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Boehringer Ingelheim to accelerate development of cystic fibrosis gene therapy

03 Nov 2021

Boehringer Ingelheim has exercised options arising from an agreement facilitated by Edinburgh Innovations to develop a new long-lasting therapy for patients with cystic fibrosis.

The German biopharmaceutical company formed a partnership in 2018 with the UK Cystic Fibrosis Gene Therapy Consortium (GTC), of which the University of Edinburgh is a member, and Oxford Biomedica, to develop a new lentiviral vector-based therapy.

It has now exercised an option on intellectual property and know-how from the partners to progress and accelerate the development of a potential new treatment for patients.

Industry-academic partnership

IP Group, acting on behalf of the three GTC host universities – Edinburgh, Oxford and Imperial College London – is granting exclusive global rights to develop, manufacture, register and commercialise BI 3720931, an inhaled cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy.

The GTC is additionally contributing its knowledge in pre-clinical research and clinical gene therapy development.

Oxford Biomedica is providing its expertise in manufacturing lentiviral vector-based therapies, complementing Boehringer Ingelheim’s record in the development of novel breakthrough therapies for respiratory diseases.

Life-threatening disease

Cystic fibrosis 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 between academia, life science investors, pharma, and biotech focuses 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.

The formulation of BI 3720931 builds on nearly two decades of research by the GTC to develop a clinically viable treatment for people with cystic fibrosis.

Edinburgh research

University of Edinburgh Principal Investigators Dr Chris Boyd and Dr Gerry McLachlan have been GTC members since its inception and serve on its Strategy Group. Both were involved in the pre-clinical work culminating in the successful Phase IIb trial of a non-viral gene therapy formulation for cystic fibrosis. Their efforts since then have been focused on contributing to the development of the lentivirus-based vector system.

Dr Boyd leads the Gene Therapy Group in the University’s Centre for Genomic and Experimental Medicine, which has investigated the distribution of lentiviral insertion sites in lung-derived cells.

The decision by Boehringer Ingelheim to exercise an intellectual property option concerning the GTC's lentiviral vector for CF gene therapy is a significant milestone which will greatly facilitate the product's further development. It is a vindication of how successful the collaboration between Imperial, Oxford and Edinburgh has been in its ability to work together towards this translational goal for many years. We look forward to continuing to work alongside BI and Oxford Biomedica as we progress towards clinical studies.
Dr Chris Boyd, Reader, the Centre for Genomic and Experimental Medicine.

Dr McLachlan leads a group in the Roslin Institute, which has focused on evaluating the delivery, safety and efficacy of the vector system in large animal models.

Our goal in working with our GTC collaborators on a gene therapy for cystic fibrosis was always to reach the stage where a commercial partner would seek to take the product forward, through clinical trials, and eventually to deliver benefit for patients in the clinic. We are really excited to have reached this stage, and grateful to our partners, funders and everyone involved in this research.
Dr Gerry McLachlan, Senior Research Fellow, the Roslin Institute.

EI support

The collaboration between Boehringer Ingelheim, the GTC and Oxford Biomedica was formed in 2018 with Edinburgh Innovations’ support. Working in concert with Imperial and Oxford, EI and the University’s legal team negotiated the contracts for the partnership, and EI provided support for the intellectual property generated by the Edinburgh researchers in the GTC.

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.
David Ramsden, Chief Executive of the Cystic Fibrosis Trust.