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.
… Board with Leaders in Inherited Retinal Disease and RNATherapy LEIDEN, Netherlands & CAMBRIDGE, Mass., July 21, 2020 … to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today …
… diagnosed with a rare disease, he started ProQR to develop RNA therapies for rare diseases. Before founding ProQR, … In 1992 Dr. Valerio was appointed professor of gene therapy at the University of Leiden, where he also received …
At ProQR science is literally at the heart of what we do. Our passion lies in discovering the latest RNA technologies and translating them to life-changing treatments.
He is one of the founders of ProQR, but even long before that Gerard Platenburg was involved in the development of RNA therapies. Even so, after decades of working on them he still gets enthusiastic when talking about RNA.
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.
