… retinal disease, into the clinic and continuing the development of our portfolio of transformative RNA medicines … ProQR. “QR-421a has shown promising activity in both the optic cup and zebra fish models and we are excited about the … gene and will be conducted at expert sites in the US and Europe. It will be a double-masked, randomized study …
Partnership focused on utilizing Axiomer™ to develop editing oligonucleotides targeting an underlying genetic variant that causes Rett syndromeCollaboration expands ProQR’s commitment to advance the development of therapies for central nervous system diseases
The trial, called WINGS, will evaluate the safety and efficacy of QR-313 in subjects with recessive dystrophic epidermolysis bullosa due to mutations in exon 73 of the COL7A1 gene.