Cystic Fibrosis

Positive Phase I AAV-CFTR Trials Announced

Published in Gene Therapy Weekly, November 11th, 1996

Targeted Genetics Corp., Seattle, Washington, announced that preliminary results of two Phase I clinical trials testing the AAV-CFTR gene product in patients with cystic fibrosis (CF) demonstrated safety, efficient gene transfer, and long-term gene persistence.

"We are very encouraged with the results we have seen so far," said Richard Daifuku, M.D., Ph.D., Targeted Genetics. "The most exciting data to date come from the trial at Stanford University, where administration of the AAV-CFTR gene product resulted in consistent gene transfer, and persistence of the gene in one patient out to 70 days.

"CF gene therapies using other vectors have shown much...

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Source: Gene Therapy Weekly (1996-11-11)

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