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Encouraging results for Copernicus' non-viral gene transfer system to

correct defect in CF

Copernicus Therapeutics presented data at the 6th Annual Meeting of the

American Society of Gene Therapy, held from 4th to 8th June, in

Washington DC, showing that its compacted DNA formulation partially

restored chloride channel function in the nasal epithelial cells of

human subjects with cystic fibrosis (CF). No serious adverse events

occurred in the trial, demonstrating that the compacted DNA

nanoparticles could be safely administered to the nostrils of CF

subjects. This Phase I/II trial was a joint collaboration of Copernicus,

University Hospitals of Cleveland, Case Western Reserve University

School of Medicine, The Children's Hospital of Denver and Cystic

Fibrosis Foundation Therapeutics.

A recently completed clinical trial in CF subjects indicates that the

DNA drug was safe and well tolerated when applied to the nasal mucosa,

and encouraging gene transfer endpoints were observed. Two-thirds of the

trial subjects demonstrated evidence of DNA expression, with partial

correction of the underlying chloride channel defect that is responsible

for CF disease manifestations.

Becki

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