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.
AX-0810 clinical target engagement data in healthy volunteers on track for this quarter; biliary atresia selected as initial Phase 2 indicationAdditional programs advancing toward the clinic, including AX-0811 and AX-0422Axiomer platform supporting multiple addi
Divestment of sepofarsen and ultevursen completed – Théa to continue development of sepofarsen and ultevursen for patients with LCA10 and Usher syndrome