Platform demonstrates robust in vivo editing capabilities reporting up to 70% editing of ACTB in the liver of non-human primates (NHPs) and miceFunctional effect demonstrated in mice in vivo via modulation of ANGPLT3 protein properties leading to favora
Post-hoc analyses from Illuminate trial of sepofarsen demonstrate an encouraging efficacy signal when comparing active treatment and sham eyes to their corresponding contralateral eyes across multiple endpoints – Company plans to discuss findings with regulators in Q3
Divestment of sepofarsen and ultevursen ophthalmic programs supports ProQR’s strategic focus on Axiomer® RNA editing platform technology and continued advancement of pipeline
€129 M cash and cash equivalents as of June 30, 2023 providing runway into mid-2026