Valentis Reports on Studies with Factor IX, EPO Gene Medicines

Valentis Inc.'s (Burlingame, CA) proprietary plasmid-based Factor IX gene medicine demonstrated correction of bleeding in a large animal model of hemophilia following a single administration of the therapy, company researchers reported at the American Society for Gene Therapy's Third Annual Meeting in Denver. The researchers also reported that regulated expression, after a single administration of the company's erythropoietin gene medicine controlled by the company's GeneSwitch, was shown for up to one year.

Faxtor IX Study
Valentis' Factor IX gene medicine consist of a DNA plasmid containing the Factor IX gene formulated with one of the company's proprietary PINC polymers. The non-viral product is administered via intra-muscular injection; expression of the therapeutic protein is enhanced by electroporation, the application of a small amount of electricity at the site of injection for a few milliseconds.

In the study, four hemophiliac dogs were administered the Factor IX gene medicine using in vivo electroporation into the muscle. The dogs tolerated the electroporation procedure very well and did not require administration of the factor IX protein during the procedure. Whole blood clotting times began to decline within 24 hours of administration.

GeneSwitch Study
In small animal studies using Valentis' EPO gene medicine under the control of the company's proprietary GeneSwitch technology, researchers administered plasmids encoding erythropoietin and the GeneSwitch to mice. Upon oral administration of a small molecule inducer, a dose-dependent increase in erythropoietin protein was detected in the blood. Several cycles of increased hematocrit levels were achieved throughout the one-year study.

According to Alain Rolland, VP of research and development and head of Valentis' Woodlands Center in Texas, the GeneSwitch technology was designed to provide the type of control demonstrated in the study, and has successfully been shown in animal models to enable tight regulation of gene expression in response to oral administration of specific inducers for up to one year.

About Valentis
Valentis Inc. develops proprietary technologies and applies its preclinical and early clinical development expertise to create novel therapeutics. The company's core technologies include multiple gene delivery and gene expression systems and PEGylation technologies designed to improve the safety, efficacy, and dosing characteristics of genes, proteins, peptides, peptidomimetics, antibodies and replicating and non-replicating viruses.

For more information: Valentis Inc., 863-A Mitten Rd., Burlingame, CA 94010. Tel: 650-697-1900. Fax: 650-652-1990.

Edited by Jim Pomager