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
… RNA medicines for the treatment of severe genetic rare diseases, today announced results for the second quarter … cause loss of functional usherin protein that causes the disease. QR-421a is designed to exclude the genetic defect …
… of transformative RNA therapies for severe genetic rare diseases, today reported its financial results for the … with a clinical diagnosis of an inherited retinal disease, such as LCA and Usher syndrome. Financial Highlights …
Substantial progress in 2019 with final data from Phase 1/2 trial of sepofarsen for Leber’s congenital amaurosis 10 showing rapid, significant and durable improvements in vision; Phase 2/3 pivotal trial Illuminate initiated with data expected in H1 2021Phase 1/2 St