PRQR

ProQR Therapeutics N.V. is a Dutch biotechnology company focused on developing RNA-based therapeutics for the treatment of rare genetic disorders. Headquartered in Leiden, Netherlands, the company operates through its proprietary RNA repair platform technology called Axiomer, which targets specific genetic mutations at the RNA level. ProQR concentrates on developing treatments for inherited retinal diseases, including Leber congenital amaurosis 10 (LCA10) and autosomal dominant optic atrophy (ADOA), as well as other rare genetic conditions affecting the eyes and potentially other organ systems. The company's lead clinical-stage asset is sepofarsen, an antisense oligonucleotide therapy under investigation for LCA10. ProQR's Axiomer technology platform utilizes RNA editing to address disease-causing mutations without permanently altering DNA. As a clinical-stage biopharmaceutical company, ProQR does not currently generate product revenue and relies on funding through equity offerings and strategic partnerships to advance its pipeline. In recent years, the company has faced clinical setbacks, including discontinuation of certain programs following disappointing trial results. ProQR maintains collaborations with academic institutions and patient advocacy groups to support its research efforts. The company employs approximately 100 professionals and maintains research facilities in the Netherlands. ProQR's shares trade on the Nasdaq Global Market under the ticker symbol PRQR, providing access to U.S. capital markets.