The UK Cystic Fibrosis Gene Therapy Consortium: Clinical Trial Programme

Clinical Trial Programme

Our clinical programme is broken down into a number of different studies. In our preclinical studies we have defined assays and patient groups. We have then progressed onto the ongoing clinical trials.

In this section you will learn more about our clinical trial product (GL67A/pGM169), the clinical teams and our clinical programme.

Background

Prior to the formation of the Consortium, members of the group had carried out 5 clinical trials using cationic lipids to carry genes into the nose and lungs of Cystic Fibrosis (CF) patients.

In these trials we (and other researchers in this field) used a variety of gene transfer agents (GTA) and proved the principle that CFTR (cystic fibrosis transmembrane regulator) gene transfer is possible. We have shown that partial correction of the basic defect in the airways of CF patients can be achieved without major safety issues.

Despite these encouraging human studies, gene transfer to the CF lung is still sub-optimal and needs further improvement. The aim of the UK Cystic Fibrosis Gene Therapy Consortium is to develop a clinically effective gene therapy for CF.