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
… for directing site-specific A-to-I editing by endogenous ADAR enzymes Presenter: Antti Aalto, Ph.D., senior … an endogenously expressed RNA editing system called ADAR, which can direct the change of an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is …