Boehringer joins consortium to develop new cystic fibrosis gene therapies

In a statement BI said that the collaboration "brings together the academic partners’ leading expertise in developing gene therapy for CF and OXB’s leading expertise in manufacturing lentiviral vector-based therapies with Boehringer Ingelheim’s capabilities in drug discovery and the clinical development of novel breakthrough therapeutic agents".

Cystic fibrosis is a genetic disease that causes persistent lung infections and usually limits a person’s ability to breathe over time. 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. Existing treatments and those under development can only slow the progression of disease. As such, CF continues to be associated with considerable morbidity, mortality, and a high burden of therapy. In addition, existing treatments will have varying effects depending on a patient’s mutation status, as determined by the particular gene variation that causes their disease.

The collaboration will focus 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 offers the possibility of repeated administration to maintain the therapeutic effect. Gene therapy is the only therapeutic approach to date that can address all CFTR gene mutations, thus potentially offering a universal treatment option.

"The UK CF Gene Therapy Consortium has, for the last 17 years, vigorously sought to establish whether gene therapy can become a clinically viable option for patients with CF," said Professor Eric Alton, Coordinator of the UK Cystic Fibrosis Gene Therapy Consortium, which consists of Imperial College London and the Universities of Oxford and Edinburgh. "From the beginning the GTC identified that this goal would require incremental increases in knowledge. We have, therefore, built on our non-viral gene therapy experience to develop a new viral vector-based product, which is currently funded by the Health Innovation Challenge Fund (a partnership between the Wellcome Trust and the Department of Health and Social Care) and the Cystic Fibrosis Trust

"The GTC believes that this partnership provides CF patients with the optimal chance to establish gene therapy as routine clinical practice, relevant to all patients, irrespective of their mutation status, and in due course to both prevent lung disease as well as treat established problems."

Boehringer has received an option to license the exclusive global rights to develop, manufacture, register, and commercialise this lentiviral vector-based gene therapy for the treatment of cystic fibrosis. Financial terms were not disclosed. During the option period the partners will work closely together to pursue the development of this innovative approach financed by Boehringer Ingelheim.

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