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New Partnership to Develop First-in-Class Gene Therapy for Cystic Fibrosis

Boehringer Ingelheim steps into the field of gene therapy

Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium (GTC)—consisting of Imperial College London and the Universities of Oxford and Edinburgh—Imperial Innovations, and Oxford BioMedica (OXB) announce a global collaboration to develop a first-in-class, long-term therapy for patients with cystic fibrosis (CF). The new partnership brings together the academic partners’ leading expertise in developing gene therapy for CF and OXB’s leading expertise in manufacturing lentiviral vector-based therapies with Boehringer Ingelheim’s capabilities in drug discovery and the clinical development of novel breakthrough therapeutic agents.

Cystic fibrosis is a genetic disease that causes persistent lung infections and usually limits a person’s ability to breathe over time. There are more than 2,000 known mutations in the gene for the cystic fibrosis transmembrane conductance regulator (CFTR), many of which result in a person developing CF. The disease is estimated to affect 70,000 people worldwide, with the majority of those located in the US and Europe. Existing treatments and those under development can only slow the progression of disease. As such, CF continues to be associated with considerable morbidity, mortality, and a high burden of therapy. In addition, existing treatments will have varying effects depending on a patient’s mutation status, as determined by the particular …

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