Gene therapy reprise

Caused by a single gene defect, hemophilia was supposed to be an easy target for gene therapy, but it proved hard to get enough expression to have a therapeutic effect. Bayer AG thinks sufficient progress has been made since the turn of the decade to take another shot at the disease.

In 2000, Bayer (FSE:BAYG; BAY, Leverkusen, Germany) started a collaboration with Avigen Inc. (AVGN, Alameda, Calif.) for Coagulin-B adeno-associated virus (AAV) gene therapy to treat hemophilia B. However, development was terminated after two of seven patients in a Phase I trial had minor elevation of liver enzymes (see BioCentury, May 31, 2004). ...