2017 may become a very exciting year for Patricia Biasutto, sepofarsen captain. Her work, developing a novel RNA therapy for Leber congenital amaurosis 10, has progressed steadily. The team expects to start a first clinical trial this year.
ProQR to host a conference call to discuss the top-line results of the Phase 2/3 Illuminate clinical trial of investigational RNA therapy sepofarsen in CEP20-mediated LCA10.
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
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.