Repeated Application of Gene Therapy in CF Patients

Clinical Publications

• Safety of a single aerosol administration of escalating doses of the cationic lipid GL-67/DOPE/DMPE-PEG5000 formulation to the lungs of normal volunteers
• A Phase I/IIa Safety and Efficacy Study of Nebulized Liposome-mediated Gene Therapy for Cystic Fibrosis Supports a Multidose Trial
• Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial
• Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial
• (Abstract P204) Immune Responses To Single And Repeated Administration Of Pgm169/gl67a: The Uk Cf Gene Therapy Consortium Clinical Trials
• A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis

News and Press Releases

• UK Respiratory Gene Therapy Consortium: Our First Clinical Trial Product
• The UK Respiratory Gene Therapy Consortium are delighted to announce the start of recruitment for a second major gene therapy trial for people with cystic fibrosis.

Preclinical Publications

• Toxicology study assessing efficacy and safety of repeated administration of lipid/DNA complexes to mouse lung
• Limitations of the murine nose in the development of nonviral airway gene transfer
• The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airways
• Pre-clinical evaluation of three non-viral gene transfer agents for cystic fibrosis after aerosol delivery to the ovine lung
• Assessment of the nuclear pore dilating agent trans-cyclohexane-1,2-diol in differentiated airway epithelium
• The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep
• (Abstract 915) Towards Gene Therapy for Cystic Fibrosis: Bio-Distribution of GL67A/pGM169 DNA and mRNA Following Aerosol Delivery to the Mouse Lung - ASGCT 2008
• CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression

Related NCTID

• Phase 1/2: NCT00789867