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.
… for Fuchs endothelial corneal dystrophy and Stargardt’s disease at the ARVO annual meeting. The company will also … RNA medicines for the treatment of severe genetic rare diseases, today announced that eight out of twelve …
… RNA medicines for the treatment of severe genetic rare diseases, today announced results from a planned interim … utility of RNA-based therapeutics in a human photoreceptor disease, particularly one with a severe unmet medical need. …
QR-421a showed early and encouraging evidence of activity, with 25% of patients showing a benefit across multiple concordant outcome measures and was well tolerated with no serious adverse eventsQR-421a is the second ophthalmology program where clinical activity was predicted by tra
Encouraging clinical data reported from Phase 1/2 trial of sepofarsen for LCA10
Initial clinical data from Phase 1/2 trial of QR-421a for Usher syndrome type 2 on track for Q1 2020
QR-1123 Investigational New Drug application active for autosomal dominant re
Reported rapid, significant and durable improvements in vision at twelve months
Concordant improvement in key secondary outcome measures
The target registration dose of sepofarsen was well-tolerated with a favorable benefit/risk profile
Strengthens confidence