INGELHEIM, Germany—As the saying goes, 'Many hands make light work,' and a newly announced global collaboration is bringing together a plethora of hands from a number of heavy hitters in hopes of developing a treatment for cystic fibrosis.
The collaboration unites Boehringer Ingelheim, Oxford BioMedica (OXB), the UK Cystic Fibrosis Gene Therapy Consortium (which includes Imperial College London, the University of Oxford and the University of Edinburgh) and Imperial Innovations. The combination of academic and industry organizations pits extensive experience in developing gene therapies, manufacturing, drug discovery and clinical development against a genetic disease that affects roughly 70,000 individuals around the globe. The ultimate goal is a first-in-class, long-term therapy for cystic fibrosis patients.
Cystic fibrosis (CF) results from two defective copies of the gene for the cystic fibrosis transmembrane conductance regulator (CFTR). This causes the body to produce thick, sticky mucus in the lungs and pancreas, as well as other organs, resulting in difficulty breathing, frequent respiratory infections and, due to the buildup in the pancreas blocking the release of enzymes, issues with digestion. While treatments have been developed to help manage or slow the disease, leading to the average age of CF patients now being above 30, there is no cure.
A likely contributor to that lack of cure is fact that there are more than 2,000 known mutations in the CFTR gene. This makes a 'one size fits all' approach seem improbable, but gene therapy could be the answer. The partners are hoping to introduce a functional copy of the CFTR gene into the cells of the lung via a novel approach that features a replication-deficient lentiviral vector in an inhaled formulation.
“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. From the beginning the GTC identified that this goal would require incremental increases in knowledge,” Prof. Eric Alton, coordinator of the UK Cystic Fibrosis Gene Therapy Consortium, said in a press release. “We have, therefore, built on our non-viral gene therapy experience to develop a new viral vector-based product, which is currently funded by the Health Innovation Challenge Fund (a partnership between the Wellcome Trust and the Department of Health and Social Care) and the Cystic Fibrosis Trust. It is with great pleasure that we now join forces with two world-class organisations. Boehringer Ingelheim will provide its multinational industry expertise, including a rich heritage in the respiratory field, to drive the product towards the clinic, whilst Oxford BioMedica is the acknowledged leader in the field of lentiviral vector manufacturing. The GTC believes that this partnership provides CF patients with the optimal chance to establish gene therapy as routine clinical practice, relevant to all patients, irrespective of their mutation status, and in due course to both prevent lung disease as well as treat established problems. We would like to take this opportunity to warmly thank all of our fundraisers who have supported us over many years.”
The inhaled gene therapy approach has been shown to offer high gene transfer efficiency, and “offers the possibility of repeated administration to maintain the therapeutic effect,” as per a Boehringer Ingelheim press release. The GTC noted on its website that “The gene therapy may have additional benefits; currently, we envisage the effect of a single dose lasting for many months or even longer, and it is unlikely that gene therapy will suffer from drug-drug interactions.”
Imperial Innovations and Boehringer Ingelheim have established an option and license agreement under this collaboration. As noted in an Imperial Innovations press release, “As the technology transfer partner for the GTC, Innovations has worked with the Consortium and its collaborators to build up an intellectual property portfolio around the developing therapy over several years. This portfolio covers the discoveries and knowledge accumulated by the group and is the subject of the agreement with Boehringer Ingelheim, granting the pharmaceutical company global rights to develop, manufacture, register, and commercialize this lentiviral vector-based gene therapy for the treatment of cystic fibrosis. The commercial license is subject to milestones and royalties, dependent on the successful development of the potential new treatment.”
Oxford BioMedica and Boehringer Ingelheim have inked a separate option and license agreement, under which the latter has an option to license the exclusive global rights over Oxford BioMedica' lentiviral vector technology for the manufacture, registration and commercialization of the CF gene therapy. Financial details were not released. Oxford BioMedica will be in charge of process and analytical development, scale up of manufacture of the candidate and generation of material for toxicology studies for this collaboration, per a company press release.
“Through this collaboration, we are joining forces with some of the top talents in this disease space to propel treatment advances forward,” commented Dr. Clive R. Wood, senior corporate vice president, Discovery Research at Boehringer Ingelheim. “Bringing together our existing expertise as a leader for nearly a century in the discovery and development of therapies that have advanced patient care in respiratory diseases with the gene therapy knowledge of our partners, we aim to unlock unprecedented opportunities for patients with this devastating disease, who are desperately waiting for better treatment options.”