Boehringer Ingelheim and Partners to Accelerate Development of World-Class Gene Therapy for CF Patients


  • Boehringer Ingelheim has exercised intellectual property options with IP Group regarding research results generated by the UK Cystic Fibrosis Gene Therapy Consortium and Oxford Biomedica regarding their lentiviral vector technology.

  • Partners aim to accelerate development of novel inhaled cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy BI 3720931 as a long-term treatment option for patients with cystic fibrosis (CF)

INGELHEIM, Germany, October 19, 2021– (BUSINESS WIRE) – Boehringer Ingelheim, IP Group, the British Cystic Fibrosis Gene Therapy Consortium (GTC, made up of researchers from Imperial College London and the universities of Oxford and Edinburgh) and Oxford Biomedica (OXB), today announced that Boehringer Ingelheim has exercised its options on the intellectual property and know-how of partners to advance and further accelerate the development of a potential new treatment option for patients with cystic fibrosis . As part of this partnership, IP Group, acting on behalf of the three GTC host universities, grants exclusive worldwide rights to develop, manufacture, register and commercialize this lentiviral vector-based gene therapy for the treatment of cystic fibrosis. The GTC also contributes its knowledge in preclinical research and clinical development of gene therapy. OXB adds its cutting-edge competence in the manufacture of lentiviral vector therapies to Boehringer Ingelheim’s expertise in the development of revolutionary new therapies for respiratory diseases.

Cystic fibrosis is a rare, progressive and life-threatening disease that causes severe dysfunction and persistent lung infections affecting 70,000 people worldwide. It is caused by a defective or missing protein that results from mutations in the CFTR gene. This innovative development partnership between universities, life science investors, pharmacy and biotechnology focuses on advancing BI 3720931, a new replication-deficient lentiviral vector, in an inhaled formulation, which selectively introduces a gene Healthy CFTR in the affected target cells.

Prof. Eric Alton, Coordinator of the GTC, said: “The new lung targeting technology that we have developed has demonstrated high efficiency of gene transfer in preclinical models and offers the possibility of repeated administration to maintain a therapeutic effect. , a benefit that other viruses Gene-based gene therapies might not be able to provide. Our new therapy has the potential to improve CFTR function and modify disease in all CF patients, regardless of the more than 2,000 different known genetic mutations. The immediate target is patients who are not eligible for CFTR modulators. The CWG is very pleased to have reached this milestone after 21 years of focused effort. We are very grateful to our wonderful team and the people with CF who supported us in many ways, including participating in the multiple trials. We would like to thank our funders, past and present, including the Health Innovation Challenge Fund (a partnership between Wellcome and the Department of Health and Welfare Ca re) and Just Gene Therapy, as well as the CF Trust, the National Institute for Health Research and the Medical Research Council. “

“Since 2018, Boehringer Ingelheim has sponsored research and development activities with GTC and OXB. The success shared with our partners in this potentially revolutionary project allows us to be confident that we can now further accelerate this highly innovative therapeutic approach, ”said Clive R. Wood, Ph.D., senior vice president of the company. and Global Head of Discovery Research at Boehringer Ingelheim. “With our leadership in the discovery and development of therapies for respiratory disease, combined with gene therapy and manufacturing knowledge from our partners, we aim to bring the next step forward for CF patients, who are desperately waiting for better options.” . “

John Dawson, CEO of Oxford Biomedica, said: “We have enjoyed working with Boehringer Ingelheim, IP Group and GTC since 2018. Building on the great progress made to date, we are delighted that Boehringer Ingelheim, one of the world leaders, large respiratory medicine organizations, has chosen to exercise the license option for the manufacturing technology of OXB lentiviral vectors for this highly innovative inhaled gene therapy formulation for cystic fibrosis developed by the GTC. has the potential to provide a new treatment option for many cystic fibrosis patients around the world. “

Under the terms of the option and license agreement with Boehringer Ingelheim, initially announced in August 2018, Boehringer Ingelheim will pay IP Group, on behalf of GTC, a short-term option exercise fee of sales and regulatory success-based development milestone payments as well as net sales royalties. OXB will receive an option exercise fee of £ 3.5million and be entitled to payouts totaling up to £ 27.5million upon achievement of various development milestones, regulation and sales, in addition to a tiered single-digit royalty on net sales. of a gene therapy product for cystic fibrosis.

David Ramsden, CEO of the Cystic Fibrosis Trust, said: “It is great news that Boehringer Ingelheim is embarking on the next step in the development of a gene therapy treatment for people with cystic fibrosis. the whole cystic fibrosis community and in particular those who do not benefit from the drugs currently available. Anyone who has helped us make a long-term investment in the work of the UK CF Gene Therapy Consortium should be proud of what they have made possible. “

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Boehringer Ingelheim
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