Partnership Formed to Develop Gene Therapy for Cystic Fibrosis

August 6, 2018

Rare Daily Staff

Oxford BioMedica said it has entered into a process development collaboration agreement with the UK Cystic Fibrosis Gene Therapy Consortium and Imperial Innovations to develop a gene therapy for all patients with cystic fibrosis and separately signed an option and license agreement with Boehringer Ingelheim for the therapy.

Cystic fibrosis is a rare, life-shortening genetic disease. It is caused by a defective or missing cystic fibrosis transmembrane conductance regulator, or CFTR protein. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working or too few CFTR proteins at the cell surface.

The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death.

The continued development of conventional therapies has greatly improved both the quality of life and the mean life expectancy of people with CF which has reached around 40 years. However, there is still no treatment for the underlying defect causing the disease and individuals with the disease suffer from a high therapeutic burden and a severely compromised quality of life. While treatments do exist, the disease continues to be fatal for most patients as existing treatments and treatments under development can only slow the progression of disease and patients will still ultimately succumb to lung failure.

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 System, and the development of stable producer cell lines for large scale production of the lentiviral vector.

Under the separate option and license agreement 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. The financial terms for the option and license agreement with Boehringer Ingelheim were not disclosed.

“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, CEO of 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.”

August 6, 2018
Photo: John Dawson, CEO of Oxford BioMedica

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