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ProQR Reports Positive AX-0810 Phase 1 Data, Advances Rett and MASH Candidates, and Outlines 2026 Pipeline Plans

Story Highlights
  • On January 8, 2026, ProQR reported early AX-0810 Phase 1 safety data in healthy volunteers with no serious issues and PK matching preclinical results.
  • ProQR advanced its Axiomer platform by selecting AX-2402 and AX-2911 as development candidates, backed by strong preclinical data and Lilly milestone funding into 2027.
  • Looking for the best stocks to buy? Follow the recommendations of top-performing analysts.
ProQR Reports Positive AX-0810 Phase 1 Data, Advances Rett and MASH Candidates, and Outlines 2026 Pipeline Plans

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An update from ProQR ( (PRQR) ) is now available.

On January 8, 2026, ProQR reported encouraging initial Phase 1 data for its lead candidate AX-0810, showing no serious safety issues and pharmacokinetics in line with preclinical findings after four weeks of dosing in the first cohort of healthy volunteers, with target engagement data expected in the first half of 2026 and plans to add a patient cohort thereafter. The company also selected development candidates AX-2402 for Rett syndrome and AX-2911 for MASH based on robust preclinical proof-of-concept data, highlighted non-clinical results suggesting disease-modifying potential versus existing approaches, and noted that milestone payments from its collaboration with Eli Lilly in 2025 support a cash runway into mid-2027 as it advances its Axiomer platform and executes its 2026 development and partnering agenda.

The most recent analyst rating on (PRQR) stock is a Buy with a $5.00 price target. To see the full list of analyst forecasts on ProQR stock, see the PRQR Stock Forecast page.

Spark’s Take on PRQR Stock

According to Spark, TipRanks’ AI Analyst, PRQR is a Neutral.

ProQR’s overall stock score is primarily impacted by its financial performance and technical analysis. The company’s significant financial challenges, particularly in profitability and cash flow, are the most critical factors. Technical indicators suggest bearish momentum, and the valuation reflects ongoing losses. These factors collectively contribute to a lower overall stock score.

To see Spark’s full report on PRQR stock, click here.

More about ProQR

ProQR Therapeutics N.V., based in Leiden, Netherlands and Cambridge, Massachusetts, is a clinical-stage biotechnology company focused on developing transformative RNA therapies using its proprietary Axiomer RNA base-editing platform. The company’s pipeline includes editing oligonucleotide candidates such as AX-0810 for cholestatic liver diseases, AX-2402 for Rett syndrome and AX-2911 for metabolic-associated steatohepatitis (MASH), targeting high unmet medical needs in both rare and prevalent genetic disorders.

Average Trading Volume: 547,604

Technical Sentiment Signal: Sell

Current Market Cap: $218.8M

For detailed information about PRQR stock, go to TipRanks’ Stock Analysis page.

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