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LEIDEN, Netherlands & CAMBRIDGE, Mass.—ProQR Therapeutics N.V., a company focused on RNA medicines for the treatment of severe genetic rare diseases, in late March announced the strategic spin out of its dystrophic epidermolysis bullosa (DEB) activities into the newly formed company Wings Therapeutics.
 
Wings Therapeutics will focus on developing therapies for DEB and continue to conduct clinical trials with QR-313 in exon 73, as well as progress other RNA molecules that are designed for other mutations that cause DEB. ProQR has a minority stake in Wings Therapeutics and will be eligible for milestone and royalty rights to commercial products. Subsequent to a planned interim analysis and strategic review, management has elected to transfer conduct and completion of the ongoing Phase 1/2 study to Wings Therapeutics. The ongoing Phase 1/2 trial in patients with DEB due to a mutation in exon 73 will remain blinded and continues to enroll patients. ProQR will work closely with Wings Therapeutics and EBRP to support its efforts to advance QR-313 for patients with DEB.
 
“DEB is a very severe disease for which we hope QR-313 one day will become a therapy for patients that suffer from it. We are pleased to have entered into this agreement to see the programs getting developed in a dedicated effort,” said Daniel A. de Boer, CEO at ProQR. “With this transaction, we’ve now fully focused our development pipeline on the ophthalmology programs in pursuit of our Vision 2023 strategic objectives.”

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Volume 15 - Issue 5 | May 2019

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