ProQR Therapeutics

ProQR presented an accelerated RNA‑editing pipeline at the Cantor conference, with AX‑0811, an improved version of AX‑0810, now entering clinical development. An AI‑powered screening approach, the company says, greatly improves editing efficiency (projected 40–65% for AX‑0811 vs. 10–15% for AX‑0810) and is intended to speed molecule optimization. The lead program targets NTCP for cholestatic diseases, notably biliary atresia, with a planned phase II study in mid‑2025 and a focus on biomarker‑driven, accelerated regulatory pathways. ProQR expects five clinical data readouts from four programs over the next 12 months, but stresses that patient efficacy and regulatory acceptance still need to be demonstrated. » Plus sur de.investing.com

ProQR shares slipped about 3% even though Phase 1 data from the first cohort for AX-0810 showed no serious adverse events and the pharmacokinetics matched the nonclinical data. Target-engagement data for AX-0810 are expected in the first half of 2026. ProQR also selected AX-2402 (Rett syndrome, MECP2-R270X) and AX-2911 (MASH, PNPLA3) as development candidates after convincing preclinical results, and received $4.5 million in milestone payments from Eli Lilly in 2025, extending its cash runway to mid-2027. » Plus sur de.investing.com

The CCMO has granted ProQR permission to start a phase-1 trial of the RNA-editing agent AX-0810 in healthy volunteers. The approval was issued under the new centralized EMA review procedure and permits dosing in the Netherlands. The study will assess safety, tolerability, and pharmacokinetics and -dynamics using biomarkers; AX-0810 is the first Axiomer program to enter clinical development. ProQR will hold a virtual investor event on Nov. 3; an analyst most recently confirmed a "Market Outperform" rating with a $8.00 price target. » Plus sur de.investing.com