ProQR Therapeutics N.V., a clinical-stage biotechnology company, focuses on the discovery and development of novel therapeutic medicines. The company develops an RNA editing platform through its Axiomer RNA editing technology for genetic disorders with unmet needs. Its lead product candidate, AX-0810, targets na-taurocholate cotransporting polypeptide (NTCP) for the treatment of cholestatic diseases. The company also develops AX-2402, which targets methyl CpG binding protein 2 mutations for Rett syndrome; AX-2911, which targets PNPLA3 for metabolic dysfunction-associated steatohepatitis; and AX-1412, which targets the B4GALT1 gene for cardiovascular diseases. It has a research and collaboration agreement with Eli Lilly and Company for the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system. The company was incorporated in 2012 and is headquartered in Leiden, the Netherlands. Show more

Zernikedreef 9, Leiden, 2333 CK, Netherlands

Biotechnology
Healthcare

Market Cap

191.7M

52 Wk Range

$1.07 - $3.10

Previous Close

$1.82

Open

$1.84

Volume

458,642

Day Range

$1.70 - $1.84

Enterprise Value

87.59M

Cash

106.9M

Avg Qtr Burn

-12.24M

Insider Ownership

18.10%

Institutional Own.

50.80%

Qtr Updated

09/30/25


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Drug & IndicationStage & EventCatalyst Date
AX-0810 Details
NTCP for Cholestatic diseases

Phase 1

Data readout

AX-2911 (RNA Editing Oligonucleotide) Details
Metabolic-Associated Steatohepatitis (PNPLA3 I148M)

Phase 1

Initiation

Phase 1

Initiation

QR-1123 (RNase H mediated cleavage) Details
Mediated retinitis pigmentosa, Rho mediated retinitis pigmentosa

Failed

Discontinued

Ultevursen (QR-421a) (Exon 13) Details
Mediated retinitis pigmentosa, Usher syndrome, Heart disease, Heart failure

Failed

Discontinued

Sepofarsen (QR-110) (CEP290 gene) Details
Leber congenital amaurosis, Eye disease , Genetic disorder

Failed

Discontinued

QR-504a Details
Genetic disorder, Eye disease , Leber congenital amaurosis

Failed

Discontinued