with or associated with a nucleotide sequence, wherein the cationic liposome is formed from a cationic lipid compound, the compound comprising a cholesterol group having linked thereto a head group; wherein the head group is more positive than the head group of DC-Chol; further wherein the head group is a polyamine group which is a straight chain polyamine group; further wherein two or more of the amine groups of the polyamine group are separated by an ethylene group.  
     
     
         69 . A method for the treatment of a genetic disorder or condition or disease in a patient in need thereof, comprising administering a pharmaceutical composition comprising 
 (i) a cationic lipid compound, the compound comprising a cholesterol group or derivative thereof having linked thereto a head group; wherein the head group is more positive than the head group of DC-Chol; further wherein the head group is a polyamine group which is a straight chain polyamine group; further wherein two or more of the amine groups of the polyamine group are separated by an ethylene group; and    (ii) a pharmaceutical and, optionally, a pharmaceutically acceptable diluent, carrier or excipient.    
     
     
         70 . A method for treatment of a genetic disorder or condition or disease in a patient in need thereof, comprising administering a pharmaceutical composition comprising: 
 (i) a cationic liposome formed from a cationic lipid compound, the compound comprising a cholesterol group having linked thereto a head group; wherein the head group is more positive than the head group of DC-Chol; further wherein the head group is a polyamine group which is a straight chain polyamine group; further wherein two or more of the amine groups of the polyamine group are separated by an ethylene group; and    (ii) a pharmaceutical and, optionally, a pharmaceutically acceptable diluent, carrier or excipient.

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