Partnership focused on utilizing Axiomer™ to develop editing oligonucleotides targeting an underlying genetic variant that causes Rett syndromeCollaboration expands ProQR’s commitment to advance the development of therapies for central nervous system diseases
… 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 …
Agreement provides ProQR with initial payment of €12.5M and up to €135M in further payments, as well as potential additional earn outs based on commercial sales in the US and EU
Divestment of sepofarsen and ultevursen supports ProQR’s strategic focus on the Axiomer®&
… an endogenously expressed RNA editing system called ADAR, which it can direct to change an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is …
ProQR is accelerating the development of its Axiomer® RNA base-editing technology platform across multiple therapeutic areas and will provide an update on first targets in H2 2022
Company plans to discuss findings from sepofarsen Illuminate trial w
… 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 …