Neutral-cationic lipid for systemic delivery of Factor VIII gene
OwnerShi-Kun Huang, Bei Jin +3inventors
InventorsShi-Kun Huang, Bei Jin, Weiming Zhang, Yolanda Quinn, Samuel Zalipsky
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Abstract
A composition for delivery of Factor VIII is described. The composition is comprised of an expression vector encoding for Factor VIII and a lipid represented by the formula: 1 where each of R1 and R2 is an alkyl or alkenyl chain having between 8-24 carbon atoms; n=0-20, preferably 1-20; L is selected from the group consisting of (i) —X—(C═O)—Y—, (ii) —X—(C═O)—, and (iii) —X—, where X and Y are independently selected from oxygen, NH and a direct bond; and Z is a weakly basic moiety that has a pK of less than 7.4 and greater than about 4.0.
Claims33
A liposome composition, comprising:
- a lipid having the formula
- 4
- where each of R1 and R2 is an alkyl or alkenyl chain having between 8-24 carbon atoms, and each of R1 and R2 are independently selected;
- n=0-20, preferably n=1-20;
- L is selected from the group consisting of (i) —X—(C═O)—Y—, (ii) —X—(C═O)—, and (iii) —X—, where X and Y are independently selected from oxygen, NH and a direct bond;
- Z is a weakly basic moiety that has a pK of less than 7.4 and greater than about 4.0.
A lipid having the formula:
- 5
- where each of R1 and R2 is an alkyl or alkenyl chain having between 8-24 carbon atoms, and each of R1 and R2 are independently selected;
- n=0-20, preferably n=1-20;
- L is selected from the group consisting of (i) —X—(C═O)—Y—, (ii) —X—(C═O)—, and (iii) —X—, where X and Y are independently selected from oxygen, NH and a direct bond;
- Z is a weakly basic moiety that has a pK of less than 7.4 and greater than about 4.0.
A method for delivery of a therapeutic agent to the cytoplasm of a cell, comprising
- preparing liposomes comprising a lipid having the formula
- 6
- where each of R1 and R2 is an alkyl or alkenyl chain having between 8-24 carbon atoms, and each of R1 and R2 are independently selected;
- n=0-20, preferably n=1-20;
- L is selected from the group consisting of (i) —X—(C═O)—Y—, (ii) —X—(C═O)—, and (iii) —X—, where X and Y are independently selected from oxygen, NH and a direct bond;
- Z is a weakly basic moiety that has a pK of less than 7.4 and greater than about 4.0;
- and
- administering the liposomes to a subject.
Dependencies are inferred from the claim text.