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ProQR Therapeutics N.V. (PRQR)

ProQR Therapeutics N.V. operates as a biopharmaceutical enterprise dedicated to discovering and developing RNA-based therapies designed to address genetic disorders. The company's pipeline includes two primary drug candidates in advanced clinical stages: Sepofarsen is currently in a Phase II/III clinical trial, known as the ILLUMINATE trial, targeting Leber Congenital Amaurosis 10 (LCA10). Ultevursen is also undergoing Phase II/III evaluation for the treatment of USH2A-mediated retinitis pigmentosa and Usher syndrome. Beyond its clinical programs, ProQR is actively developing its proprietary Axiomer RNA base-editing platform technology. Strategic collaborations are central to ProQR's strategy, evidenced by its licensing agreements with institutions such as Radboud University Medical Center, Inserm Transfert SA, Ionis Pharmaceuticals, Inc., and Leiden University Medical Center. Furthermore, a significant license and research partnership exists with Eli Lilly and Company, focused on the discovery, advancement, and commercialization of novel medicines for genetic conditions impacting the liver and nervous system. Established in 2012, ProQR Therapeutics N.V. maintains its corporate headquarters in Leiden, the Netherlands.

Sector: Pharmaceutical Preparations

Industry: Biotechnology

ProQR Therapeutics N.V. logo

ProQR Therapeutics N.V.

NASDAQ:PRQRHealthcare
-3.47%$1.39
AH$1.43+2.88%vs close
Mkt Cap$196.16M
Float118.18M
O/S141.12M
Vol
$ Vol
RVOL
Inst Own43.0%
SI2.5%
Earnings
Dilution
ProQR Therapeutics N.V. logoNASDAQ:PRQR$1.39-3.47%
Phase Distribution
7 lead-sponsored trials·2 active·3 completed
Phase 12
Phase 1/23
Phase 2/32
Status Overview
Active, not recruiting2
Completed3
Stopped1
Unknown1

Trials need funding. See this company's dilution risk and cash position.

Clinical Trials
Status
Phase
  • Phase 2/3
    Active, not recruiting

    A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

    sepofarsen

    Leber Congenital Amaurosis 10, Blindness +8

    Enrollment
    36
    Start
    Apr 2019
    Primary Completion
    Jan 2022
    Completion
    Mar 2023est.
    NCT03913143
  • Phase 2/3
    Unknown status

    An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.

    sepofarsen

    Leber Congenital Amaurosis 10, Blindness +9

    Enrollment
    15
    Start
    Mar 2021
    Primary Completion
    Dec 2023est.
    Completion
    Dec 2023est.
    NCT04855045
  • Phase 1/2
    Completed

    Dose Escalation Study of QR-010 in Homozygous ΔF508 Cystic Fibrosis Patients

    QR-010, Placebo

    Cystic Fibrosis

    Enrollment
    70
    Start
    Jun 2015
    Primary Completion
    Sep 2017
    Completion
    Sep 2017
    NCT02532764
  • Phase 1/2
    Completed

    Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene

    QR-421a

    Retinitis Pigmentosa, Usher Syndrome Type 2 +6

    Enrollment
    20
    Start
    Mar 2019
    Primary Completion
    Oct 2021
    Completion
    Oct 2021
    NCT03780257
  • Phase 1/2
    Active, not recruiting

    A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene

    QR-1123

    Autosomal Dominant Retinitis Pigmentosa, Eye Diseases +6

    Enrollment
    11
    Start
    Oct 2019
    Primary Completion
    Jun 2022est.
    Completion
    Jun 2022est.
    NCT04123626
  • Phase 1
    Completed

    Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ΔF508 CFTR Mutation

    QR-010

    Cystic Fibrosis

    Enrollment
    18
    Start
    Sep 2015
    Primary Completion
    Sep 2016
    Completion
    Sep 2016
    NCT02564354
  • Phase 1
    Withdrawn

    Study With QR-504a to Evaluate Safety, Tolerability & Corneal Endothelium Molecular Biomarker(s) in Subjects With FECD3

    QR-504a

    Fuchs Endothelial Corneal Dystrophy, FECD3 +3

    Enrollment
    0
    Start
    Aug 2021
    Primary Completion
    Apr 2022
    Completion
    Apr 2022
    NCT05052554
Search ClinicalTrials.gov for “ProQR Therapeutics”