… of ProQR’s pipeline and contributing to improved patient outcomes. As a subject matter expert in CNS diseases, … key opinion leaders, investigators, academic centers, and patientadvocacy partners. Ability to build and maintain …
Partnership focused on utilizing Axiomer™ to develop editing oligonucleotides targeting an underlying genetic variant that causes Rett syndromeCollaboration expands ProQR’s commitment to advance the development of therapies for central nervous system diseases
Top-line results expected H1 2022
Sepofarsen is a potential first-in-class RNA therapy for the treatment of LCA10, a rare inherited retinal disorder that leads to blindness
LEIDEN, Netherlands & CAMBRIDGE, Mass., Jan.