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
Bart Klein was at the forefront of ProQR's Axiomer® RNA editing technology development. This platform technology makes it possible to use the cells own machinery to reverse mutations that cause a genetic disease. Read the interview.
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.
Submitted CTA for lead program AX-0810, targeting NTCP for cholestatic diseasesAdvancing AX-2402 program toward clinical candidate selection, targeting MECP2 (R270X) for Rett SyndromeHosting fall Analyst and Investor Event (virtual), featuring detailed AX-0810 P
