Develops RNA-based therapeutics for genetic diseases, focusing on inherited retinal disorders and cystic fibrosis.
ProQR Therapeutics N.V. is a pioneering biopharmaceutical company headquartered in Leiden, the Netherlands, dedicated to advancing RNA-based therapeutics for the treatment of genetic disorders. At the forefront of its pipeline is sepofarsen, currently in phase II/III clinical trials under the Illuminate trial, targeting Leber congenital amaurosis 10 disease, a severe genetic condition affecting vision. Additionally, the company is developing ultevursen, also in phase II/III trials, aimed at treating USH2A-mediated retinitis pigmentosa and usher syndrome, further underscoring its commitment to addressing unmet medical needs in ophthalmology.
ProQR Therapeutics N.V. is renowned for its innovative Axiomer RNA base-editing platform technology, which holds promise for precise genetic therapeutic interventions. This platform enhances the company's capabilities in developing therapies that target the underlying genetic causes of diseases. ProQR has established strategic collaborations and license agreements with leading institutions and pharmaceutical companies, including Radboud University Medical Center, Inserm Transfert SA, Ionis Pharmaceuticals, Inc., and Leiden University Medical Center. Moreover, its partnership with Eli Lilly and Company focuses on discovering, developing, and commercializing potential new medicines for genetic disorders affecting the liver and nervous system.
Founded in 2012, ProQR Therapeutics N.V. continues to expand its scientific expertise and research capabilities in RNA therapeutics, aiming to bring transformative treatments to patients worldwide. The company's dedication to innovation and collaboration underscores its commitment to advancing the field of genetic medicine and improving outcomes for patients with rare and debilitating genetic conditions.