Targeted Genetics Corp. is beginning a Phase I trial of its new genetherapy based on a modified adeno-associated virus (AAV) vector fortreatment of cystic fibrosis.Jon Case, Targeted Genetics' director of corporate development, saidthe study will be the first in which AAV is used to deliver genes to thebody. The Seattle-based company's trial will involve 16 patients withcystic fibrosis and mild lung disease at the Johns Hopkins Children'sCenter in Baltimore. The participants will be treated during an 18-dayhospital stay and follow-up evaluations will be conducted on anoutpatient basis for one year.People afflicted with cystic fibrosis have a defective cystic fibrosistransmembrane conductance regulator (CFTR) gene. The CFTR is acell surface protein that acts as a chloride channel moving fluids acrossthe lungs. Deficiencies in that process cause a build up of mucus,which can produce infections and scarring in the lungs, eventuallyleading to death. Targeted Genetics' tgAAV-CF vector is designed todeliver a normal copy of the CFTR gene to be integrated into the lungs'epithelial cells.The company received approval from the Recombinant DNA AdvisoryCommittee of the National Institutes of Health to conduct the trial.Targeted Genetics expects to file an investigational new drugapplication with the FDA by the end of this year.Case said AAV is a common, naturally occurring virus, which is notknown to be associated with any disease.The Phase I trial is designed to assess the safety of tgAAV-CF and testits expression in cells of the nose and lungs. _ Charles Craig
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