HepaVec AG

With technology licensed from the Max Planck Society, HepaVec is developing liver-specific viral vector systems for human gene therapy. Also liver tissue engineering.

• Business:Liver-specific viral vector systems for human gene therapy, liver tissue engineering

• Contact:Herbert Stadler, PhD, CEO; Heidemarie Rohdewohld, PhD, COO

• Address:Robert-Rossle-Strasse 10

• D-13122 Berlin, Germany

• Phone:[phone]

• Fax: [phone]

• Founded: March 1996

Explored as an alternative to surgery and radiation, gene therapy approaches to treating liver cancer have not been without their share of drawbacks. Ex-vivoapproaches have not yet proven effective, are very costly, and require surgery at least once; in vivo approaches have been hampered by difficulties in finding and validating high-efficiency vectors specific to the liver.

With technology licensed from the Max Planck-Society, HepaVec AG is developing baculovirus- and human adenovirus-derived viral vector systems that, the company claims, will provide effective in vivo gene therapies for liver cancer and genetic liver disease.

HepaVec's approach to liver cancer is based on reconstruction of cell cycle regulation through the p16 gene and inducement of apoptosis through the p53 gene in in vivotumor cells. According to company reports, recent studies with murine models have shown that simultaneous adenoviral transfer of p16 and p53 genes leads to cell death in tumor cells only (published in Nature Medicine 1997, Vol. 3).

In a second program, HepaVec is attempting to reverse the effects of Wilson's disease by transferring the ATP7B gene (key protein that can restore proper liver function even in small amounts) through a recombinant, complement-resistant baculovirus modified to integrate into the cellular DNA. In May 1998, HepaVec acquired exclusive North American rights to the Wilson's disease gene from its discoverer, Dr. Diane Cox at the Toronto Hospital for Sick Children [See Deal].

Through its investment in Munich-based start-up CMI GMBH, HepaVec has a hepatitis B development program. This program is based on a compound derived from the Phyllanthus amarus plant, and is scheduled to enter Phase II/III in Europe and the US in 1999. HepaVec and CMI will also collaborate to characterize individual antiviral compounds from the plant extract as potential next generation drugs against hepatitis B.

Lastly, HepaVec has proprietary non-cancerous immortal human liver cell lines that it plans to use for the production of correctly glycosylated recombinant proteins and/or for the development of a liver replacement device. HepaVec is applying a new concept, which makes use of recent results in cell control and anti-apoptosis strategies, to develop immortalized non-tumorigenic human liver cell lines for use in liver support devices.

HepaVec also has R&D collaborations with the following universities: Stanford University for development of a recombinant adeno-associated vector, the University of Heidelberg for gene therapy of Wilson's disease, and Humboldt University in Berlin, and its medical school, for viral vector development and gene therapy of liver metastasis.

In May 1997, HepaVec closed a $609,000 seed round with Techno Venture Management, Dansk Kapitalananlaeg (Denmark), and Alpinvest (Netherlands) [See Deal]. In addition, the company received $457,000 from the tbg (Technologie-Beteili-gungsgesellschaf) in Bonn. Prior to co-founding HepaVec, Dr. Herbert Stadler founded several biotech ventures. Co-founder Dr. Michael Strauss holds a chair in the cell biology department at Humboldt University and is a pioneer in viral vector development for gene therapy. COO Dr. Heidemarie Rohdewohld is a molecular biologist and was previously the group leader of the section Human Genetics and Gene Therapy at the Robert-Koch Institute in Berlin.–MR