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Eluforsen for cystic fibrosis

ProQR is developing an investigational product, eluforsen (QR-010) for people with cystic fibrosis (CF) that have the F508del mutation. F508del is the most common mutation in CF and affects more than 85% of all CF patients. Despite significant efforts in drug development, people with CF still have a limited life expectancy. Eluforsen is being developed as a regularly inhaled therapy. Unlike any other CF drug currently on the market, it is designed to bind specifically to the defective CFTR RNA and to restore the function of the CFTR protein. With this we aim to treat the underlying cause of the disease and stop the progression of cystic fibrosis. Two global clinical trials for eluforsen in people with CF have been completed. Study 001, a Phase 1b safety and tolerability clinical trial in 70 people with CF due to two copies of the F508del mutation (homozygotes) and Study 002, a proof of concept clinical trial in 18 people with CF due to one or two copies of the F508del mutation (compound heterozygotes and homozygotes). In both clinical trials eluforsen was observed to be safe and well-tolerated and both trials showed encouraging signals that eluforsen has the potential to be a meaningful therapy for people with CF that have two copies of the F508del mutation (homozygotes). Read the interview with Daniel de Boer and Noreen Henig on the eluforsen development program and our patient centric approach. A more detailed explanation of the preliminary top-line results were published in press releases on September 25, 2017 (results for Study 001) and October 27, 2016 (results for Study 002).

Eluforsen has received orphan drug designation from the U.S. Food and Drug Administration and European Medicines Agency and was granted fast-track status by the U.S. Food and Drug Administration. The eluforsen project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No. 633545.

Clinical trials & participation

The first clinical trials of eluforsen were completed with encouraging results and currently there are no trials ongoing for eluforsen. The first trials were necessary to move to the next phase of trials designed to evaluate the safety and efficacy of an investigational drug over a longer duration. Please check this website as we will post updates on our plans for further clinical trials here. Select a study for more detailed information.

Programs for other CF causing mutations

Building on the progress in our eluforsen program for CF caused by the F508del mutation we are pursuing therapeutics for other CFTR mutations that are amenable to RNA technologies. We are exploring the possibilities to target stop-codon (or Class I) mutations in the CFTR gene including the W1282X, G542X, R553X, 621+1G->T and 1717-1G->A mutations. These programs are currently in early (discovery) stage of development.

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