… to changing lives through the creation of transformative RNA therapies for inherited retinal diseases (IRDs), today … retinal degenerations, autoimmune retinopathy, and gene therapy associated uveitis. He is a principal investigator …
… to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare … lead to blindness in early childhood. No form of therapy is currently available,” said Dr. Katarina Stingl of … Tübingen, Investigator in the ILLUMINATE trial. “ProQR’s RNAtherapy for the p.Cys998X mutation showed the potential …
Study did not meet primary endpoint nor notable secondary endpoints – no benefit observed in either treatment arm versus shamAdditional analyses to be conducted and presented at a future medical congressSepofarsen continues to be generally well tolerated
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