ProQR Therapeutics
We are ProQR Therapeutics, a biotechnology company dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer®, which uses ADAR, the cell’s own editing machinery to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression. Axiomer® has the potential to yield a new class of medicines for both rare and prevalent diseases with unmet medical need. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
ProQR is based in Leiden, the Netherlands, with offices in Cambridge, MA, USA. Since 2014 we are listed on the Nasdaq stock exchange (ticker: PRQR).
ProQR's Axiomer technology explained - What is Axiomer?
ProQR's Axiomer technology explained - How does ADAR work?
"I work on designing and optimizing new therapies" - Jeroen van de Giessen Researcher at ProQR
"In rare diseases patients are the experts" - Nanyamka Braham from Regulatory Affairs at ProQR
"Towards removing all barriers in rare diseases" - Agathe Plichta from Medical Affairs at ProQR
Daniel de Boer started ProQR when one of his children was diagnosed with a rare disease
"That's the most amazing part of my job" - Scientist Marko Potman on working on rare diseases
Rare is Proud! - Andy Bolan's message for Rare Disease Day 2021
ProQR Talks episode #3 "Empowering the IRD community with knowledge of their genetic diagnosis"
Eye on the Future Forum Fall 2020 Summit
Inherited retinal diseases and the importance of genetic testing - Dr. Tim Stout
Dr. Maldonado explains why it is important to refer IRD patients to a retinal specialist
ProQR Expert Perspectives Call – Sepofarsen RNA therapy for CEP290 Leber congenital amaurosis
The impact of the My Retina Tracker genetic testing program
RNA therapy sepofarsen for Leber congenital amaurosis 10 (CEP290) - ARVO 2020 presentation and Q&A
ProQR Talks episode #2 “Why is the patient perspective in research important?”
ProQR Talks episode #1 "Accessibile information in an isolated time"
Mobility course responder in ProQR's sepofarsen clinical trial for LCA10 patients
How do ProQR's RNA therapies work?