ProQR Therapeutics

PRQR

時価総額 $1.85億

ProQR Therapeutics NASDAQ: PRQR said its first-in-human data for AX-0810 provided dose-dependent evidence that its RNA-editing approach can modulate NTCP, a liver transporter involved in bile acid uptake. The company is developing NTCP programs for cholestatic liver diseases, initially focusing on pediatric biliary atresia.

ProQR Therapeutics N.V. (PRQR) Shareholder/Analyst Call Transcript

Appointments support ProQR's strategy to scale AI across the discovery and development of medicines using its Axiomer RNA editing platform and pipeline AX-0811, the first ProQR program identified through its AI-enabled discovered engine in clinical testing, with target engagement data expected in early January 2027 ProQR's expanded AI strategy is directly supporting the ongoing development programs; the Company expects up to 5 clinical data readouts over the next 12 months across 4 development programs LEIDEN, Netherlands & CAMBRIDGE, Mass., Sept. 23, 2026 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V.

LEIDEN, Netherlands & CAMBRIDGE, Mass., Sept. 09, 2026 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a clinical-stage company dedicated to changing lives through transformative RNA therapies based on its proprietary ADAR-mediated Axiomer™ RNA editing technology platform, today announced that the first participant has been dosed in the Phase 1 clinical trial of AX-0811 in healthy volunteers.

LEIDEN, Netherlands & CAMBRIDGE, Mass., Sept. 02, 2026 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V.

I initiate ProQR Therapeutics N.V. with a Buy rating, driven by strong initial human proof-of-concept for its Axiomer RNA editing platform. The company's AX-0810 achieved 8-fold serum bile acid increases in phase 1, surpassing the 2-fold target and supporting its disease-modifying potential in biliary atresia [BA]. Upcoming catalysts include full phase 1 data for AX-0810 by end-2026, pediatric BA data from China in H1 2027, and phase 1 data for next-gen AX-0811 by end-2026.

LEIDEN, Netherlands & CAMBRIDGE, Mass., Aug. 13, 2026 (GLOBE NEWSWIRE) --  ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a clinical-stage company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today reported its financial and operating results for the second quarter ended June 30, 2026, and provided a business update.

ProQR Therapeutics (PRQR) has transitioned its RNA editing platform from preclinical promise to demonstrated human pharmacological activity, notably with AX-0810's Phase 1 target engagement data. PRQR differentiates itself by prioritizing NTCP modulation in rare pediatric liver disease (biliary atresia), aiming for a faster, cleaner clinical validation path than competitors. Recent financings, including a $50M direct offering and Eli Lilly's $9.2M investment, extend PRQR's cash runway to mid-2027, supporting multiple clinical programs.

LEIDEN, Netherlands and CAMBRIDGE, Mass., June 25, 2026 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (“ProQR”), a clinical-stage biotechnology company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced the pricing of an underwritten registered direct offering of 27,624,310 ordinary shares (the “Offering”) at an offering price of $1.81 per share (the “Offering Price”), for total gross proceeds of approximately $50.0 million, before deducting underwriting discounts and commissions and other offering expenses payable by ProQR.

LEIDEN, Netherlands & CAMBRIDGE, Mass., June 25, 2026 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR or the Company), a clinical-stage biotechnology company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced positive target engagement data from its ongoing Phase 1 clinical study evaluating AX-0810, the Company's first investigational Axiomer RNA editing oligonucleotide (EON), in healthy volunteers.