This website contains forward-looking statements about potential therapeutic uses and effects of ProQR Therapeutics’ products, including but not limited to our technologies.
At ProQR science is literally at the heart of what we do. Our passion lies in discovering the latest RNA technologies and translating them to life-changing treatments.
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.
ARVO 2020 clinical trial results presentation by Stephen Russel et al. Sepofarsen had a manageable safety profile and showed improvement in mean visual acuity, and full-field stimulus testing.
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).
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.
Featuring Dr. Byron Lam, Professor, Bascom Palmer Eye Institute, University of Miami School of Medicine
Hosted by Cantor Biotechnology Analysts Emma Nealon, Alethia Young
ProQR Therapeutics is a biotechnology company listed on NASDAQ with offices in Leiden, the Netherlands and Cambridge, Massachusetts, USA. ProQR is focused on the development of life changing RNA therapies to meet the unmet need of individuals and families with common and rare diseases.
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.
