Novel colloid synthetic vectors for gene therapy

A compound having the formula I

• 43
• wherein m is 3 or 4;
• Y signifies a group —(CH2)n—, in which n is 3 or 4, or may also signify a group —(CH2)n—, in which n is an integer from 5 to 16, or may also signify a group —CH2—CH═CH—CH2—, if R2 is a group —(CH2)3—NR4R5 and m is 3;
• R2 is hydrogen or lower alkyl or may also signify a group —(CH2)3—NR4R5 if m is 3;
• R3 is hydrogen or alkyl or may also signify a group —CH2—CH(—X′)—OH, if R2 is a group —(CH2)3—NR4R5 and m is 3;
• X and X′, independently of one another, signify hydrogen or alkyl;
• and the radicals R, R1, R4 and R5, independently of one another, are hydrogen or lower alkyl;
• with the proviso that the radicals R, R1, R2, R3 and X cannot all together signify hydrogen or methyl, if m is 3 and Y signifies a group —(CH2)3—;
• and their pharmaceutically acceptable salts.

A non-naturally occurring gene therapy vector comprising an inner shell comprising:

• (1) a core complex comprising a nucleic acid and at least one complex forming reagent;
• (2) a nuclear targeting moiety;
• (3) a fusogenic moiety;
• and (4) an outer shell comprising (i) a hydrophilic polymer that stabilizes said vector and reduces nonspecific binding to proteins and cells and (ii) a tageting moiety that provides binding to target tissues and cells, wherein said outer shell is linked via a cleavable linkage that enables the outer shell to be shed.