Rhone-Poulenc Rorer S.a.

Transgene S.a.

Otmane Boussif

Daniel Scherman

Thierry Delair

Jean-Paul Behr

Barbara Demeneix

Hanno V J Kolbe

Franck Lezoualch

Laurent Veron

Mojgan Mergny

The invention concerns novel cationic polymers of formula (I) in which n is a whole number between 0 and 5 and p is a whole number between 2 and 20,000, more particularly p ranges between 10 and 18,000, and advantageously between 200 and 1,000, characterized in that: at least 10%, advantageously 30 to 80%, preferably 70%, of the free NH functions are substituted by identical or different hydrophilic R groups; said cationic polymer can further comprise at least a targeting element associated covalently or not with the free NH functions and/or said hydrophilic R groups provided that said cationic: polymer contains at least 20%, preferably at least 30%, of free NH functions. The invention also concerns a complex comprising at least said cationic polymer associated with at least a therapeutically active substance, in particular a nucleic acid comprising at least a negative charge. The invention is useful for transferring a nucleic acid into target cells, in particular for gene therapy.

Cationic polymers, complexes associating said cationic polymers with therapeutically active substances comprising at least a negative charge, in particular nucleic acids, and their use in gene therapy

Nucleic acid containing composition, preparation and uses of same

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Strasbourg, France

Otmane Boussif