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ProQR Therapeutics (PRQR) investor relations material
ProQR Therapeutics Evercore ISI 8th Annual HealthCONx Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Evercore ISI 8th Annual HealthCONx Conference summary4 Dec, 2025
Company overview and technology
Focuses on RNA editing medicines to treat genetic and common diseases by modifying messenger RNA.
Holds foundational IP for its RNA editing technology, invented about 10 years ago.
Entered a $4 billion partnership with Eli Lilly in 2021, covering up to 15 targets, with $125 million upfront and up to $250 million in milestones per target plus royalties.
Maintains a wholly owned pipeline targeting liver and CNS indications, including rare and common diseases.
Lead program: AX-0810 for cholestatic diseases
AX-0810 targets NTCP to modulate bile acid uptake for diseases like PSC and BA.
Phase 1 trial in healthy volunteers is starting, with 33 subjects across three cohorts, randomized, placebo-controlled, and double-blinded.
Initial safety and PK data expected by year-end; full PD and biomarker data in the first half of next year.
Phase 1b in PSC patients planned for the second half of next year, with results before year-end.
Indication selection between PSC and BA for phase 2 will be announced with phase 1 results.
Rationale and trial design
Chose NTCP as the initial target due to measurable engagement in healthy volunteers and high unmet need in PSC/BA.
Trial measures bile acid concentration, conjugated/unconjugated ratio, and response to TUDCA challenge.
Success defined as at least a twofold increase in serum bile acid, shift toward conjugated bile acids, and improved clearance post-TUDCA.
Editing percentage correlates linearly with bile acid increase; 5% editing yields twofold, 10% yields fourfold increase.
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Next ProQR Therapeutics earnings date
Q4 202513 Mar, 2026
Next ProQR Therapeutics earnings date
Q4 202513 Mar, 2026
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Frequently asked questions
ProQR Therapeutics N.V. is a biotechnology company focused on developing innovative RNA therapies for genetic diseases. Established in 2012 in Leiden, Netherlands, ProQR pioneers RNA editing technologies, specifically its proprietary platform called Axiomer. This technology enables precise RNA editing by using a cell's own ADAR enzymes to alter specific nucleotides in RNA molecules, potentially reversing mutations or modulating protein expression to address unmet medical needs in various diseases. The company's approach to drug development is particularly significant for conditions that currently lack effective treatments. Over the years, ProQR has expanded its pipeline beyond its initial focus on cystic fibrosis to include a range of rare and common diseases, with ongoing research and development efforts in fields such as inherited retinal diseases and liver diseases. The company is headquartered in Leiden, Netherlands, and its shares are listed on the NASDAQ.
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