Boehringer Ingelheim starts gene therapy collaboration for cystic fibrosis

German pharma company and CDMO Boehringer Ingelheim (BI) steps into the lucrative field of gene therapy. Together with the UK Cystic Fibrosis Gene Therapy Consortium (GTC), BI aims at developing replacement therapies compensating for mutations that lead to cystic fibrosis.

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Under the deal, Boehringer Ingelheim acquires an option to licence IP from Imperial Innovations, the tech transfer arm of Imperial College London, and from gene therapy developer and manufacturer Oxford Biomedica and will collaborate in R&D with the UK Cystic Fibrosis Gene Therapy Consortium, which has a clinical track record of six successful early stage CF gene therapies and has developed a novel lentiviral vector (rSIV.F/HN) that will serve as a gene therapy development platform within the partnership.

There are more than 2,000 known mutations in the gene for the cystic fibrosis transmembrane conductance regulator (CFTR), many of which result in a person developing CF. The disease is estimated to affect 70,000 people worldwide, with the majority of those located in the US and Europe. According to Boehringer Ingelheim, current treatments and drug candidates can only slow the progression of disease.

The collaboration will be based on a novel approach using a replication-deficient lentiviral vector in an inhaled formulation, to introduce a healthy copy of the CFTR gene into the cells of the lung. This method has demonstrated high gene transfer efficiency and can be repeatedly administered.

Financial terms of the agreement were not disclosed. 

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