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ProQR

ProQR

PRQR
Netherlands

ProQR Therapeutics is a clinical-stage biotech company developing transformative RNA therapies for genetic diseases, particularly in ophthalmology. The company leverages its innovative Axiomer® RNA editing platform to create oligonucleotide-based treatments that can correct single nucleotides in RNA, offering a potentially safer and more versatile alternative to DNA editing. While facing clinical setbacks with its earlier candidate sepofarsen, ProQR is advancing a new pipeline of RNA editing candidates and maintains a strategic collaboration with Eli Lilly. The company is publicly traded on the NASDAQ.

proqr.com

PRQR · Stock Price

USD 1.58+0.04 (+2.60%)
Market Cap: $162.3M

Historical price data

Market Cap: $162.3MPipeline: 7 drugs (2 Phase 3)Employees: 100-200HQ: Netherlands

AI Company Overview

ProQR Therapeutics is a clinical-stage biotech company developing transformative RNA therapies for genetic diseases, particularly in ophthalmology. The company leverages its innovative Axiomer® RNA editing platform to create oligonucleotide-based treatments that can correct single nucleotides in RNA, offering a potentially safer and more versatile alternative to DNA editing. While facing clinical setbacks with its earlier candidate sepofarsen, ProQR is advancing a new pipeline of RNA editing candidates and maintains a strategic collaboration with Eli Lilly. The company is publicly traded on the NASDAQ.

OphthalmologyRare Genetic DiseasesMetabolic DiseasesNeurology

Technology Platform

Axiomer® RNA editing platform: uses engineered oligonucleotides (EONs) to recruit endogenous ADAR enzymes for precise single-nucleotide (A-to-I) correction at the RNA level.

Pipeline

7
7 drugs in pipeline2 in Phase 3
DrugIndicationStageWatch
sepofarsenLeber Congenital Amaurosis 10Phase 2/3
sepofarsenLeber Congenital Amaurosis 10Phase 2/3
QR-1123Autosomal Dominant Retinitis PigmentosaPhase 1/2
QR-421aRetinitis PigmentosaPhase 1/2
QR-010 + PlaceboCystic FibrosisPhase 1/2

Funding History

4
Total raised:$230M
PIPE$100M
IPO$70M
Series B$40M
Series A$20M

Opportunities

The Axiomer® platform has the potential to treat a wide array of genetic disorders beyond ophthalmology, particularly through the Lilly collaboration in liver and CNS diseases.
Success in clinical trials could position ProQR as a leader in the emerging RNA editing field, creating significant partnership or acquisition interest.

Risk Factors

High clinical development risk, as demonstrated by the failure of lead candidate sepofarsen.
The novel Axiomer® platform is unproven in humans.
The company is pre-revenue and dependent on capital markets and partnership milestones for funding, facing potential dilution and financing challenges.

Competitive Landscape

Faces competition from gene therapy (Spark/Roche), antisense oligonucleotide (Biogen/Ionis), and DNA-editing (Editas, Intellia) companies in retinal diseases. In RNA editing, competes with Shape Therapeutics (Roche), Korro Bio, and Ascidian. Differentiation hinges on proving Axiomer's precision, safety, and clinical efficacy.

Company Timeline

2014IPO

IPO — $70.0M

2014Series B

Series B: $40.0M

2020PIPE

PIPE: $100.0M

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