ProQR Therapeutics N.V. (PRQR) ProQR Therapeutics N.V. operates as a biopharmaceutical enterprise dedicated to discovering and developing RNA-based therapies designed to address genetic disorders. The company's pipeline includes two primary drug candidates in advanced clinical stages: Sepofarsen is currently in a Phase II/III clinical trial, known as the ILLUMINATE trial, targeting Leber Congenital Amaurosis 10 (LCA10). Ultevursen is also undergoing Phase II/III evaluation for the treatment of USH2A-mediated retinitis pigmentosa and Usher syndrome. Beyond its clinical programs, ProQR is actively developing its proprietary Axiomer RNA base-editing platform technology. Strategic collaborations are central to ProQR's strategy, evidenced by its licensing agreements with institutions such as Radboud University Medical Center, Inserm Transfert SA, Ionis Pharmaceuticals, Inc., and Leiden University Medical Center. Furthermore, a significant license and research partnership exists with Eli Lilly and Company, focused on the discovery, advancement, and commercialization of novel medicines for genetic conditions impacting the liver and nervous system. Established in 2012, ProQR Therapeutics N.V. maintains its corporate headquarters in Leiden, the Netherlands.
Sector: Pharmaceutical Preparations
Industry: Biotechnology
ProQR Therapeutics N.V. NASDAQ:PRQR Healthcare · Biotechnology
Mkt Cap $196.16M
Vol 0
$ Vol 0
RVOL 0
Earnings 0
Press Release
ProQR to Present Clinical and Preclinical Progress from AX-0810 and AX-0811 at Upcoming Conferences
ProQR announced upcoming conference presentations covering its AX-0810 Phase 1 data and next-generation AX-0811 program at TIDES Europe, AASLD The Liver Meeting 2026, and Nature's RNA at the Bench and Bedside V.
The AASLD presentations include a late-breaking poster with proof-of-concept evidence of in vivo Axiomer RNA editing of NTCP from the AX-0810 Phase 1 trial, which is being developed for cholestatic disease.
AX-0811, a next-generation NTCP-targeting RNA editing oligonucleotide generated with ProQR's AI-enabled discovery engine, is in a Phase 1 study in healthy volunteers with initial data expected in early January 2027.
• 4d ago AI analysis › Press Release
ProQR Announces Appointment of Chris Hart as Chief Data and AI Officer and Thomas Wolf to join the Board of Directors as Strategic AI Advisor
ProQR appointed Chris Hart, PhD, formerly VP of Data Science and AI/ML at Eli Lilly, as Chief Data and AI Officer, and Thomas Wolf, PhD, co-founder of Hugging Face, to its Board as strategic AI advisor.
The appointments support scaling AI across the Axiomer RNA editing platform, with AX-0811 — the first AI-discovered program in the clinic — on track for target engagement data in early January 2027.
The company expects up to 5 clinical data readouts over the next 12 months across 4 development programs.
• 1mo ago AI analysis › Press Release ⚠ 2
ProQR Announces First Participant Dosed in Phase 1 Study of AX-0811 and Virtual Investor and Analyst Educational Event on September 30, 2026
ProQR dosed the first participant in a Phase 1 trial of AX-0811, its AI-discovered RNA editing oligonucleotide designed to modulate NTCP as a potential disease-modifying treatment for cholestatic liver diseases including biliary atresia.
Preclinical work showed AX-0811 with greater potency and a longer half-life than predecessor AX-0810, and the Phase 1 in healthy volunteers will assess safety, tolerability, PK and PD biomarkers, with target engagement data from the first two cohorts expected in early January 2027.
The company also scheduled a virtual investor and analyst educational event on NTCP modulation in biliary atresia for September 30, 2026 at 11am ET, featuring hepatology KOL Prof. Gideon Hirschfield alongside management and a review of recently reported AX-0810 Phase 1 data.
• 1mo ago AI analysis › Press Release
ProQR to Participate in Upcoming Investor Conferences in September 2026
ProQR Therapeutics announced that management will participate in two September investor conferences: the Wells Fargo 21st Annual Healthcare Conference in Boston on September 8 and the 2026 Cantor Global Healthcare Conference in New York on September 9.
At the Cantor event, ProQR will host a fireside chat at 11:30am ET plus 1x1 meetings; webcast details will be posted on the company's website, with archived webcasts available for roughly 30 days.
The release contains no new clinical, financial, or pipeline data on the company's Axiomer RNA editing platform — it is a routine scheduling notice.
• 1mo ago AI analysis › Press Release ● Major
ProQR Announces Second Quarter 2026 Operating and Financial Results
ProQR reported Q2 2026 revenue of €8.8 million, more than doubling the prior year, while narrowing its net loss to €8.7 million, or €0.08 per share.
The company achieved the first clinical validation of its Axiomer RNA editing platform with AX-0810 Phase 1 data showing an 8-fold increase in target engagement and a favorable safety profile.
ProQR strengthened its balance sheet with $59.2 million in financing, ending the quarter with €117.1 million in cash to fund operations into mid-2028 and support multiple upcoming clinical milestones.
• 2mo ago AI analysis › Press Release
ProQR Prices $50.0 Million Underwritten Registered Direct Offering and Concurrent Private Placement
ProQR Therapeutics priced a $50 million registered direct offering of approximately 27.6 million ordinary shares at a price of $1.81 per share.
Strategic partner Eli Lilly participated concurrently, purchasing roughly 5.1 million shares in a private placement to maintain its ownership stake, contributing an additional $9.2 million in gross proceeds.
The company intends to use the combined $59.2 million in net proceeds primarily to fund research and clinical development of its pipeline candidates, as well as for working capital and general corporate purposes.
• 4mo ago AI analysis › Press Release ● Major
ProQR Announces Positive Phase 1 Target Engagement Data for AX-0810, Establishing First Clinical Validation of the Axiomer RNA Editing Platform
ProQR reported positive Phase 1 data for AX-0810, showing dose-dependent target engagement with up to an 8-fold increase in serum bile acids at the 6 mg/kg dose, significantly exceeding the 2-fold threshold.
The drug demonstrated a favorable safety profile with no serious adverse events and a half-life of eight weeks, supporting the expected pharmacologic profile for sustained engagement.
These results validate the Axiomer RNA editing platform and support the continued development of the NTCP franchise, including the next-generation candidate AX-0811 for biliary atresia.
• 4mo ago AI analysis › Press Release ● Major
ProQR Announces First Quarter 2026 Operating and Financial Results
globenewswire.com • 5mo ago Press Release
ProQR Announces Multiple Upcoming Scientific Conference Presentations Highlighting the Breadth of its Axiomer™ RNA Editing Platform
globenewswire.com • 5mo ago Press Release
ProQR Announces Annual General Meeting of Shareholders to be Held June 2, 2026
ProQR Therapeutics announced its Annual General Meeting of Shareholders will be held on June 2, 2026, at 1500 CEST in Amsterdam.
Relevant documents, including the notice and agenda, will be available on the company's website and the SEC's website.
The company reiterated its focus on its Axiomer RNA editing technology platform.
• 5mo ago AI analysis › Press Release ● Major
ProQR Announces Nomination of Dr. Lykke Hinsch Gylvin to its Board of Directors
globenewswire.com • 6mo ago Press Release
ProQR Highlights Pipeline Expansion and Multiple Upcoming Clinical Catalysts at Investor and Analyst Event
globenewswire.com • 6mo ago Press Release
ProQR Announces Partnership with Ginkgo Bioworks and Formation of AI Advisory Board
globenewswire.com • 6mo ago 
