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Boehringer Ingelheim Licenses Global Rights to Cystic Fibrosis Gene Therapy

Boehringer Ingelheim joins Oxford BioMedica, UK Cystic Fibrosis Gene Therapy Consortium, and Imperial Innovations to form a partnership for developing a new gene therapy to treat cystic fibrosis.

Boehringer Ingelheim has signed an option and license agreement with Oxford BioMedica, a cell and gene therapy company, for exclusive global rights over Oxford BioMedica’s lentiviral vector technology to manufacture, register, and commercialize a lentiviral vector-based gene therapy for treating cystic fibrosis (CF), Oxford BioMedica said in an Aug. 6, 2018 press release. The agreement is part of a partnership formed between Boehringer Ingelheim, Oxford BioMedica, UK Cystic Fibrosis Gene Therapy Consortium (GTC), and Imperial Innovations, the technology transfer office for Imperial College London. The financial terms for the option and license agreement with Boehringer Ingelheim were not disclosed.
In addition to the option and license agreement, Oxford BioMedica has entered into a process development collaboration agreement with GTC and Imperial Innovations to develop a long-term therapy for CF.

The collaboration will use a new, replication-deficient lentiviral vector in an inhaled formulation to selectively introduce a cystic fibrosis transmembrane conductance regulator (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 2000 different, known gene mutations across patients equally well, offering a disease-modifying treatment option for all patients, the company reports.

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. The parties may also 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.
Treating CF patients with a gene therapy requires a large volume of lentiviral vectors, which Oxford BioMedica can produce using its GMP-compliant manufacturing process in bioreactors.
“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. 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,” said John Dawson, CEO of Oxford BioMedica, in a company press release.
“Our contribution to this partnership reaffirms our leading position in the development and manufacture of lentiviral vector gene therapy products at large scale. We look forward to working with our new academic and industry partners,” he added.

“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. We have developed a novel 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. Boehringer Ingelheim will provide their multinational industry expertise, including a proven track record in the respiratory field, to drive the product towards the clinic, whilst Oxford BioMedica is the acknowledged leader in the field of lentiviral manufacturing. The GTC believes that this partnership provides CF patients with the optimal chance to establish gene therapy as routine clinical practice. We would like to take this opportunity to warmly thank all of our fundraisers who have supported us over many years,” said Deborah Gill, professor of gene medicine at the Department of Medicine of Oxford University, in a company press release.
“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, senior corporate vice president, Discovery Research at Boehringer Ingelheim, in the press release.

“The UK Cystic Fibrosis Gene Therapy Consortium shows the power of world-class academic groups collaborating to develop advanced potential therapies. We were delighted to have been chosen as the technology transfer partner for the GTC and have worked closely with them during the development of this potential new gene therapy, securing the intellectual property necessary to drive forward its commercial development and to support the collaboration with Boehringer Ingelheim and Oxford BioMedica. The combination of expertise and resources realized by this deal will give the project a unique opportunity to develop an advanced therapy that could significantly impact the lives of thousands of people living with cystic fibrosis, and we are delighted to have played the lead role in securing this partnership,” said Andrew Tingey, director of healthcare licensing at Imperial Innovations, in the press release.