Safety and Efficacy of Recombinant Adeno-Associated Virus Containing the CFTR Gene in the Treatment of Cystic Fibrosis

Cystic Fibrosis

Gene Transfer, Pulmonary function

The purpose of this study is to confirm the improvement in pulmonary function and cytokine levels observed in the recently completed multidose aerosol study for the treatment of Cystic Fibrosis (CF).

Cystic Fibrosis is an autosomal recessive disorder with an incidence of approximately 1 in 33000 live births. It is due to defects in the CFTR gene, which is located on chromosome 7. Gene Therapy holds the promise of addressing the primary defect in CF by reconstituting the CFTR function in the lung. tgAAVCF, which has been genetically engineered to contain the CFTR gene, has been extremely well tolerated following single and multiple dose administrations to the nose, sinus, and lung. Dose-dependent gene transfer has been demonstrated. Although vector gene expression has not been detected, evidence consistent with biological activity was observed in maxillary sinus study, and statistically significant changes in the FEV1 and IL-8 levels were observed in the recently completed multidose aerosol study. These findings are worthy of further investigation.

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United States