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 ...
ProQR Therapeutics N.V. (Nasdaq: PRQR) is a biotech company dedicated to creating potentially life-changing RNA-based therapies for patients with severe rare and common diseases, with a particular emphasis on genetic disorders. Headquartered in Leiden, the Netherlands, ProQR’s strategy centers on developing therapeutics that address disease-causing genetic variants through RNA-directed approaches, ...ProQR Therapeutics N.V. (Nasdaq: PRQR) is a biotech company dedicated to creating potentially life-changing RNA-based therapies for patients with severe rare and common diseases, with a particular emphasis on genetic disorders. Headquartered in Leiden, the Netherlands, ProQR’s strategy centers on developing therapeutics that address disease-causing genetic variants through RNA-directed approaches, rather than conventional protein replacement or small-molecule pathways.
At the product and pipeline level, ProQR’s portfolio includes clinical-stage RNA therapy candidates. Sepofarsen is being evaluated in a Phase II/III program (the ILLUMINATE trial) targeting Leber Congenital Amaurosis 10 (LCA10). Ultevursen is also in Phase II/III evaluation for conditions associated with USH2A-mediated retinitis pigmentosa and Usher syndrome (US H syndrome). These programs reflect ProQR’s focus on advancing therapies where RNA-targeting may enable modulation of underlying genetic mechanisms.
Underpinning the pipeline is ProQR’s proprietary Axiomer RNA base-editing platform technology. From a business perspective, this platform supports both internal program development and external partnerships by providing a differentiated technical foundation for discovery and translational research. ProQR’s approach includes licensing and collaboration arrangements with academic and industry partners—such as agreements involving Radboud University Medical Center, Inserm Transfert SA, Ionis Pharmaceuticals, and Leiden University Medical Center—plus a notable license and research partnership with Eli Lilly focused on discovering, advancing, and commercializing medicines for genetic conditions affecting the liver and nervous system.
Cost and operational structure in this sector typically relies heavily on research and development rather than manufacturing-heavy cost of goods. ProQR’s financial metrics (as presented in the dataset) suggest a R&D-intensive model with limited or negative cash-flow generation at this stage, which is common for clinical-stage biotechnology companies where expenses for trials, regulatory work, CMC-related development, and platform operations precede product revenues. In this context, “BOM” is not a traditional bill-of-materials manufacturing model; instead, the cost base includes trial operations, specialized laboratory work, analytical development, regulatory submissions, and platform engineering—often complemented by collaboration arrangements that can share certain scientific or development responsibilities.
In terms of key people, Daniel Anton de Boer serves as the founder and CEO, having led the company since its incorporation in 2012. With a workforce of about 187 employees, ProQR has a relatively compact organization for late pre-commercial development, consistent with a specialty biotech model that leverages external partners and scientific networks.
Looking ahead, ProQR’s success depends on continued clinical progress, regulatory outcomes, and the ability to translate RNA-editing platform capabilities into robust therapeutic candidates. The company’s collaboration-driven strategy is designed to broaden opportunities across therapeutic areas and disease targets while maintaining focus on advancing its key internal programs.
YoYYoY means Year-over-Year. It compares the latest annual value with the previous annual value to show long-term trend strength.
QoQQoQ means Quarter-over-Quarter. It compares the latest quarter with the immediately previous quarter to show short-term momentum changes.
RevenueThe total money that came through the front door from selling things, before paying a single bill. Think of it as the grand total of every credit card swipe from customers. (YoY compares this year to last year's performance, while QoQ compares the current three months to the previous three).
$15.3M
-19.2%
+330.9%
Net IncomeThe absolute bottom line. If the company paid every single supplier, employee, banker, and tax collector, this is the actual money left in their pocket at the end of the day.
$-40.5M
-45.9%
+34.8%
Gross MarginThe basic markup. If they sell a $100 pair of sneakers, this percentage tells you how much of that price tag is profit right after paying for the rubber and shoelaces, but before paying for things like store rent or TV commercials.
+83.0%
-17.0%
+39.6%
Operating MarginThe 'day job' efficiency score. Out of every dollar a customer spends, this shows how many cents the company keeps after making the product AND paying for all the everyday corporate overhead (like salaries, marketing, and keeping the lights on).
-275.9%
-71.2%
+85.0%
Net MarginThe final take-home percentage. When you strip away every conceivable cost, tax, and interest payment, this is the exact number of cents the company truly gets to keep from every dollar in sales.
-265.2%
-80.6%
+84.9%
Free Cash FlowThe holy grail of corporate cash. It's the spendable, physical money left over after the business pays for its daily operations AND buys the big, expensive upgrades (like new factories or servers) it needs to survive. This is the 'free' money they can use to pay dividends or buy back stock.
$-53.8M
-42.3%
-12.3%
FCF MarginThe ultimate cash conversion rate. It shows how good the company is at turning regular sales directly into cold, hard, spendable cash. A high percentage means the business is an absolute cash-printing machine.
-352.2%
-76.1%
+73.9%
Debt / EquityThe financial risk gauge. It compares how much of the company's empire was built using borrowed money (loans) versus the owners' own money (shareholders). A high number means they are heavily leveraged and playing a riskier game; a low number means they are playing it safe.
28.3%
+61.6%
-54.4%
Current RatioThe 12-month survival check. It simply compares the cash they have right now (plus things they can quickly turn into cash) against the immediate bills they absolutely must pay this year. A score above 1 means they have enough in the wallet to cover the upcoming bills without panicking.
3.09x
-22.0%
+40.6%
Total AssetsThe absolute size of the company's empire. It bundles together absolutely everything of value they own—from the cash in the register and the inventory in the warehouse, to the software patents in the vault and the factories on the ground.
