Boehringer, Partners Boost Gene Therapy Development For CF

Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium (GTC), and Oxford Biomedica (OXB) will further accelerate the development of a potential new treatment option for patients with cystic fibrosis (CF).

This partnership among academics, life science investors, pharma, and biotech, will advance BI 3720931. BI 3720931 is a novel, replication-deficient lentiviral vector that introduces a healthy CFTR gene into the relevant target cells of individuals with CF. 

IP Group, acting on behalf of the three researchers from GTC, including Imperial College London and the Universities of Oxford and Edinburgh, will grant exclusive global rights to develop, manufacture, register, and commercialize BI 3720931.

The GTC will contribute its knowledge in preclinical research and clinical gene therapy development. At the same time, OXB will add its strong capability in manufacturing therapies to Boehringer Ingelheim’s expertise in developing novel therapies for respiratory diseases. 

“With our leadership in the discovery and development of therapies in respiratory diseases combined with the gene therapy and manufacturing knowledge of our partners, we aim to bring the next breakthrough to patients suffering from CF, who are desperately waiting for better options,” Clive R. Wood, PhD, corporate senior vice president and global head of discovery research at Boehringer Ingelheim, said in the announcement. 

Under the terms of the agreement, Boehringer Ingelheim will pay the IP group an option exercise fee, near term, success-based development, regulatory, sales milestone payments, and royalties on net sales.

OXB will receive an option exercise fee of $4.7 million US dollars and can qualify for payments of up to $32 million based on achievement of development, regulatory, and sales milestones. 

CF is a rare, progressive, life-threatening disease that results in severe dysfunction of the lung. 

About 70,000 individuals are affected by the disease globally. There are nearly 2,000 known mutations of the CFTR gene. But the most common mutation is F508del. 

There is currently no cure for CF. But treatments can reduce symptoms and complications and improve the quality of life for those living with the disease. 

“It is great news that Boehringer Ingelheim have committed to the next stage of the development of a gene therapy treatment for people with cystic fibrosis. This is an important step as it brings hope to the whole cystic fibrosis community and in particular to those who don’t benefit from the currently available medicines,” David Ramsden, chief executive of the Cystic Fibrosis Trust, said in the recent announcement. 

“All of those who have helped us to invest long term in the work of the UK CF Gene Therapy Consortium should be proud of what they have made possible,” Ramsden continued.