Wings Therapeutics is conducting its Phase Ib/II clinical trial of QR-313 for the treatment of both Dominant Dystrophic Epidermolysis Bullosa (DDEB) and Recessive Dystrophic Epidermolysis Bullosa (RDEB). QR-313 is an investigational RNA therapy candidate for the treatment of DEB due to exon 73 mutations.
Primary objectives of the study are to test whether QR-313 is safe and well-tolerated and to evaluate proof of mechanism by testing, at a molecular level, whether QR-313 is able to exclude the mutated exon from the messenger RNA. Secondary objectives are to test the effects on wound healing, skin strength, the presence of collagen type VII protein and anchoring fibrils in the skin as well as systemic distribution of QR-313 after topical administration.
- Male or female, ≥ 4 years of age at Screening with a clinical diagnosis of DDEB or RDEB and at least one pathogenic mutation in exon 73 of the COL7A1 gene.
- Have at least one TWA, ie, a skin area of 7 x 7 cm that ishows no signs of local infection, and contains a target wound that is either new or shows dynamic wound healing and complies to the following additional criteria:
- surface area of the target wound ranging from 5 to 30 cm2, located centrally in the selected 7 x 7 cm TWA.
- exposed sub-epidermal tissue to allow absorption of the IMP.
- no suspicion of current squamous cell carcinoma (SCC) upon visual inspection
- Please click the link below for exclusion criteria.