ProQR has announced the completion of an in-depth strategic review designed to deliver on our commitment to advance RNA therapies for diseases with high unmet need. We are also sharing additional analyses of the sepofarsen Illuminate trial.
On behalf of all ProQRians, happy Rare Disease Day 2022. Both within ProQR and outside, we join the community in raising awareness and trying to generate change for people worldwide living with a rare disease, their families, and supporters.
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.
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.
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.
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.
Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for lead program AX-0810 targeting NTCP for Cholestatic diseases Company expects up to four clinical data readouts in 2025 and 2026 across multi
