ProQR Therapeutics - Creating RNA therapies for patients in need

On a mission

At ProQR, we are passionate about pushing the boundaries of science and technology to create innovative solutions for unmet medical needs. Our team is dedicated to conducting cutting-edge research and development in the RNA editing field to improve the lives of patients and families affected by genetic disorders.

We believe that every person deserves access to the best possible care, and we are committed to working together as a community to make that happen. Join us on our mission to make a real difference in the lives of those we serve.

Our science

Axiomer technology - RNA base editing

Scientists at ProQR have invented a new way to use RNA oligonucleotides to alter RNA. The Axiomer® technology has the potential to become a new class of medicine and treat currently untreatable diseases.
How Axiomer and ADAR work

What is RNA and how can we use ADAR, the body’s own RNA editing machinery to treat disease? Check out our latest video explaining how it works.
Internal link Working at ProQR

Latest press releases

Internal link ProQR Announces Appointment of Chris Hart as Chief Data and AI Officer and Thomas Wolf to join the Board of Directors as Strategic AI Advisor
Date: 23 September 2026
Internal link ProQR Announces First Participant Dosed in Phase 1 Study of AX-0811 and Virtual Investor and Analyst Educational Event on September 30, 2026
Date: 09 September 2026
Internal link ProQR to Participate in Upcoming Investor Conferences in September 2026
Date: 02 September 2026

Internal link More press releases

ProQR Community News

Why AI, Why Now

Community news

AI now sits at the center of how ProQR pursues its mission: discovering, developing, and delivering transformative RNA-editing medicines to patients with high unmet medical needs, faster and with a higher chance of success. Getting that right takes more than a new algorithm layered onto old workflows - it means rethinking how discovery and development teams work together across the company, it means rethinking how and what data we generate to answer out scientific and development questions, end to end.
Embracing Diversity: Why it Matters

Community news

As a mission-driven company dedicated to changing lives by developing RNA therapies for severe rare and common diseases. ProQR recognizes the importance of advancing diversity, equity, and inclusivity (DEI) in the workplace to inspire each employee to fully thrive and to make their maximum contribution to ProQR.
Exploring why companies partner their technology

Community news

Partnerships in our industry are very common as companies look to benefit from knowledge and resource sharing. In this blog we explore why companies, like ProQR, partner with other companies.

ProQR & Eli Lilly and Company partnership

Since 2021 ProQR has partnered with Eli Lilly and Company focusing on the discovery, development, and commercialization of potential new using ProQR’s proprietary Axiomer® RNA editing platform.

Internal link Read about our partnership

Meet us at

Internal link Investor and Analyst educational event: “NTCP Modulation in Biliary Atresia via Liver-Targeted RNA Editing”

Webcast

ProQR will review the recently reported AX-0810 Phase 1 target engagement data and discuss the translational rationale and clinical development strategy for NTCP modulation in biliary atresia, including the framework for evaluating upcoming next-generation AX-0811 Phase 1 data and its potential to treat cholestatic disease.
- Add Investor and Analyst educational event: “NTCP Modulation in Biliary Atresia via Liver-Targeted RNA Editing” to calender
Investor
Sep 30 2026

11am ET

Internal link More events