… ProQR Announces Clearance of IND to Start ClinicalTrial of QR-421a in Usher Syndrome Type 2 Patients LEIDEN, … To date, there are no approved treatments or products in clinical development that treat the vision loss associated …
QR-110 demonstrated rapid and sustained improvement in vision in the majority of subjects, as measured by visual acuity and mobility course
QR-110 was well-tolerated with no serious adverse events
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.
QR-421a showed early and encouraging evidence of activity, with 25% of patients showing a benefit across multiple concordant outcome measures and was well tolerated with no serious adverse eventsQR-421a is the second ophthalmology program where clinical activity was predicted by tra
ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need.