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PubMed · 11713770

Technology evaluation: AAV-CFTR vector, targeted genetics.

S J Tebbutt
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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].

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AnimalsClinical Trials, Phase I as TopicClinical Trials, Phase II as TopicCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorDefective VirusesDependovirusDouble-Blind MethodDrug Administration RoutesGenes, ViralGenetic TherapyGenetic VectorsHumansLungMacaca mulattaMaxillary SinusPatents as TopicRabbitsRandomized Controlled Trials as TopicSafetySinusitisStructure-Activity RelationshipTreatment OutcomeVirus IntegrationJournal Article

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S J Tebbutt. 1999. Technology evaluation: AAV-CFTR vector, targeted genetics.. https://pubmed.ncbi.nlm.nih.gov/11713770/

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