… 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
… platform uses the body’s own editing machinery called ADAR to make single nucleotide edits in RNA,” said Gerard … as targets in our collaboration with Eli Lilly. With broad applicability, and a leading patent portfolio in the ADAR editing space, Axiomer ® …
AX-0810 program targeting NTCP for cholestatic diseases and AX-1412 program targeting B4GALT1 for cardiovascular diseases advancing to the clinic in late 2024/early 2025 with translational data and trial design details to be announced in H2 2024$4.5 million in milestones
… ® , which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to … 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 …