Therapy Areas: Hereditary Disorders
Oxford BioMedica forms cystic fibrosis partnership
6 August 2018 -

Gene and cell therapy group Oxford BioMedica plc (LSE: OXB) has entered into a collaboration to develop a novel gene therapy treatment for cystic fibrosis, the company revealed on Monday.

Oxford BioMedica will work with the UK Cystic Fibrosis Gene Therapy Consortium (GTC) and Imperial Innovations to develop a long-term therapy for patients with cystic fibrosis, focusing on a novel, replication deficient lentiviral vector, in an inhaled formulation, to selectively introduce a CFTR gene into the relevant target cell.

According to Oxford BioMedica, 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 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. In the future, the parties also may establish a clinical supply agreement.

Additionally, the collaboration may 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.

Under a separate option and licence agreement with Boehringer Ingelheim, Oxford BioMedica has granted the German company an option to license 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 cystic fibrosis. Financial terms of the agreement were not disclosed.

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