The EFF is a growing multiple stakeholder platform for the exchange and sharing of insights and innovations to drive the future of clinical development in inherited retinal diseases. Organized by ProQR
… 2/3 Illuminate trial of sepofarsen for CEP290 -mediated Leber Congenital Amaurosis 10 (LCA10) due to the p.Cys998X … are secondary endpoints in the Illuminate trial. About Leber Congenital Amaurosis 10 (LCA10) Leber congenital …
… trial results of intravitreal sepofarsen RNA therapy in Leber congenital amaurosis 10 (LCA10) Presenter: Stephen R. … testing (FST) to assess sepofarsen patient response in Leber congenital amaurosis type 10 (LCA10) Presenter: Allen …
… call on July 20, 2020 at 5:00 pm EDT, which will focus on Leber congenital amaurosis 10 (LCA10) and the investigational … RNA therapy designed to address the underlying cause of Leber congenital amaurosis 10 due to the p.Cys998X mutation …
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
QR-110 demonstrated rapid and sustained improvement in vision in the majority of subjects, as measured by visual acuity and mobility course
QR-110 was well-tolerated with no serious adverse events
… (IVT) sepofarsen (QR-110) antisense oligonucleotide in Leber congenital amaurosis 10 (LCA10) due to p.Cys998X … RNA therapy designed to address the underlying cause of Leber congenital amaurosis 10 due to the p.Cys998X mutation …
ProQR will focus exclusively on the development of the Axiomer® RNA editing technology platform across multiple therapeutic areas. This comes following feedback from the European Medicines Agency (EMA) related to ProQR’s sepofarsen for Leber Congenital Amaurosis type 10 (LCA10).
There are currently around 160 people working at ProQR, but that wasn’t always the case. When Hee Lam Chan joined in 2013, she was only employee number six. As a result, she has seen the company grow and develop.
