New Publication - Preparation for a Lentivirus Gene Therapy Clinical Trial in Cystic Fibrosis

Wednesday, November 16th 2016

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btslogo.jpgPreparation for a first-in-man lentivirus trial in patients with cystic fibrosis, Thorax, 2016 In Press.

The Consortium are pleased to announce the publication of a new research paper outlining the progress made with our Lentiviral vector (LV) gene therapy platform for Cystic Fibrosis lung disease.

The LV has been pseudotyped to achieve efficient gene transfer into airway epithelial cells. Production and purification of the vector has been demonstrated in a scalable method suitable for clinical production.

The efficacy of the vector has been demonstrated in mouse studies. 14% of airway epithelial cells were positive for green fluorescent protein in one study and the duration of expression from all tested vector constructs was at least 6 months.

Further studies demonstrated the ability of the vector to express function CFTR and the ability of the vector to avoid detection by the immune system.

The data support the progression of the F/HN-pseudotyped lentiviral vector into a first-in-man CF trial.

The publication is available to download here.

 

Thorax_2016_GFP_Images.jpg

(A) Representative image of a lentivirus transduced mouse lung. (B) Representative image of an untransduced control mouse. Scale bar=50 µm. AW, airway; P, parenchyma.

 

 

Further information


 

A CFTR Western blot, to confirm protein production in cell culture.

 

Pellets of DNA following precipitation.

 

Light microscope image of a human airway liquid interface cultures. Dark patches are mucous.