Announced today (August 11, 2022), ProQR will focus exclusively on the development of the Axiomer® RNA editing technology platform across multiple therapeutic areas. This comes following feedback from the European Medicines Agency (EMA) related to ProQR’s sepofarsen for Leber Congenital Amaurosis type 10 (LCA10).
At ProQR we strive to create an inclusive culture where everyone is valued for who they are and individual differences and contributions are recognized and appreciated. Therefore, we decided for this Pride Month June 2022, to visually demonstrate our support and acknowledgment of the LGBTQ+ community by changing our logo to incorporate the pride rainbow flag.
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.
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.