ProQR Therapeutics (NASDAQ:PRQR) is a clinical-stage biotechnology company dedicated to developing RNA-based therapies for the treatment of rare genetic disorders. Leveraging its proprietary RNA repair and editing platforms, the company designs therapeutic candidates that target the underlying molecular causes of disease by modulating RNA splicing, editing, or translation. ProQR’s early focus on ophthalmology and pulmonary applications has expanded into neuromuscular and other systemic indications, reflecting its commitment to tackling a broad range of inherited diseases with high unmet medical need.
The company’s flagship programs include sepofarsen (QR-110) for Leber congenital amaurosis type 10, which is an intravitreal RNA oligonucleotide designed to restore correct CEP290 protein expression in photoreceptor cells, and QR-421a for Usher syndrome and certain forms of retinitis pigmentosa, aimed at correcting splicing defects in the USH2A gene. In the pulmonary arena, ProQR is advancing QR-010, a therapeutic candidate for cystic fibrosis that seeks to repair the common F508del mutation in the CFTR gene. These programs underscore the versatility of ProQR’s RNA repair approach and its ability to address mutations unamenable to conventional small molecules or gene therapies.
Founded in 2012 and headquartered in Leiden, the Netherlands, ProQR has established a presence in the United States with offices in Boston, Massachusetts. The company is led by Chief Executive Officer Matthew L. Davis, who brings extensive experience in biotech leadership and RNA therapeutics. The management team combines scientific expertise with drug development acumen, supported by a board of directors and advisory panels composed of clinicians, geneticists, and regulatory specialists.
ProQR pursues strategic collaborations and partnerships to accelerate clinical development and broaden its pipeline reach. Collaboration agreements with leading pharmaceutical and biotech organizations have provided access to complementary technologies and additional resources for advancing ProQR’s candidates through clinical trials. With an international footprint and a robust R&D engine, ProQR continues to drive toward regulatory milestones and potential first approvals, aiming to transform care for patients with inherited rare diseases worldwide.
