Wings Therapeutics is developing an investigational drug called QR-313 for people that have DEB due to a mutation in a specific part of the COL7A1 gene called exon 73. We estimate there are approximately 2,000 patients in the Western world that have DEB due to a mutation in this specific part of the gene. QR-313 is designed to exclude exon 73 from the COL7A1 RNA. This approach is also known as exon skipping.
RNA and C7 protein
RNA is the “blueprint” for protein synthesis. Skipping of exon 73 in the “blueprint” is expected to lead to a shortened but functional C7 protein. By restoring the functionality of C7 protein QR-313 aims to bring back the anchoring fibrils and strengthen the skin. This should result in better wound healing and prevent blistering. QR-313 is a water based gel (hydrogel) that will be applied directly onto DEB wounds (topical administration).
QR-313 has received orphan drug designation from the U.S. Food and Drug Administration and European Medicines Agency.
Programs for other mutations
Beyond QR-313 Wings Therapeutics is working on multiple other programs targeting other mutations causing DEB.