ProQR

ProQR is a biopharmaceutical company that focuses on the development of transformative RNA therapies for the treatment of severe genetic rare diseases such as Leber's congenital amaurosis, Usher syndrome, and cystic fibrosis. The company's unique approach to RNA therapy involves the use of small molecules that can bind to the RNA in cells and modulate protein production through a process known as RNA splicing.

History

Founded in 2012, ProQR is headquartered in Leiden, the Netherlands. The company was established with the aim of developing RNA therapies for patients with severe genetic disorders. ProQR's initial focus was on ophthalmology, but it has since expanded its research and development efforts to include other rare diseases.

Research and Development

ProQR's research and development strategy is centered on the use of its proprietary Axiomer® technology platform. This platform enables the design and development of RNA therapies that can specifically target and correct the RNA mutations that cause certain genetic diseases.

One of the company's most advanced programs is sepofarsen, a potential treatment for Leber's congenital amaurosis 10 (LCA10), the most common form of LCA. Sepofarsen is currently in Phase 2/3 clinical trials.

Clinical Trials

ProQR conducts numerous clinical trials to evaluate the safety and efficacy of its RNA therapies. These trials are critical steps in the drug development process and are necessary for obtaining regulatory approval from agencies such as the Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

Partnerships and Collaborations

ProQR actively seeks partnerships and collaborations with other biopharmaceutical companies, research institutions, and patient advocacy groups. These partnerships are crucial for advancing the company's research and development efforts and for bringing new therapies to patients.

See Also

• RNA therapy
• Leber's congenital amaurosis
• Usher syndrome
• Cystic fibrosis