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.
Le QR-110 a permis une amélioration rapide et soutenue de la vision chez la majorité des sujets, tant en termes d’acuité visuelle que de mobilité Le QR-110 a été bien toléré sans événements indésirables graves