Community stories and news

In top-line analyses, the Illuminate study did not meet its primary endpoint of Best Corrected Visual Acuity (BCVA) and secondary endpoints. No benefit was observed in either sepofarsen treatment arm versus the sham treated control arm.

Even in lockdown we have been working hard as ProQRians to stay connected to the inherited retinal disease community. Rare Disease Day is a unique awareness day due to its global reach and significance.

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.

The potential of this new technology for RNA therapeutics is immense, says Bart Klein, who was at the forefront of the Axiomer® technology development. ProQR’s Sr. Vice President Innovation makes his point about the promise of Axiomer® technology: “This opens up a whole new way of designing medicines for genetic diseases that were previously ‘out of reach’ for existing technologies.”

2017 may become a very exciting year for Patricia Biasutto, sepofarsen captain. The work of her department, aimed at developing a novel drug for Leber congenital amaurosis 10 (LCA10) patients, has progressed steadily. The team is expecting to start a first clinical trial in patients this year.