Bayer AG and US biotechnology company Avigen have forged a deal worth upto $60 million for the latter's Coagulin-B gene therapy for hemophilia B. Coagulin-B is Avigen's proprietary adeno-associated viral vector containing the gene for the blood-clotting protein Factor IX, which is deficient or absent in hemophilia B patients.
At the end of last year, Avigen provided a boost to the much-scrutinized gene therapy industry when it revealed efficacy data from an early-stage clinical trial of Coagulin-B in three patients. The company said that even at the lowest dose administered, Factor IX protein activity was detected. The researchers also noted that this increase in the biological activity of the protein correlated with a significant reduction in Factor IX concentrate usage by patients, a result it "would not have anticipated at this dose level" (Marketletter December 13, 1999).
The news helped restore confidence in the field following the unexpected death of a patient in gene therapy trials at the University of Pennsylvania (Marketletters October 18 and November 29, 1999), and this latest investment from a large pharmaceutical company should further this progress.
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