ProQR

ProQR Therapeutics

ProQR Therapeutics logo

ProQR Therapeutics is a biotechnology company focused on the development of RNA therapies for the treatment of genetic disorders. The company is headquartered in Leiden, Netherlands, and was founded in 2012. ProQR's mission is to create transformative RNA therapies for patients with severe genetic diseases.

History

ProQR Therapeutics was founded by Daniel de Boer and a team of experts in the field of RNA biology and genetic medicine. The company was established with the goal of developing innovative treatments for rare genetic disorders, particularly those that currently have no effective therapies.

Technology

ProQR's core technology is based on RNA therapeutics, which involves the use of RNA molecules to modulate gene expression and correct genetic mutations. This approach allows for the targeting of specific genetic defects at the RNA level, potentially providing a more precise and effective treatment compared to traditional small molecule drugs or protein therapies.

RNA Editing

One of the key areas of focus for ProQR is RNA editing, a technique that allows for the correction of specific mutations in RNA transcripts. This technology has the potential to treat a wide range of genetic disorders by directly repairing the faulty RNA that results from genetic mutations.

Pipeline

ProQR has a robust pipeline of RNA-based therapies targeting various genetic disorders. Some of the key programs in their pipeline include:

• QR-110: A therapy for Leber congenital amaurosis 10 (LCA10), a rare genetic eye disease that leads to blindness.
• QR-421a: A treatment for Usher syndrome type 2A, a condition that causes combined hearing and vision loss.
• QR-313: A therapy for Dystrophic Epidermolysis Bullosa, a severe skin disorder.

Research and Development

ProQR is committed to advancing the field of RNA therapeutics through rigorous research and development. The company collaborates with leading academic institutions and research organizations to explore new applications of RNA technology and to bring innovative treatments to patients in need.

Collaborations and Partnerships

ProQR has established partnerships with various organizations to enhance its research capabilities and accelerate the development of its therapies. These collaborations include partnerships with academic institutions, research consortia, and other biotechnology companies.

Future Directions

ProQR continues to explore new opportunities in the field of RNA therapeutics, with a focus on expanding its pipeline to include treatments for additional genetic disorders. The company aims to leverage its expertise in RNA biology to develop therapies that can address unmet medical needs and improve the lives of patients worldwide.

Related Pages

• RNA Therapeutics
• Genetic Disorders
• Biotechnology
• Leber Congenital Amaurosis
• Usher Syndrome