Industry collaboration to develop novel gene therapies for cystic fibrosis

US accepts Editas’ IND for experimental CRISPR medicine
Credit: Shutterstock.com/ Gek

Gene and cell therapy group, Oxford BioMedica, has entered into a 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, 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 more than 2,000 different, known gene mutations across patients equally well, and thus offer a disease-modifying treatment option for all patients.

As per the 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.

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, 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.