Sinus Gene Therapy Update

Spring 1998

It is very exciting to see the progress in a clinical trial being conducted at Stanford University Medical Center. Targeted Genetics Corporation of Seattle, Washington and Stanford University Medical Center have started a Phase II clinical trial using a Targeted Genetics gene therapy product tgAAV-CFTR vector in the sinuses of 50 subjects with cystic fibrosis. The preliminary results of this study are causing great optimism and excitement in the cystic fibrosis community. CFRI is particularly proud because it has helped provide seed money for this study.

Two Phase I clinical trials completed last Spring demonstrated that tgAAV-CFTR is safe, provides efficient gene transfer and provides long-term gene persistence (up to 70 days). The Phase II trial is designed to test the efficacy of tgAAV-CFTR in preventing sinusitis in patients with CF. This study has amassed solid preclinical and clinical data showing the longest gene persistence of any gene therapy product.

Targeted Genetics is developing aerosol formulations of tgAAV-CFTR and plans to initiate clinical trials during the summer of 1998 to evaluate aerosol delivery of the drug to the lungs of CF patients.

Phyllis Gardner, M.D., Associate Professor of Molecular Pharmacology and Medicine at Stanford, is Principal Investigator for the trial. Co-investigator, John A. Wagner, M.D., Ph.D., is a fellow in Clinical Pharmacology and Clinical Associate Physician in the General Clinical Research Center at Stanford.

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