… 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 … may selectively enter additional partnerships designed to advance and capture the full potential value of the platform. …
… 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 …
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
… 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 …
… as of end of Q2 providing runway into mid-2026, plus additional potential milestones from Lilly partnership … mid-2026, we look forward to continued execution as we advance our science and pipeline programs.” Recent Progress … P-705 ). ProQR scientists showed for the first time in the ADAR RNA editing field in vivo proof of target engagement …
… 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 …