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Boehringer Ingelheim takes its first step into gene therapy

Boehringer Ingelheim office

The company have together with the UK Cystic Fibrosis Gene Therapy Consortium (GTC), consisting of Imperial College London and the Universities of Oxford and Edinburgh, Imperial Innovations, and Oxford BioMedica (OXB) announced a global collaboration to develop a first-in-class, long-term therapy for patients with cystic fibrosis (CF).

The new partnership brings together the academic partners’ leading expertise in developing gene therapy for CF and OXB’s leading expertise in manufacturing lentiviral vector-based therapies with Boehringer Ingelheim’s capabilities in drug discovery and the clinical development of novel breakthrough therapeutic agents, reports the collaborators.

A possible universal treatment option

The collaboration will focus on a novel approach using a replication-deficient lentiviral vector in an inhaled formulation, to introduce a healthy copy of the CFTR gene into the cells of the lung. This method has demonstrated high gene transfer efficiency and offers the possibility of repeated administration to maintain the therapeutic effect. Gene therapy is the only therapeutic approach to date that can address all CFTR gene mutations, thus potentially offering a universal treatment option.

Three-way partnership

“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,” stated John Dawson, Chief Executive Officer of Oxford BioMedica. “The GTC has been working determinedly for over 15 years to get to this exciting point of forming a partnership with Boehringer Ingelheim, a global pharmaceutical company with respiratory expertise. Our contribution to this partnership reaffirms our leading position in the development and manufacturing of lentiviral vector gene therapy products at large scale. We look forward to working with our new academic and industry partners.”

“Through this collaboration, we are joining forces with some of the top talents in this disease space to propel treatment advances forward,” said Clive R. Wood, Ph.D., 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.”

Collaboration details

Boehringer Ingelheim has received an option to license the exclusive global rights to develop, manufacture, register, and commercialise this lentiviral vector-based gene therapy for the treatment of cystic fibrosis. Financial terms are not disclosed. During the option period the partners will work closely together to pursue the development of this innovative approach financed by Boehringer Ingelheim. The new collaboration is a joint initiative of Boehringer Ingelheim’s Respiratory Therapeutic Area and Research Beyond Borders (RBB), which is one of the pillars of Boehringer Ingelheim’s research and development strategy and explores emerging science, disease areas and technology. It builds on Boehringer Ingelheim’s commitment to early science and its comprehensive approach to respiratory research and development to investigate new treatments that have significant clinical value in areas of high unmet need.

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