Targeted Genetics Ends CF Gene Therapy Study

Targeted Genetics Corporation today announced that it is ending its cystic fibrosis (CF) gene therapy study. This decision was made after learning that results from its most recent clinical trial with the adeno-associated vector, tgAAVCF, failed to show sufficient improvement in lung function, in people with CF. The Cystic Fibrosis Foundation is disappointed that improvements were not seen.

The CF Foundation’s collaboration with Targeted Genetics has answered many critical research questions that will help guide future gene therapy research. Other CF research programs are underway to evaluate both viral vectors and non-viral vectors to deliver CF gene therapy. The CF Foundation met with CF gene therapy experts and the Food and Drug Administration last fall to continue to evaluate the field of CF gene therapy and we remain committed to moving it forward.

In addition to gene therapy, the CF Foundation supports many other approaches to identify new treatments for CF. These include several studies aimed at correcting the basic defect, either by correcting the faulty protein made by the CF gene, or bypassing the cell defect entirely with new therapies.  For more details on the CF Foundation’s broad approach to new treatments, click here to see what's in the CF drug discovery and development pipeline.

The CF Foundation will meet with Targeted Genetics and scientists to discuss this trial in April and more information will be available at that time.  For more information about Targeted Genetics announcement, visit their Web site at

Posted 3/17/05