First line gene therapy for cystic fibrosis; Safe repeat administration using nebuliser; Phase IIb randomised clinical trial complete; Orphan Drug Designation secured in US and EU

About

The UK Gene Therapy Consortium – a collaboration across Imperial College London, University of Oxford and University of Edinburgh – has developed a non-viral, lipid/DNA formulated gene therapy product for cystic fibrosis. We have clinical proof of concept for lung gene transfer in CF patients. The GL67A liposome designed by the GTC has shown superior transduction efficiency compared to other non-viral liposomal or nanoparticle gene transfer agents. The CFTR transgene has been sequence optimised and a novel promoter/enhancer designed to increase transgene expression.

Evidence/Results:

(i) Preclinical studies showing expression of human CFTR mRNA and functional protein
(ii) Scale-up and production of GMP plasmid and liposome 
(iii) Phase I/IIa clinical studies established a safe dose and demonstrated ~20% correction of the CF electrophysiological defect 
(iv) An MHRA-approved, two-species, toxicology programme for repeated administration to the airways 
(v) Phase IIb double-blind placebo-controlled clinical trial which reached its primary endpoint with a significant beneficial effect in FEV1 compared with placebo

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