Oxford BioMedica has entered into a process development collaboration agreement with the UK Cystic Fibrosis Gene Therapy Consortium (GTC) and Imperial Innovations to develop a long-term therapy for patients with cystic fibrosis (CF). Concurrently with this, a separate option and license agreement has been signed between Oxford BioMedica and Boehringer Ingelheim.

The collaboration will use a novel, replication deficient lentiviral vector, in an inhaled formulation, to selectively introduce a CFTR gene into the relevant target cell. This approach has demonstrated high gene transfer efficiency, and offers the possibility of repeated administration to maintain a therapeutic effect. In addition, the approach has the potential, according to the company, to address all of the more than 2,000 different, known gene mutations across patients equally well, and thus offer a disease-modifying treatment option for all patients.

Oxford BioMedica will be responsible for process and analytical development, scale up of manufacture of the candidate and generation of material for toxicology studies. There is scope within the terms for the parties to establish a clinical supply agreement in the future. The collaboration may also include an evaluation of Oxford BioMedica’s Transgene Repression in vector Production (TRiP) System, and the development of stable producer cell lines for large scale production of the lentiviral vector.

In order to treat CF patients with a gene therapy, there is a requirement for a large volume of lentiviral vectors. Oxford BioMedica can produce large quantities of lentiviral vectors using its GMP-compliant manufacturing process in bioreactors.

Under a separate option and license agreement between Oxford BioMedica and Boehringer Ingelheim, Oxford BioMedica has granted Boehringer Ingelheim an option to license the exclusive global rights over Oxford BioMedica’s lentiviral vector technology to manufacture, register and commercialize this lentiviral vector-based gene therapy for the treatment of CF.

“This novel three-way partnership brings together an unparalleled combination of clinical, scientific, manufacturing and commercial skills in an effort to develop new treatments and make a major contribution to the lives of patients affected by cystic fibrosis,” said John Dawson, chief executive officer, Oxford BioMedica. “The GTC has been working determinedly for over 15 years to get to this exciting point of forming a partnership with a global pharmaceutical company with respiratory expertise, Boehringer Ingelheim. Our contribution to this partnership reaffirms our leading position in the development and manufacture of lentiviral vector gene therapy products at large scale. We look forward to working with our new academic and industry partners.”

Deborah Gill, professor of gene medicine at the Department of Medicine of Oxford University, said, “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. We have developed a novel viral vector-based product which is currently funded by the Health Innovation Challenge Fund and the Cystic Fibrosis Trust. It is with great pleasure that we now join forces with two world-class organizations. Boehringer Ingelheim will provide their multinational industry expertise, including a proven track record in the respiratory field, to drive the product towards the clinic, while Oxford BioMedica is the acknowledged leader in the field of lentiviral manufacturing. The GTC believes that this partnership provides CF patients with the optimal chance to establish gene therapy as routine clinical practice. We would like to take this opportunity to warmly thank all of our fundraisers who have supported us over many years.”