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 …
… 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 …
… “We have started testing sepofarsen, our investigational RNA therapy for Leber congenital amaurosis, in the organoids … diseases? Check out our pipeline of investigations RNA therapies. Hee Lam Chan, Principal Researcher …
Positive preliminary data from InSight extension study of sepofarsen for LCA10 reported – consistent with benefit seen in Phase 1/2 and building confidence in Phase 2/3 Illuminate trial;
Strategic convertible debt financing extends runway into 2023 if fully draw
Phase 2/3 pivotal Illuminate trial of sepofarsen for LCA10 enrollment complete with top-line data anticipated in H1 2022; Brighten pediatric trial of sepofarsen to begin in 2021; additional data from Phase 1/2 InSight extension study to be reported in
