ProQR Therapeutics, a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced a collaboration with nonprofit organizations EB Research Partnership (EBRP) and EB Medical Research Foundation (EBMRF) to develop QR-313 for patients with dystrophic epidermolysis bullosa (DEB) caused by mutations in exon 73 of the COL7A1 gene. Under the agreement, EBRP and EBMRF will provide up to approximately $5 million in matching funding to ProQR under a venture philanthropy model, to co-fund the clinical development of QR-313.
“The EB Research Partnership and EB Medical Research Foundation are exceptional organizations and we look forward to collaborate on our mission to develop a life changing treatment for patients with this devastating disease," said Daniel A. de Boer, Chief Executive Officer of ProQR. “With the funding from these partners, we plan to accelerate the development of QR-313 for the treatment of DEB. And if we are successful with QR-313, we believe there is potential to expand this approach of exon skipping into other mutations that cause DEB and help even more patients.”
DEB is a rare debilitating skin disease caused by the absence of a protein called collagen type VII (C7). Symptoms are present from birth and include easy blistering of the skin, poorly healing wounds, skin infections and, in adulthood, some patients develop very aggressive forms of skin cancer. There are currently no approved treatment options for DEB. The WINGS trial, a first-in-human Phase 1/2 clinical trial of QR-313 in patients with DEB, is expected to initiate enrollment of patients in the first half of 2018. The trial is expected to report interim proof of mechanism results later this year, with full results expected in 2019.
“DEB has a huge impact on patients' quality of life and there are currently no approved disease modifying treatment options beyond wound care," said Alex Silver, founder and Chairman of EBRP and Paul Joseph, CFO of EBMRF. "Our organizations are excited to partner with the ProQR team, in the hopes that the development of QR-313 will provide a much-needed treatment option to this community.”