ProQR Therapeutics

Focus: ProQR Therapeutics is a Cambridge-based biotech company focused on oligonucleotide therapies for rare diseases, particularly in ophthalmology and genetic disorders. The company operates as a pre-revenue stage biotech with a focused pipeline of genetic disease programs.

Rare Diseases Ophthalmology

Profile data last refreshed Yesterday · AI intelligence enriched 2w ago

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About ProQR Therapeutics

AI assessmentNot Hiring

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Why Work Here

Data completeness

1/9 — Limited

Company HealthSolid

Pipeline

Early-stage

7 total

LOE Risk

Protected

No near-term cliffs

Hiring

0 open

Automated analysis based on publicly available data (FDA, SEC, ClinicalTrials.gov). May be incomplete or delayed. This score does not reflect insider knowledge and should not be used as the sole basis for investment or employment decisions.

Pipeline & Clinical Trials

QR-504a

Fuchs Endothelial Corneal Dystrophy

Phase 1

Clinical Trials (1)

NCT05052554Study With QR-504a to Evaluate Safety, Tolerability & Corneal Endothelium Molecular Biomarker(s) in Subjects With FECD3

Phase 1

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Data Sources

Drug label: DailyMed / FDA
Clinical trials: ClinicalTrials.gov
Patent data: FDA Orange Book
Spending data: CMS Medicare

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.

Rare disease focus offers deep clinical impact in ophthalmology and genetic disorders where unmet medical need is highest
Oligonucleotide platform (splice-switching, exon-skipping) is a differentiated, emerging modality with significant IP moat potential
Early-stage pipeline with sepofarsen in Phase 2/3 for Leber Congenital Amaurosis 10 provides near-term clinical validation opportunities

Watch Out For

Zero active job openings signal possible hiring freeze or cash constraints limiting career mobility within the company
Pre-revenue stage with no disclosed funding or financial data creates uncertainty around runway and long-term stability

Generated by Claude from ProQR Therapeutics's SEC filings, pipeline programs, hiring velocity, FDA actions, and WARN data. Inference, not editorial — verify before quoting.

QR-010

Cystic Fibrosis

Phase 1

Clinical Trials (1)

NCT02564354Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ΔF508 CFTR Mutation

Phase 1

QR-421a

Retinitis Pigmentosa

Phase 1/2

Clinical Trials (1)

NCT03780257Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene

Phase 1/2

QR-010

Cystic Fibrosis

Phase 1/2

Clinical Trials (1)

NCT02532764Dose Escalation Study of QR-010 in Homozygous ΔF508 Cystic Fibrosis Patients

Phase 1/2

QR-1123

Autosomal Dominant Retinitis Pigmentosa

Phase 1/2

Clinical Trials (1)

NCT04123626A 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

Phase 1/2

sepofarsen

Leber Congenital Amaurosis 10

Phase 2/3

Clinical Trials (1)

NCT04855045An 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.

Phase 2/3

sepofarsen

Leber Congenital Amaurosis 10

Phase 2/3

Clinical Trials (1)

NCT03913143A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

Phase 2/3