Targeted genetics presents promising data from cystic fibrosis clinical trial

Studies suggest that aerosolized tgAAV-CF is safe, well tolerated, and efficiently delivered to the lung

Seattle, WA-June 5, 2000-Targeted Genetics Corporation (Nasdaq: TGEN) presented data from its Phase I clinical trial of an aerosol formulation of tgAAV-CF, the Company's gene therapy product for the treatment of cystic fibrosis (CF). Dr. Thomas C. Reynolds, Vice President, Clinical Affairs at Targeted Genetics, presented the data in an abstract titled "A Phase I Study of Aerosolized Administration of tgAAV-CF to CF patients with Mild Lung Disease" this weekend at the American Society of Gene Therapy Third Annual Meeting in Denver, Colorado.

The Phase I dose-escalation study evaluated the safety and tolerability of tgAAV-CF delivered via nebulizer to 12 patients age 19-41 (median age 29) with mild CF lung disease. All patients in the study had FEV1 levels (a standard measure of lung function) at least 60% of normal. Doses of 1010, 1011, 1012 and 1013 DNAse Resistant Particles (DRP) were evaluated, with each cohort containing three patients. Patients received a single dose of tgAAV-CF and were followed for 90 days post treatment.

"The aerosolized formulation of tgAAV-CF, which we are developing in collaboration with Celltech Group, plc, is well-tolerated in this patient population," said Dr. Reynolds. "Our ability to achieve broad distribution of tgAAV-CF throughout the lungs of these patients validates the aerosol delivery approach we are taking in developing this product. We are on track to initiate a Phase II multi-dose study of aerosolized tgAAV-CF within the next several months. As with the Phase I study presented today, we are working closely with the Cystic Fibrosis Foundation's Therapeutics Development Network (CFF TDN) in designing and conducting the Phase II study. tgAAV-CF is the first gene therapy approach for CF to be selected by the TDN for collaboration and we are proud to be working with the leading physicians

Contact: Page Sargisson
415-677-4455 ext 229
Noonan/Russo Communications

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