Wetenschappelijk Onderzoeker Geneeskunde Specialist vacatures in Leiden

Principal Scientist Liver

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ProQR Therapeutics is a NASDAQ-listed biotechnology company headquartered in Leiden, the Netherlands with an office in and Cambridge, Massachusetts, USA.

At ProQR, we are on a mission to edit RNA, treat disease and change lives. A team of 180+ enthusiastic ProQRians from all walks of life and more than 30 different nationalities is committed to pioneering transformative RNA therapies to make a difference in the lives of the communities we serve.

Our proprietary Axiomer™ RNA editing technology platform enables the editing of specific single nucleotides in RNA using the cells own editing machinery. Axiomer editing technology harnesses ADAR (Adenosine Deaminase Acting on RNA), a protein which is present in all human cells, to repair G-to-A mutations, modulate protein, and enable protective mutation functions. Axiomer could potentially yield a new class of innovative medicines and treat currently untreatable diseases. By harnessing the power of RNA editing, we aim to create a world where genetics inform your health - but never limit it.

To strengthen our Axiomer Unit, we are looking for a senior Principal Scientist Liver to lead our liver portfolio

Location: Leiden, NL (on-site)

The Role:

As Principal Scientist Liver , you will provide scientific leadership for our liver programs, driving the discovery and development of novel RNA-based drug candidates for chronic liver diseases. You will shape scientific strategy, guide project teams in a matrix environment (without direct line management), and collaborate closely with internal and external partners to advance our liver pipeline and platform.

You will work closely with scientific program directors, the VP Axiomer Science, and the Chief Scientific Officer to identify and develop new opportunities, optimize our portfolio, and ensure scientific excellence across the liver programs.

Key Responsibilities

• Define and drive the scientific strategy for liver programs within the Axiomer platform.
• Provide expert input into target selection, disease biology, and translational strategy for chronic and genetic liver diseases.
• Lead efforts to elucidate mechanisms of action for targets and candidates using state-of-the-art tools and analytics.

• Oversee design and interpretation of in vitro and in vivo studies relevant to liver biology and pathology.
• Support the definition and assay implementation of target-related biomarkers.

• Interpret complex datasets (omics, translational data, pharmacology), including in vitro/in vivo correlation and predictive modeling and make data-driven decisions
• Collaborate with internal teams (e.g. R&D, Clinical Operations) and external partners (CROs, academic groups) to align research plans and deliverables.
• Proactively identify scientific and operational risks, propose mitigation strategies, and drive resolution.

• Identify and evaluate new scientific and technological opportunities in nucleic acid therapeutics and delivery platforms for liver.
• Serve as a scientific mentor and role model within project teams, fostering high-quality science and effective teamwork.
• Communicate complex concepts clearly to both scientific and non-scientific stakeholders, including senior leadership.

We expect our Principal Scientist Liver to have:

• PhD in a relevant scientific area, such as Liver Biology, accompanied by at least 10 years of post-graduate experience in pharmaceutical/biotech R&D or academic research dedicated to liver diseases.
• Deep understanding of liver biology and complex biological systems .
• Proven track record in liver disease drug discovery , ideally including genetic liver diseases .
• Experience with oligonucleotide and/or RNA/base editing therapeutics and their liver-targeted delivery technologies is considered a strong plus.
• Demonstrated experience in designing and leading in vivo and in vitro studies in liver disease models.
• Experience supervising, mentoring teams or project groups in a matrix.

• Solid understanding of in silico and computational approaches supporting drug discovery and development.

• Strategic thinker, comfortable working in a fast-paced, dynamic biotech environment.
• Flexible, proactive, and solutions-oriented mindset.
• Excellent communication skills in English, both written and verbal.
• Collaborative team player and pragmatic self-starter

ProQR as an employer

As an employer, we value a diverse workforce in which people are encouraged to develop themselves both personally and professionally. We believe that when people are happy, energized and supported by an environment in which they can thrive, they can achieve extraordinary things.

We are committed to ensuring that no employee, candidate, or job applicant receives less favorable treatment on the grounds of race, age, disability, pregnancy, religion, gender identity and expression, sexual orientation, marriage or civil partnership status. At ProQR we want to create an inclusive culture where everyone can be valued for who they are and in which individual differences and contributions in all forms are recognized and valued.

We offer an exciting and innovative working environment in a very committed organization, with the following benefits:

• 8% holiday allowance
• 30 vacation days
• An annual performance bonus
• Stock options
• A premium-free pension plan
• A commuting allowance
• A subsidized staff restaurant
• Unlimited (un)healthy snacks and drinks
• Employee events (boot camp, Coffee and Collaboration Café and other social events)

If you are inspired by our mission to edit RNA, treat disease, and change lives , and you bring deep expertise in liver diseases and nucleic acid therapeutics, we would like to hear from you.

Submit your application via the Apply button below.

Please contact Silvia Catellani via if you have any questions.

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