Several organisations are collaborating to develop a gene therapy that targets cystic fibrosis. Boehringer Ingelheim, Imperial Innovations, Oxford BioMedica, and the UK Cystic Fibrosis Gene Therapy Consortium will work together on the project. You can read the source press release here, on the website of Boehringer Ingelheim.
About Cystic Fibrosis (CF)
Cystic fibrosis is a lifelong genetic condition that causes a build-up of sticky mucus and can cause a range of symptoms. It is caused by an alteration to a gene that is involved in water movement in and out of cells. As a result, mucus can build up inside the body, and this often occurs in the lungs and digestive system. Symptoms can affect many different parts of the body and can include difficulty digesting food and nutrients, coughing, and more frequent lung infections, amongst others.
According to the source press release, an estimated 70,000 people in the world are thought to have cystic fibrosis. In addition, the Cystic Fibrosis Foundation says that over 1,700 different genetic alterations in the CFTR gene have been found to cause CF. Many existing treatments for CF are influenced in their effects by a what specific genetic alteration they have, and so new treatment options that work on all CFTR alterations could significantly help patients. Boehringer says that a gene therapy could potentially meet this need.
The Collaboration
Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium (made up of Imperial College London, Oxford University, and Edinburgh University), Imperial Innovations, and Oxford BioMedica will collaborate on the development of a gene therapy for CF. This partnership will combine each organisation’s expertise to bring together skills in drug development, research, clinical trials, and manufacture.
The organisations will work on developing an inhaled form of a replication-deficient lentiviral vector, which can be used to help put a healthy form of the CFTR gene into lung cells.