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. With ProQR’s technology, the mutation is repaired at the RNA level, leading to normal functional protein, relieving patients from their symptoms.

QR-010, the current treatment developed by ProQR, has been granted orphan drug designation in the United States and the European Union. ProQR expects its drug to be effective in 70% of patients, compared to Vertex’s Kalydeco (ivacaftor) which only works for patients with a specific rare mutation in the CFTR gene. The IPO aims to pay QR-010’s Phase IIa trials and support the preclinical development of QR-110, an early-stage treatment for the rare Leber’s congenital amaurosis, which leads to blindness.

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Vertex Pharmaceuticals has been a long-standing leader in the race to cure cystic fibrosis. Kalydeco drug is already available to patients throughout numerous countries in Europe and scientists found in a 144-week study that it provided durable treatment effects in lung function when continuously administered. Robert Kauffman, M.D., Ph.D., Senior Vice President and Chief Medical Officer at Vertex said:

“Kalydeco was the first medicine to treat the underlying cause of CF, and we believe that Kalydeco is just the first step of our work in CF. Our goal in CF is to help many more people with this disease and to evaluate multiple combinations of CFTR modulators aimed at providing further benefit for people with CF.”

ProQR’s strategy may eventually make it a strong competitor to Vertex in the cystic fibrosis market. However, Vertex is already prepared for the counter-attack with another drug in the pipeline. A combination of experimental treatment lumacaftor with its ivacaftor showed good results in CF patients with two copies of the F508del mutation.

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