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 …
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.
This month several ProQRians will attend the Oligonucleotide and Peptide Therapeutics Conference (TIDES Europe) 2022 to highlight our Axiomer® RNA-editing platform technology.
There are currently around 160 people working at ProQR, but that wasn’t always the case. When Hee Lam Chan joined in 2013, she was only employee number six. As a result, she has seen the company grow and develop.
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
