Boehringer Ingelheim Enters CF Gene Therapy Development Pact

Little girl receives breathing treatment
[Source: SDI Productions/Getty Images]

Boehringer Ingelheim (BI) is partnering for intellectual property and know-how from IP Group and Oxford Biomedica (OXB) to advance a potential gene therapy for cystic fibrosis (CF).  BI 3720931 is a novel, replication deficient lentiviral vector in an inhaled formulation.

IP Group is acting on behalf of the three UK Cystic Fibrosis Gene Therapy Consortium (GTC) host Universities (Imperial College London and the Universities of Oxford and Edinburgh).  The Group Is granting exclusive global rights to develop, manufacture, register, and commercialize a lentiviral vector-based CF gene therapy. The GTC is also contributing its knowledge in pre-clinical research and clinical gene therapy development. OXB is adding its competence in manufacturing lentiviral vector-based therapies.

CF is a rare, progressive, life-threatening disease that results in severe dysfunction and persistent infections of the lung affecting 70,000 people worldwide. It is caused by a defective or absent protein that results from mutations in the CFTR gene. Nearly 90% of people with CF have at least one copy of the ΔF508 mutation, but there are hundreds of CFTR mutations which cause a range of disease severity. After discovery of the CFTR gene in 1989, it was quickly established that gene therapy was a viable option for this disease. Since then, however, there have been dozens of attempts at this without success.

Professor Eric Alton, Coordinator of the GTC, said: “The novel lung-targeting technology we have developed has demonstrated high gene transfer efficiency in pre-clinical models and offers the possibility of repeated administration to maintain a therapeutic effect, a benefit that other viral-based gene therapies may not be able to provide. Our novel therapy has the potential to improve CFTR function and modify disease in all CF patients, independent of the more than 2,000 different known gene mutations.”

He added that “the immediate target is those patients who are not eligible for CFTR modulators. The GTC is very excited to have reached this milestone after 21 years of focused effort. We are very grateful to our wonderful team and those with CF who have supported us in many ways including taking part in the multiple trials.”

Under the terms of the option and license agreement with Boehringer Ingelheim, originally announced in August 2018, BI will pay IP Group, on behalf of the GTC, an option exercise fee, near term, success-based development, regulatory and sales milestone payments as well as royalties on net sales. OXB will receive an option exercise fee of £3.5 million ($4.83 million) and will be entitled to payments in an aggregate amount of up to £27.5 million ($38 million) upon achievement of various development, regulatory, and sales milestones.

“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,” said Clive R. Wood, Ph.D., corporate senior vice President and global head of Discovery Research at BI.

David Ramsden, chief executive of the Cystic Fibrosis Trust said: “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.  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.”

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