title: Technology evaluation: AAV-CFTR vector, targeted genetics.
abstract: 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].
authors.affiliation: Department of Biochemistry, University of Otago, Dunedin, New Zealand. scott.tebbutt@stonebow.otago.ac.nz
authors.forename: S J
authors.initials: SJ
authors.lastname: Tebbutt
mesh_terms: D000818:Animals; D017321:Clinical Trials, Phase I as Topic; D017322:Clinical Trials, Phase II as Topic / Q000191:economics; D003550:Cystic Fibrosis / Q000235:genetics / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000172:deficiency / Q000235:genetics* / Q000502:physiology; D003673:Defective Viruses / Q000235:genetics*; D000229:Dependovirus / Q000235:genetics*; D004311:Double-Blind Method; D004333:Drug Administration Routes; D005814:Genes, Viral; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000008:administration & dosage / Q000009:adverse effects / Q000235:genetics / Q000627:therapeutic use; D006801:Humans; D008168:Lung / Q000378:metabolism / Q000821:virology; D008253:Macaca mulatta; D008443:Maxillary Sinus; D010330:Patents as Topic; D011817:Rabbits; D016032:Randomized Controlled Trials as Topic; D012449:Safety; D012852:Sinusitis / Q000628:therapy; D013329:Structure-Activity Relationship; D016896:Treatment Outcome; D016662:Virus Integration
chemical_list: C505032:CFTR protein, human; D019005:Cystic Fibrosis Transmembrane Conductance Regulator