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 …
… ® , 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 …
… that will fund operations through potential clinical data readouts in three different programs. Positive results from … 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 …
AX-0810 clinical target engagement data in healthy volunteers on track for this quarter; biliary atresia selected as initial Phase 2 indicationAdditional programs advancing toward the clinic, including AX-0811 and AX-0422Axiomer platform supporting multiple addi
ProQR and Ginkgo Bioworks to collaborate on autonomous lab high throughput data generation to support ProQR’s AI-enabled drug discovery for Axiomer™ProQR establishes AI Advisory Board with leaders from industry and academiaProQR’s first AI-discovered Development
LEIDEN, Netherlands & CAMBRIDGE, Mass., July 28, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced that it will