… that it further strengthened its leading intellectual property estate for ADAR-mediated RNA editing by successfully defending against an opposition filed in Japan by a strawman against ProQR’s … of deal to 15 targets, which would result in a $50 million opt-in payment to ProQR. ProQR may selectively form new …
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
… visual acuity, visual fields, and the objective measure of optical coherence tomography (OCT) retinal imaging, after a … – Axiomer ® and Trident ® ProQR has been further optimizing the Axiomer ® Editing Oligonucleotide (EON) … editing levels in retinal organoid models. ProQR will develop selected genetic eye disease targets with Axiomer ® , …
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