Boehringer Ingelheim and Partners to Accelerate Development of First-In-Class Gene Therapy for Patients with Cystic Fibrosis

• Boehringer Ingelheim has exercised intellectual property options from IP Group regarding research results generated by the UK Cystic Fibrosis Gene Therapy Consortium, and from Oxford Biomedica regarding their lentiviral vector technology  
• Partners aim to expedite the development of the novel, inhaled cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy BI 3720931 as a long-lasting therapeutic option for patients with cystic fibrosis (CF)

  Ingelheim, Germany, 19 October 2021 – Boehringer Ingelheim, IP Group, the UK Cystic Fibrosis Gene Therapy Consortium (GTC, consisting of researchers from Imperial College London and the Universities of Oxford and Edinburgh) and Oxford Biomedica (OXB), announced today that Boehringer Ingelheim has exercised its options on intellectual property and know-how from the partners to progress and further accelerate the development of a potential, new treatment option for patients with CF. In the partnership, IP Group, acting on behalf of the three GTC host Universities, 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.
  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, 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.”
  “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 Clive R. Wood, Ph.D., 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.”
  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. 
  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.”   About Boehringer Ingelheim Boehringer Ingelheim is working on breakthrough therapies that improve the lives of humans and animals. As a leading research-driven biopharmaceutical company, the company creates value through innovation in areas of high unmet medical need. Founded in 1885 and family-owned ever since, Boehringer Ingelheim takes a long-term perspective. Around 52,000 employees serve more than 130 markets in the three business areas, Human Pharma, Animal Health, and Biopharmaceutical Contract Manufacturing. Learn more at www.boehringer-ingelheim.com   
  Boehringer Ingelheim’s Intended Audiences Notice This press release is issued from our Corporate Headquarters in Ingelheim, Germany and is intended to provide information about our global business. Please be aware that information relating to the approval status and labels of approved products may vary from country to country, and a country-specific press release on this topic may have been issued in the countries where we do business.
  About the UK CF Gene Therapy Consortium The UK Cystic Fibrosis Gene Therapy Consortium (GTC) comprises researchers from Imperial College London and the Universities of Oxford and Edinburgh. In 2001, the groups joined together as one organisation to share expertise and funding with a single common goal of making gene therapy for CF patients a clinical reality. The GTC has undertaken six non-viral Phase 1/2a proof-of-concept studies and the first Phase 2b gene therapy trial for CF, which met its primary endpoint. In parallel, it has developed the novel lentiviral vector which is the subject of this option exercise. Further details and commentary can be found at www.respiratorygenetherapy.org.uk
  Health Innovation Challenge Fund The Health Innovation Challenge Fund was a parallel funding partnership between Wellcome and the Department of Health and Social Care.  The main goal of the partnership was to stimulate the creation of innovative healthcare products, technologies and interventions and to facilitate their development for the benefit of patients in the NHS and beyond. 
  About Oxford Biomedica Oxford Biomedica (LSE:OXB) is a leading, fully integrated, gene and cell therapy group focused on developing life changing treatments for serious diseases. Oxford Biomedica and its subsidiaries (the "Group") have built a sector leading lentiviral vector delivery platform (LentiVector^®), which the Group leverages to develop in vivo and ex vivo products both in-house and with partners. The Group has created a valuable proprietary portfolio of gene and cell therapy product candidates in the areas of oncology, CNS disorders and liver diseases. The Group has also entered into a number of partnerships, including with Novartis, Bristol Myers Squibb, Sio Gene Therapies, Orchard Therapeutics, Santen, Beam Therapeutics and Boehringer Ingelheim, through which it has long-term economic interests in other potential gene and cell therapy products. Additionally, the Group has signed a 3-year master supply and development agreement with AstraZeneca for large-scale manufacturing of the adenoviral based COVID-19 vaccine candidate, AZD1222. Oxford Biomedica is based across several locations in Oxfordshire, UK and employs more than 740 people. Further information is available at www.oxb.com     About Cystic Fibrosis 

• Cystic fibrosis is an inherited disease caused by a faulty gene. This gene controls the movement of salt and water in and out of your cells, so the lungs and digestive system become clogged with mucus, making it hard to breathe and digest food
• Cystic fibrosis affects around 70,000 people worldwide. The incidence of CF is estimated, on average, at between 1/3000 and 1/6000 in populations of European descent. Large differences exist between countries and regions regarding prevalence, incidence, available treatments, and clinical outcomes. In UK, other Western European countries, and also Northern America with Canada and USA, half of people with cystic fibrosis alive today are expected to live into their forties, thanks to earlier diagnosis and ongoing developments in care and treatments.
• There are over 10,655 people with cystic fibrosis living in the UK and the population is growing every year. 
• In terms of the nations the numbers of people with cystic fibrosis is as follows: England 8,372; Scotland 868; Northern Ireland 421 and Wales 409. 
• Of those who died from cystic fibrosis in 2019, the median age of death was 31 years of age.  
• Two million people in the UK are carrying the faulty gene without realizing it. If two carriers have children, there’s a one in four chance their child will have the condition, which slowly destroys the lungs and digestive system 
• People with cystic fibrosis often look perfectly healthy. But it’s a lifelong challenge involving a vast daily intake of drugs, time-consuming physiotherapy and isolation from others with the condition. It places a huge burden on those around them and the condition can critically escalate at any moment. 
• Half of people with cystic fibrosis alive today are expected to live into their forties, thanks to earlier diagnosis and ongoing developments in care and treatments  

About the Cystic Fibrosis Trust 

• The Cystic Fibrosis Trust is the only UK-wide charity dedicated to fighting for a life unlimited for everyone affected by cystic fibrosis.
• For confidential advice, support and information on any aspect of cystic fibrosis, including help with financial support contact the Cystic Fibrosis Trust helpline on (+44) 0300 373 1000 or 020 3795 2184.
• The work we do is only made possible by the generous donations from our supporters. Visit www.cysticfibrosis.org.uk to find out more about cystic fibrosis, the work of the Trust and how you can help our fight for a life unlimited.
• To support our fight for a life unlimited by cystic fibrosis text BEATCF to 70500 to give £5 to the Cystic Fibrosis Trust.