ProQR Therapeutics is a Netherlands-based biotech company focused on developments of drugs to treat genetic disorders. Daniel de Boer, Gerard Platenburg, Dinko Valerio and Henri Termeer (former Genzyme’s CEO) founded the company in May 2012 with the main goal of finding a treatment for cystic fibrosis. ProQR has now expanded to more than 50 employees who are working toward this cause. The $75 million IPO is aiming to give advantages to ProQR against his main competitor, the US-based company Vertex Pharmaceuticals.
Cystic fibrosis (CF) is a genetic disease affecting more than 70.000 patients worldwide. CF patients have viscous mucus accumulating in their vital organs. The mucus clogs tubes and organs thereby disrupting several processes in the body. ProQR’s aims to relieve patients from their symptoms by repairing the basic defect underlying the disease. CF is caused by mutations on the CFTR (CF transmembrane conductance regulator) gene in the DNA, this mutation is then copied in the RNA leading to a non-functional CFTR protein that causes the severe symptoms in patients.