Boehringer Ingelheim has entered a global partnership with the UK Cystic Fibrosis Gene Therapy Consortium (GTC), Imperial Innovations and Oxford BioMedica (OXB) to develop a new long-term gene therapy for cystic fibrosis (CF).
The alliance combines the expertise of the GTC in CF gene therapy development, OXB’s experience in lentiviral vector-based therapies manufacturing and Boehringer’s drug discovery and clinical development capabilities.
Cystic fibrosis is a genetic disorder characterised by persistent lung infections and breathing difficulty.
More than 2,000 mutations are known to be associated with the gene for the cystic fibrosis transmembrane conductance regulator (CFTR), and the majority of these mutations lead to the disease.
Existing therapies and treatments under development are said to only slow the disease progression. They also have different effects based on the mutation status of a patient.
The new alliance is intended to address the unmet need for drugs that target all CFTR mutations; gene therapy is believed to possess such potential.
The partners will work towards creating a new gene therapy for cystic fibrosis that will introduce a healthy CFTR gene copy into the lung cells by using a replication-deficient lentiviral vector in an inhaled formulation.
Boehringer Ingelheim Discovery Research senior corporate vice-president Clive Wood said: “Through this collaboration, we are joining forces with some of the top talents in this disease space to propel treatment advances forward.
“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.”
Boehringer has obtained the option to licence exclusive global rights to develop, manufacture, register and commercialise the lentiviral vector-based gene therapy for cystic fibrosis treatment.
During the option period, the company will fund the development project. The financial terms of the collaboration have not been disclosed.
Oxford BioMedica will manufacture the viral vector, while the GTC will be responsible for developing and completing preclinical studies, as well as establishing a package to support clinical trials for the gene therapy for cystic fibrosis.