PRQR Profile
ProQR Therapeutics N.V., headquartered in Leiden, the Netherlands, is a leading biopharmaceutical company focused on the development of RNA-based therapeutics aimed at addressing genetic disorders. Established in 2012, the company is at the forefront of pioneering treatments for rare and challenging diseases through its innovative approach to RNA science. ProQR’s pipeline includes several advanced therapeutic candidates that leverage its expertise in RNA technology to tackle genetic conditions with high unmet medical needs.
The company’s lead product, sepofarsen, is currently in Phase II/III clinical trials under the Illuminate study, targeting Leber congenital amaurosis 10 (LCA10), a rare genetic disorder leading to blindness. Sepofarsen is designed to correct the underlying genetic defect associated with LCA10 by utilizing RNA-based therapy. In addition, ProQR is advancing ultevursen, another RNA-based therapeutic, which is undergoing Phase II/III clinical trials for the treatment of USH2A-mediated retinitis pigmentosa and Usher syndrome, both of which cause progressive vision loss and hearing impairment.
ProQR is also developing the Axiomer RNA base-editing platform, a groundbreaking technology aimed at providing more precise and effective genetic corrections. This platform is designed to enable targeted edits in RNA sequences, potentially offering new avenues for treating a variety of genetic disorders. The company’s efforts are bolstered by strategic collaborations and licensing agreements with leading research institutions and pharmaceutical companies, including Radboud University Medical Center, Inserm Transfert SA, Ionis Pharmaceuticals, Inc., and Leiden University Medical Center. Additionally, ProQR has a research collaboration with Eli Lilly and Company, focusing on the discovery and development of new treatments for genetic disorders affecting the liver and nervous system.
ProQR Therapeutics N.V. continues to build its portfolio and expand its therapeutic capabilities through these strategic partnerships and its robust pipeline of RNA-based therapies. By combining cutting-edge technology with a deep understanding of genetic disorders, the company aims to bring transformative treatments to patients worldwide, addressing some of the most challenging and rare genetic diseases.
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