European Partners Combining Efforts to Develop New Gene Therapy for Cystic Fibrosis

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by Diogo Pinto |

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Pharmaceutical companies Boehringer Ingelheim and Oxford BioMedica are teaming up with the U.K. Cystic Fibrosis Gene Therapy Consortium (GTC) and Imperial Innovations in a collaborative effort to develop a first-in-class, long-term gene therapy to treat cystic fibrosis (CF).

The GTC is made up of researchers at Imperial College London and the Universities of Oxford and Edinburgh. This new collaboration will combine the consortium’s leading expertise in gene therapy with Boehringer’s knowledge on drug discovery and clinical development and Oxford Biomedica’s skills in the production of lentiviral vector-based therapies, a method used in gene therapy to insert or modify genes using the lentivirus.

Imperial Innovations, the technology transfer office of Imperial College London, will assist with securing the necessary intellectual property of the collaborative effort.

“The U.K. 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,” Eric Alton, coordinator of the GTC, said in a press release.

“From the beginning the GTC identified that this goal would require incremental increases in knowledge. 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 organizations,” Alton said.

The goal of the partnership is to create a new gene therapy based on a lentiviral vector (carrier), modified so it cannot replicate the virus, containing a healthy copy of the CFTR gene, which is the gene that is defective in CF patients.

It will be developed as an inhaled formulation to be delivered into the cells of the lungs. This delivery method has demonstrated high clinical effectiveness, and allows the possibility of repeated administrations to maintain its therapeutic effects.

Gene therapy is currently the only therapeutic solution that has the potential to address all CFTR gene mutations; therefore the group believes this approach offers a universal treatment option for all patients with the disease.

“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 CF,” said John Dawson, CEO of U.K.-based Oxford BioMedica.

“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,” he said.

Germany-based Boehringer has the option to license the exclusive global rights to develop, produce, register, and commercialize this lentiviral-based therapy for CF, although the financial terms have not been disclosed.

“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,” said Clive R. Wood, PhD, Boehringer’s senior corporate vice president of discovery research.