A new partnership in the UK will develop a gene therapy for cystic fibrosis that could treat the disease with a single dose.
Cystic fibrosis is a genetic disease that causes mucus to build up in a patient’s lungs. As a result, patients suffer from blocked airways and bacterial infections. While treatment advances have increased the life expectancy of patients, most of them only live to the age of 40. Furthermore, there is still no cure for the disease.
Now, a set of industry collaborations could help bring a gene therapy developed by the UK Cystic Fibrosis Gene Therapy Consortium into clinical testing. The treatment uses a type of virus called a lentivirus to deliver a healthy copy of a gene called CFTR, which causes cystic fibrosis when it carries a mutation. The gene therapy will be given by inhalation to better target the right cells.