… partnership is another validation of our leadership in ADAR-mediated RNA editing, our robust IP estate, and the … molecular machinery that is present in human cells called ADAR (Adenosine Deaminase Acting on RNA). Axiomer EONs are designed to recruit and direct endogenously expressed ADARs to change an Adenosine (A) to an Inosine (I) in the RNA …
Continued advancement of platform and initial pipeline programs with liver delivery to address Cholestatic Diseases targeting NTCP and Cardiovascular Disease targeting B4GALT1Further strengthened leading intellectual property (IP) position with issuance of new patent in the United S
… initial pipeline targets expected in late 2022/early 2023 Additional pivotal trial recommended by EMA for sepofarsen … (MAA). In light of this feedback and in order to continue advancement of the portfolio of ophthalmic product … platform. ProQR has established a leading IP estate in the ADAR-editing space related to its RNA-editing technology and …
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
… ProQR Announces Additional Sepofarsen Illuminate Trial Analyses and Provides … the Company’s runway and deliver on its commitment to advance RNA therapies for diseases with high unmet need. … in 2014, it has established a leading IP estate in the ADAR editing space, a first industry partnership, and with …
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