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].
MeSH terms
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Animals
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Clinical Trials, Phase I as Topic
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Clinical Trials, Phase II as Topic / economics
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Cystic Fibrosis / genetics
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Cystic Fibrosis / therapy*
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Cystic Fibrosis Transmembrane Conductance Regulator / deficiency
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Cystic Fibrosis Transmembrane Conductance Regulator / genetics*
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Cystic Fibrosis Transmembrane Conductance Regulator / physiology
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Defective Viruses / genetics*
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Dependovirus / genetics*
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Double-Blind Method
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Drug Administration Routes
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Genes, Viral
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Genetic Therapy*
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Genetic Vectors* / administration & dosage
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Genetic Vectors* / adverse effects
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Genetic Vectors* / genetics
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Genetic Vectors* / therapeutic use
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Humans
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Lung / metabolism
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Lung / virology
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Macaca mulatta
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Maxillary Sinus
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Patents as Topic
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Rabbits
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Randomized Controlled Trials as Topic
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Safety
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Sinusitis / therapy
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Structure-Activity Relationship
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Treatment Outcome
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Virus Integration
Substances
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CFTR protein, human
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Cystic Fibrosis Transmembrane Conductance Regulator