ProQR Therapeutics is at the forefront of developing innovative RNA therapies, leveraging its proprietary Axiomer™ technology to address unmet medical needs associated with genetic disorders. This technology utilizes the body's own RNA editing machinery, specifically the ADAR enzyme, to make precise single nucleotide edits in RNA. By employing short synthetic RNA strands known as editing oligonucleotides (EONs), ProQR aims to correct disease-causing mutations or modulate protein expression, potentially leading to a new class of medicines for both rare and prevalent diseases. The Axiomer™ platform is designed to be versatile, allowing for targeted treatments that could significantly alter the course of various conditions.

ProQR's pipeline includes promising programs such as AX-0810, aimed at treating cholestatic diseases, and AX-1412, which targets cardiovascular diseases. These programs are rooted in human genetics and focus on high unmet medical needs, with the potential for significant clinical impact. The company is also exploring treatments for rare neurodevelopmental disorders like Rett Syndrome, where Axiomer™ could restore the regulatory function of the MECP2 protein. Through collaborations, such as the one with Eli Lilly and Company, ProQR is advancing its RNA editing drug targets toward clinical development, showcasing its commitment to transforming the landscape of genetic disorder treatments.

Netherlands

We are ProQR, a biotechnology company dedicated to changing lives by developing RNA therapies for rare genetic diseases. We focus our drug development mainly on a group of blinding disorders affecting the retina, called inherited retinal diseases.

Cardiovascular Diseases

Central Nervous System (CNS)

Endocrine System Diseases

Liver Diseases

Metabolic Diseases

Rare diseases

ProQR

Cholestatic Liver Disease

AX-0810

Eye disease

Genetic disorder

Leber congenital amaurosis

QR-504a is a first-in-class investigational RNA therapy designed to address the underlying cause of Fuchs endothelial corneal dystrophy (FECD) due to trinucleotide repeat (TNR) expansion mutations in the TCF4 gene. The TNR expansions cause the TCF4 RNA to aggregate in the corneal endothelial cells forming the characteristic nuclear RNA foci and eventually resulting in FECD. QR-504a is designed to target the TNRs in the TCF4 RNA. The aim is to reduce aggregation and the formation of RNA foci to prevent or stop corneal degeneration in patients with FECD. QR-504a is intended to be administered through intravitreal injections in the eye.

QR-504a 

Mediated retinitis pigmentosa

Rho mediated retinitis pigmentosa

QR-1123 aims to prevent vision loss or restore vision in people with retinitis pigmentosa due to the P23H mutation in the RHO gene. This mutation causes a mistake in the RHO RNA and the production of mutated rhodopsin protein. Over time, the mutated protein blocks the function of the normal rhodopsin protein and eventually becomes toxic to the retina. QR-1123 works by binding to the mutated RHO RNA so that it is removed by the cell through a mechanism called RNase H mediated cleavage. Once the mutated version of the RNA is removed, the normal version of the protein can perform its function in the retina again.

QR-1123 (RNase H mediated cleavage) 

Heart disease

Heart failure

Usher syndrome

QR-421a aims to prevent vision loss or restore vision in people with Usher syndrome and retinitis pigmentosa due to mutation(s) in exon 13 of the USH2A gene. Mutations in exon 13 are a common cause of these diseases. The mutations cause a mistake in the USH2A RNA and therefore the cell cannot use the RNA to produce a working USH2A protein that is essential for vision. QR-421a works by binding to the mutated USH2A RNA and exclude exon 13 from the RNA. This approach is known as exon skipping. The cells in the retina can then produce a slightly shorter but functional USH2A protein.

Ultevursen (QR-421a) (Exon 13)

Sepofarsen aims to restore vision loss in people with Leber congenital amaurosis due to a specific mutation in the CEP290 gene. This p.Cys998X mutation, also known as c.2991+1655A&gt;G, is the most common mutation causing LCA10. The p.Cys998X mutation causes a mistake in the CEP290 RNA and therefore a process called splicing is not done properly. The cell can therefore not use the RNA to produce a working CEP290 protein that is essential for vision. Sepofarsen works by binding to the mutated CEP290 RNA to enable correct splicing. The cells in the retina can then produce the CEP290 protein again.

ProQR

Company Details