Oxford BioMedica, UK Cystic Fibrosis Gene Therapy Consortium, Boehringer Ingelheim and Imperial Innovations Form Partnership to Develop a Novel Gene Therapy Treatment for Cystic Fibrosis

Oxford BioMedica plc (LSE:OXB) (“Oxford BioMedica” or “the Group”), a leading gene and cell therapy group, today announces that it 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 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.

Under the terms of the process development collaboration agreement, 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 highly efficient 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 licence the exclusive global rights over Oxford BioMedica’s lentiviral vector technology to manufacture, register and commercialise this lentiviral vector-based gene therapy for the treatment of CF. The financial terms for the option and license agreement with Boehringer Ingelheim are undisclosed.

This collaboration brings together the UK’s leading CF academic and clinical groups within the GTC, who have already pioneered clinical development of other gene therapy programmes for CF and are recognised leaders in this important disease with high unmet medical need. Boehringer Ingelheim is a global, research-driven pharmaceutical company with a strong franchise in respiratory medicine and a depth of experience in developing novel pharmaceutical treatments.

John Dawson, Chief Executive Officer of Oxf