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).
ProQR has announced the completion of an in-depth strategic review designed to deliver on our commitment to advance RNA therapies for diseases with high unmet need. We are also sharing additional analyses of the sepofarsen Illuminate trial.
The EFF Spring Virtual Summit hosted by ProQR on April 15 is a virtual meeting featuring the latest clinical data for USH2A mediated Usher syndrome and retinitis pigmentosa and a live panel discussion.
ProQR shared additional analyses from the Phase 2/3 Illuminate trial of sepofarsen for LCA10 and provided an update on the company strategy. Management to host a conference call today at 8:00am EDT (14:00 CET).
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
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.