Accelerated development of a potential treatment for cystic fibrosis

The UK Cystic Fibrosis Gene Therapy Consortium (GTC) is one of the partners in a licensing agreement with Boehringer Ingelheim.

IP Group, acting on behalf of the three GTC host universities, which includes Imperial researchers, has agreed to license  intellectual property and know-how to progress and further accelerate the development of a potential, new treatment option for patients with cystic fibrosis (CF).

The announcement was made by a group of long-standing partners that have been working together on gene therapy options for CF.

The partners include:

• Boehringer Ingelheim
• IP Group
• the UK Cystic Fibrosis Gene Therapy Consortium (consisting of researchers from Imperial College London and the Universities of Oxford and Edinburgh)
• Oxford BioMedica (OXB).

In the partnership, IP Group is granting exclusive global rights to develop, manufacture, register, and commercialize this lentiviral vector-based gene therapy for the treatment of cystic fibrosis.

The GTC is additionally contributing its knowledge in pre-clinical research and clinical gene therapy development. OXB is adding its leading competence in manufacturing lentiviral vector-based therapies to Boehringer Ingelheim’s expertise in the development of novel breakthrough therapies for respiratory diseases.

Our novel therapy has the potential to improve CFTR function and modify disease in all CF patients Professor Eric Alton National Heart and Lung Institute

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.

This innovative development partnership among academia, life science investors, pharma, and biotech focusses on the advancement of BI 3720931, a novel, replication deficient lentiviral vector, in an inhaled formulation, which selectively introduces a healthy CFTR gene into the relevant target cells. 

Professor Eric Alton, from Imperial’s National Heart & Lung Institute and 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. 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. We would like to thank our funders, both past and present, including the Health Innovation Challenge Fund (a partnership between Wellcome and the Department of Health and Social Care) and Just Gene Therapy, as well as the CF Trust, National Institute for Health Research and the Medical Research Council.”

The shared success achieved in this potentially revolutionary project makes us confident that we can accelerate this highly innovative therapeutic approach Dr Clive R. Wood Boehringer Ingelheim

“Since 2018, Boehringer Ingelheim has sponsored research and development activities with the GTC and OXB. The shared success achieved with our partners in this potentially revolutionary project makes us confident that we can now further accelerate this highly innovative therapeutic approach,” said Dr Clive R. Wood, Corporate Senior Vice President and Global Head of Discovery Research at Boehringer Ingelheim.

“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.”

This partnership is central to our company’s mission of delivering life changing gene therapies to patients John Dawson Oxford BioMedica

John Dawson, Chief Executive Officer of Oxford BioMedica, said: “We have enjoyed working with Boehringer Ingelheim, IP Group, and the GTC since 2018. Building on the great progress made to date, we are delighted that Boehringer Ingelheim, one of the world’s leading respiratory medicine organizations, has chosen to exercise the option to license OXB lentiviral vector manufacturing technology for this highly innovative inhaled cystic fibrosis gene therapy formulation developed by the GTC.

“This partnership is central to our company’s mission of delivering life changing gene therapies to patients and has the potential to provide a new therapeutic option for many cystic fibrosis patients globally.”

Under the terms of the option and license agreement with Boehringer Ingelheim, originally announced in August 2018, Boehringer Ingelheim 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 and will be entitled to payments in an aggregate amount of up to £27.5 million upon achievement of various development, regulatory and sales milestones, in addition to a tiered low single digit royalty on net sales of a cystic fibrosis gene therapy product.

This is an important step, as it brings hope to the whole cystic fibrosis community David Ramsden Cystic Fibrosis Trust

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.”

This news story was adapted from a Boehringer Ingelheim press release.