… RNA therapy Find out how RNA therapy works, how it differs … underlying cause of the disease. Watch: How do ProQR's RNA therapies work? A short introduction to RNA therapies Why focus on RNA therapies? Why focus on RNA therapies? RNA therapies can …
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 …
… clinical endpoints. Internal link ProQR pipeline RNA therapy An RNA therapy is designed to correct the mistake, or mutation, … away the underlying cause of the disease. Internal link RNA therapy AX-0810 cholestatic diseases (NTCP) At ProQR, we …
… a rare genetic disorder, with the objective to develop RNA therapies for rare diseases. Prior to founding ProQR, … Society and the Scientific Advisory Board of the Gene and RNA Therapy Center Tübingen, Germany. During his 25 years of … Society and the Scientific Advisory Board of the Gene and RNA Therapy Center Tübingen, Germany. During his 25 years of …
… 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? …
… Biasutto, sepofarsen captain. Her work, developing a novel RNA therapy for Leber congenital amaurosis 10, has progressed … and – eventually – blindness. ProQR is developing a novel RNA therapy for LCA10 patients, called sepofarsen. The …
