Technology evaluation: AAV-CFTR vector, targeted genetics

Targeted Genetics is developing a gene therapy product, the AAV-CFTR vector system, for the treatment of cystic fibrosis (CF). This involves administration of an adeno-associated virus (AAV) vector containing CF transmembrane conductance regulator gene (CFTR) directly to the lungs of cystic fibrosis patients. The drug has Orphan Drug Status [325713]. Medeva acquired the worldwide commercial rights to the therapy in November 1998. Medeva expects to file a license application for the therapy, in the US and Europe, by 2002 [325713]. The therapy utilizing the normal CFTR gene entered phase II trials for sinusitis [197407] and phase II trials for CF in the first quarter of 1997. Lehman Brothers predicts filing in 2001/2002 [318119].