Cystic Fibrosis Trial Looks Promising - Targeted Genetics Corp's testing of tgAAV-CF, the company's gene therapy product - Company Business and Marketing

Applied Genetics News, April, 1999

Targeted Genetics Corp. (1100 Olive Way, Suite 100, Seattle, WA 98101; Tel: 206/623-7612, Fax: 206/223-0288) has announced positive findings from a phase I/II clinical trial of tgAAV-CF, the company's gene therapy product for the treatment of cystic fibrosis (CF). Cystic fibrosis results from the lack of a functional copy of the CFTR gene, which encodes a chloride ion channel protein.

The tgAAV-CF product uses an adeno-associated virus to deliver a normal copy of the CFTR gene. Conducted at Stanford University, the phase I/II study was designed to assess the safety of the gene therapy treatment and the effect of therapy in reducing sinusitis in CF patients. A total of 23 patients were treated with tgAAV-CF in one sinus and with placebo in the other. Patients were monitored for changes in serological markers, including the presence of a circulating vector and the production of neutralizing antibodies to the vector. Earlier results suggested that tgAAV-CF was safe and there was evidence of gene transfer persisting up to 70 days. In the present study, there were no adverse events related to the vector. Inflammation was reduced in the sinuses treated with tgAAV-CF but not with placebo. Full results of the study will be published at a later date. "We have observed a biologic effect associated with treatment with our gene therapy product in a proposed model of CF lung disease," notes Darrell J. Salk, vice president of clinical affairs at Targeted Genetics. "Because the degree of inflammation correlates with the amount of active disease, a reduction in inflammation is a positive sign." The company expects to move forward with a phase I trial of aerosolized tgAAV-CF in the treatment of CF lung disease. Ultimately, Targeted Genetics' development partner, Medeva PLC will take the product through the regulatory process for approval, if all goes well.