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 RNA Therapy LEIDEN, Netherlands & CAMBRIDGE, Mass., July 21, … to changing lives through the creation of transformative RNA therapies for severe genetic rare diseases, today … of leading experts in inherited retinal disease and RNA therapies,” said Daniel A. de Boer, Chief Executive …
ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need.
ProQR's leadership is a strong team with proven track record. Find out who is on our management team, board of directors and scientific advisory board.
… company dedicated to changing lives by developing RNA therapies for severe rare and common diseases. We focus … and Cambridge, Massachusetts, USA. Learn more about RNA therapy and our Axiomer technology . We are on a mission …
… Always excited about RNA therapies He is one of the founders of ProQR, but even … that Gerard Platenburg was involved in the development of RNA therapies. Even so, after decades of working on them he … as they say. Gerard Platenburg What made you pursue RNA therapy as a potential treatment for these diseases? …
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.
