Boehringer Ingelheim (BI) has entered into a landmark global collaboration with the UK Cystic Fibrosis Gene Therapy Consortium and Oxford BioMedica to develop a pioneering gene therapy treatment for cystic fibrosis (CF). This partnership marks the German pharmaceutical company's first venture into gene therapy development.
The collaboration unites three powerhouse organizations: the UK consortium (comprising Imperial College London, University of Oxford, and University of Edinburgh), Oxford BioMedica's manufacturing expertise, and Boehringer Ingelheim's extensive drug development capabilities.
Novel Therapeutic Approach
The partnership's innovative approach centers on developing an inhaled formulation using a replication-deficient lentiviral vector to deliver functional copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene directly to lung cells. Early studies have demonstrated high gene transfer efficiency, with the potential for repeated administration to maintain therapeutic effects.
This gene therapy approach represents a significant advancement in CF treatment, as it could potentially address all CFTR gene mutations – making it the first truly universal treatment option for CF patients.
Expert Perspectives
Professor Eric Alton, coordinator of the UK Cystic Fibrosis Gene Therapy Consortium, highlighted the significance of the collaboration: "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. It is with great pleasure that we now join forces with two world-class organisations."
John Dawson, CEO of Oxford BioMedica, emphasized the unique strengths of the 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."
Commercial and Development Terms
Under the agreement, Boehringer Ingelheim will secure exclusive global rights for development, manufacturing, registration, and commercialization of the lentiviral vector-based gene therapy. While specific financial terms remain undisclosed, BI will fully fund the research during the option period.
Current Treatment Landscape
The development comes at a crucial time in CF treatment. While Vertex Pharmaceuticals has developed several drugs addressing the underlying protein dysfunction in CF, access remains challenging. In the UK, pricing discussions between Vertex and NHS England continue regarding Orkambi and future CF treatments, highlighting the need for alternative therapeutic options.
