ProQR Therapeutics

What ProQR Therapeutics does

ProQR Therapeutics N.V. is a clinical-stage biotechnology company listed on NASDAQ (PRQR) and headquartered in Leiden, the Netherlands, with a U.S. office in Cambridge, Massachusetts. Founded in 2012, the company employs more than 150 people from over 30 nationalities and is focused on developing RNA editing therapeutics for patients with genetic and rare diseases. ProQR serves patient populations with limited or no treatment options across multiple therapeutic areas, including cholestatic liver disease, Rett syndrome, Hurler syndrome (MPS I), and metabolic dysfunction-associated steatohepatitis (MASH).

The company's core technology is the proprietary Axiomer™ RNA editing platform, which leverages endogenous ADAR (Adenosine Deaminase Acting on RNA) enzymes to perform single-nucleotide A-to-I base edits on disease-relevant RNA transcripts without altering the underlying genome. ProQR claims the broadest intellectual property estate in ADAR-mediated RNA editing, with proprietary chemistries and delivery approaches enabling editing in liver, central nervous system, and other tissues. The clinical-stage candidate AX-0810 (cholestatic diseases) received Phase 1 CTA authorization in October 2025, while preclinical-stage candidates AX-0811 (next-gen NTCP), AX-0422 (Hurler syndrome), AX-2911 (MASH), and AX-2402 (Rett syndrome) form the broader pipeline. In April 2026, ProQR partnered with Ginkgo Bioworks to integrate the Nebula AI-enabled protein engineering platform into next-generation Axiomer candidate discovery.

As a pre-commercial biotech, ProQR's revenue model is not based on product sales but on collaboration milestones and research grants. The company has generated revenue primarily through a multi-target strategic partnership with Eli Lilly (initiated 2021) and equity-funded collaborations with patient organizations such as the Rett Syndrome Research Trust. Reported revenue is modest (€2.0M in Q1 2026 from collaboration and grant income), with a 2025 net loss of €42.2M and a cash position of €81.1M at the end of Q1 2026, supplemented by a $50M registered direct offering in June 2026. The strategic objective is to advance the Axiomer pipeline through clinical proof-of-concept and to monetize the platform through additional partnerships and, ultimately, product commercialization.

Name

ProQR Therapeutics

Legal name

ProQR Therapeutics N.V.

Website

https://www.proqr.com/

Company type

Public

Founded year

2012

Operating status

Operating

Headcount range

101–250 employees

Short description

ProQR Therapeutics is a clinical-stage biotechnology company developing RNA editing therapeutics through its proprietary Axiomer™ ADAR-mediated platform to treat rare genetic disorders, including cholestatic liver disease, Rett syndrome, Hurler syndrome, and MASH.

Ownership category

akta.pro rank

Product category

RNA Editing Therapeutics

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

RNA Therapeutics Clinical Development (trial design, endpoints, patient stratification) (HLAAADAH)

akta.pro secondary industries

RNA Chemistry & Oligonucleotide Engineering (backbones, modifications, stereochemistry) (HLAAADAE), Target Identification & Validation Platforms (omics-driven, chemoproteomics, genetic validation) (HLAAAIAI)

Keywords

• RNA editing therapeutics
• Rare disease drugs
• ADAR-mediated editing
• Genetic medicine platform
• Oligonucleotide therapies

Headquarters

HQ city

Leiden

HQ country

Netherlands

HQ region

Europe

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales

Revenue model

1. Collaboration/Milestone Revenue: ProQR generates revenue through collaboration agreements with pharmaceutical partners including Eli Lilly. The company receives milestone payments upon achieving development, regulatory, and commercial milestones. In 2025, $4.5 million in milestones were achieved through the Eli Lilly collaboration.
2. Research Grants: The company receives research grants from organizations such as the Rett Syndrome Research Trust ($1 million initial grant, $8.1 million expansion funding) to support specific RNA editing research programs.
3. Public Offerings: ProQR raises capital through underwritten public offerings of ordinary shares. Recent offerings include $50 million registered direct offering (June 2026) and $75 million public offering (October 2024).

Go-to-market motion1 record

Distribution channels2 records

Marketing channels7 records

Core offering

ProQR Therapeutics is a clinical-stage biotechnology company developing RNA editing therapies based on its proprietary Axiomer platform. The technology uses editing oligonucleotides (EONs) that recruit the body's own ADAR enzymes to make precise A-to-I single-nucleotide edits in RNA, enabling treatment of severe rare and common genetic diseases such as biliary atresia, Hurler syndrome, Rett syndrome, and MASH without permanently altering DNA.

Product overview

ProQR Therapeutics is a clinical-stage biotechnology company developing a pipeline of RNA editing therapies based on its proprietary Axiomer™ platform technology. The platform enables targeted single nucleotide edits in RNA using the body's own ADAR machinery to correct disease-causing mutations or modulate protein expression. The company's pipeline includes multiple programs at various stages: AX-0810 (clinical stage, cholestatic diseases/NTCP), AX-0811 (next-generation NTCP), AX-0422 (Hurler syndrome), AX-2911 (MASH), and AX-2402 (Rett syndrome). The company also collaborates with Eli Lilly on additional targets through the Axiomer platform.

Differentiator

Problem solved

Functional benefit

Products and services

• Axiomer RNA Editing Platform
• AX-0810 RNA editing oligonucleotide targeting the NTCP transporter to introduce a Q68R loss-of-function variant that reduces bile acid reuptake in the liver, for treatment of cholestatic liver diseases including biliary atresia and primary sclerosing cholangitis.
• AX-0811 Next-generation NTCP-targeting RNA editing program for cholestatic diseases, designed using the AI-enabled discovery engine to optimize editing oligonucleotide performance.
• AX-0422 RNA editing oligonucleotide targeting the IDUA W402X mutation to correct the most common mutation in severe Hurler syndrome (MPS I) and restore normal enzyme function.
• AX-2911 RNA editing oligonucleotide targeting PNPLA3 I148M for metabolic dysfunction-associated steatohepatitis (MASH), designed to restore PNPLA3 enzyme function.
• AX-2402

Quantifiable outcome

• AX-0810 Phase 1: Dose-dependent increases in total bile acids up to 8-fold in healthy volunteers, exceeding 2-fold threshold for NTCP modulation (8-week half-life)
• +3 more outcomes

Named customers1 record

Segments3 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Four partnerships are on record, tiered major, core and supporting.

• Ginkgo BioworksmajorTechnology or Integration · 8 April 2026Strategic partnership providing ProQR access to Ginkgo's autonomous laboratory infrastructure (Nebula) containing over 50 instruments. Partnership enables high-throughput data generation to accelerate AI-enabled drug discovery using Axiomer RNA editing technology. Ginkgo made a strategic equity investment in ProQR as part of the collaboration. Expected to increase scale and speed of experimental data generation and improve predictive performance of AI models.
• Rett Syndrome Research Trust (RSRT)coreStrategic or Co-development Partner · 5 January 2024Collaboration since January 2024 focused on design and development of editing oligonucleotides using ProQR's Axiomer technology targeting MECP2 gene for Rett syndrome. Initial $1 million research grant from RSRT. Partnership expanded in December 2024 with additional $8.1 million in funding. Focuses on correcting mutations of interest in Methyl CpG binding protein 2 (MECP2).
• Eli Lilly and CompanycoreStrategic or Co-development Partner · 1 September 2021Global licensing and research collaboration since September 2021 focused on discovery, development, and commercialization of potential new medicines for genetic disorders using ProQR's Axiomer™ RNA editing platform. Collaboration covers multiple RNA editing drug targets for liver and nervous system disorders. Expanded in December 2022 to include additional targets with option for Lilly to opt in for more. Lilly maintains pro rata ownership through participation in financing rounds. Collaboration generated $4.5 million in milestones in 2025.
• Yarrow BiotechnologysupportingOEM/ Whitelabel/ Licensing Partner · 1 January 2021In-licensed ProQR's RNA technologies to develop new medicines for unmet medical needs, similar to Eli Lilly partnership structure.

Scale indicators6 records

Recent moves6 records

Expansion highlights5 records

Emerging players

• Stoke Therapeutics: Clinical-stage company developing antisense oligonucleotides for genetic diseases including Dravet syndrome, with TANGO RNA-based splicing platform. Comparable in targeting rare genetic neurological disorders via RNA mechanisms.
• Beam Therapeutics: Clinical-stage base editing company using CRISPR-based chemistry for precision single-nucleotide edits. Related technology class (base editing) applied to genetic diseases, though operating at the DNA level.

Broad incumbents

• Arrowhead Pharmaceuticals: Established clinical-stage RNA therapeutics company with multiple RNAi and TRiM-enabled programs targeting rare genetic, hepatic, and cardiometabolic diseases. Comparable in RNA-targeted therapeutic modality and pipeline breadth.
• Alnylam Pharmaceuticals: Established leader in RNA therapeutics (siRNA) with multiple approved products and a broad pipeline in rare genetic and cardiometabolic diseases. Sets the commercial and regulatory benchmark for RNA-targeted medicines in ProQR's disease areas.
• Ionis Pharmaceuticals: Pioneer in antisense oligonucleotide (ASO) therapeutics with numerous approved and clinical-stage programs for rare genetic and neurological disorders. Comparable in therapeutic modality (synthetic oligonucleotides) and rare disease focus.

Direct peers

• Shape Therapeutics: RNA editing company developing ADAR-based therapies with platforms for CNS and metabolic diseases. Overlapping RNA editing modality and indication focus.
• Ascidian Therapeutics: RNA editing company using ASO-driven exon editing to address genetic diseases. Operates in the same RNA editing therapeutic class targeting inherited disorders.
• AIRNA Bio: Emerging ADAR-mediated RNA editing company founded by pioneers of the field. Directly comparable modality and target approach to ProQR's Axiomer platform.
• Wave Life Sciences: Clinical-stage RNA therapeutics company with PRISM platform for stereopure oligonucleotides, including RNA editing programs via ADAR. Direct competitor in oligonucleotide engineering for genetic diseases.
• Korro Bio: Direct competitor pursuing ADAR-mediated RNA editing for genetic diseases, with lead programs in alpha-1 antitrypsin deficiency. Closest functional peer given shared ADAR editing modality and rare disease focus.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights6 records

Customer concentration

• Xtwitter.com/proqr
• YouTubeyoutube.com/channel/UCC9P-aVvX767-SFcXuVEEAQ
• Facebookfacebook.com/ProQRTx
• LinkedInlinkedin.com/company/proqr-therapeutics
• Instagraminstagram.com/proqrtherapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles7 records

Funding overview

Funding rounds13 records

Investors4 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about ProQR Therapeutics