We were founded in May 2012 in Leiden the Netherlands with the goal of developing a treatment for cystic fibrosis (CF). The founding team - Daniel de Boer, Gerard Platenburg, Dinko Valerio and Henri Termeer (28 February 1946 – 12 May 2017) - pulled together a team of motivated RNA and CF experts to work on a unique RNA therapy for CF. Our innovative and promising technology was licensed from Massachusetts General Hospital in Boston, USA. Since then, we have advanced our lead program through preclinical development and into clinical trials. Beyond CF, we have built a pipeline of programs that have the potential to treat other severe genetic diseases, such as Leber's congenital amaurosis (LCA), that are based on several of our RNA technologies. Since September 2014 we are listed on NASDAQ under ticker PRQR. Currently we are with over 100 people that work with passion and dedication on developing our therapies.
Driven to treat severe genetic disorders
Changing lives is what drives us at ProQR Therapeutics. With a highly energetic team of professionals we strive to make a meaningful impact on the lives of patients and the people around them. By combining innovative technologies and entrepreneurism, we are developing products to potentially transform the lives of patients suffering from severe genetic disorders.
Novel RNA technologies
Our programs are based on our unique, proprietary RNA repair technologies that involve chemically modified single-stranded antisense RNA oligonucleotides. Our molecules are designed to target and repair the underlying genetic defect in the messenger RNA, or mRNA, of patients. We believe that this is a unique approach that offers advantages compared with small molecule, gene therapy and other therapeutic strategies.
QR-010 in clinical development for cystic fibrosis due to the F508del mutation
QR-010 is our lead compound in development, targeting cystic fibrosis (CF) and is currently being tested in first-in-human clinical trials. CF is a genetic disease that causes viscous mucus to accumulate in vital organs like the lungs. The mucus clogs tubes and organs thereby disrupting several processes in the body, with lung function the most affected. Patients experience bacterial infections that lead to frequent hospitalization and eventually severe lung damage. With QR-010 we are targeting the most common mutation in CF, called F508del, and we estimate that about 49,000 patients worldwide have CF due to this specific mutation.
We started the first clinical trials of QR-010 in CF patients in 2015. The phase 1b clinical trial will assess the safety and tolerability of QR-010 in approximately 64 patients that are homozygous for the F508del mutation. In this trial we will also look at some measures of efficacy that are commonly used to see if a medicine is beneficial in cystic fibrosis patients. The first half of the trial that tests single doses of QR-010 has now been completed. The second half of the trial, in which subjects receive 12 doses of QR-010 over 4 weeks of treatment is currently ongoing.
The second trial is a nasal potential difference, or NPD, proof-of-concept study that was completed in 2016. It enrolled 18 patients that were either homozygous or compound heterozygous for the F508del mutation. NPD is a well-accepted diagnostic test for CF and has been used to assess therapeutic benefit in clinical trials of investigational agents. Initial results from the NPD trial were presented during the North American CF Conference and in a press release on October 27, 2016 (link to press release). The trial demonstrated that QR-010 significantly improved CFTR-mediated total chloride transport, as measured by the NPD test, in patients that are homozygous for the F508del mutation. This was supported by the change in sodium channel activity as measured by the basal potential difference. In patients that were compound heterozygous for the F508del mutation, no meaningful difference was found. QR-010 was observed to be safe and well-tolerated.
QR-110 for Leber's congenital amaurosis due to the p.Cys998X mutation in CEP290
Our second program, QR-110 is the first program to come out of our innovation unit, which is our in house discovery engine, QR-110 is being developed for patients with LCA. LCA is a genetic eye disease that is the leading cause of genetic blindness in children. With QR-110 we target the most common mutation called the p.Cys998X mutation in the CEP290 gene that we believe affects approximately 2,000 patients in the Western world.