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 RNA 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 Director Toxicology (full-time) to support our Translational Development portfolio.
Location: Leiden, NL (on-site).
The role:
ProQR is looking for an experienced Director Toxicology to join the Translational Development team and to provide scientific and strategic leadership for the nonclinical safety assessment of our pipeline. In this role, you will define and execute toxicology strategies that enable the progression of innovative RNA therapeutics from discovery through clinical development.
As Director Toxicology, you will work across multidisciplinary project teams and collaborate closely with external partners to ensure robust, science-driven safety packages that support regulatory submissions worldwide. You will play a key role in shaping development strategies while ensuring studies are delivered to the highest scientific, regulatory, and quality standards.
The activities include:
Lead the nonclinical toxicology strategy across multiple research and development programs, ensuring alignment with program objectives and timelines as well as regulatory expectations.
Provide scientific leadership for the design, execution, oversight, and interpretation of nonclinical safety studies, including GLP toxicology studies conducted at contract research organizations (CROs).
Develop integrated nonclinical safety packages that support candidate selection, IND/CTA-enabling activities, and clinical development.
Serve as the toxicology expert on cross-functional project teams, contributing to development strategy, risk assessment, and decision-making.
Oversee external CROs, including study planning, protocol review, monitoring of study progress, budget management, and timeline adherence.
Critically review study protocols, reports, and datasets, ensuring high-quality interpretation and integration of pharmacology, toxicology, pharmacokinetic, and biomarker data.
Contribute to the preparation of regulatory documents, including Investigator's Brochures, CTA/IND submissions, briefing packages, and responses to health authority questions.
Stay current with evolving regulatory guidance and scientific advances in toxicology and RNA therapeutics, translating new insights into development strategies.
Build strong collaborations across Research, Translational Development, Clinical Development, Regulatory Affairs, CMC, and external scientific experts.
Mentor and guide junior scientists, providing scientific expertise, technical coaching, and support for their professional development within a collaborative research environment.
Required qualifications:
PhD (or equivalent) in Toxicology, Pharmacology, Veterinary Medicine, or a related life sciences discipline.
Extensive pharma and/or biotech experience in nonclinical drug development, including leadership of toxicology programs supporting clinical development.
Deep expertise in the design, conduct, interpretation, and regulatory application of GLP toxicology studies and broader nonclinical safety assessment.
Strong understanding of ICH guidelines and global regulatory expectations for nonclinical safety packages.
Experience contributing to or leading regulatory submissions (INDs, CTAs, IBs, briefing documents, responses to health authorities).
Experience working with oligonucleotide therapeutics, RNA-based medicines, gene therapies, or other advanced therapeutic modalities is highly desirable.
Demonstrated ability to influence cross-functional teams and provide strategic scientific guidance in a collaborative environment.
Excellent project management skills, with experience managing external CROs, budgets, and timelines.
Strong communication and stakeholder management skills, with the ability to translate complex scientific data into clear development recommendations.
A proactive, pragmatic, and solution-oriented mindset, with a passion for advancing innovative therapies for patients.
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
We are looking for people who are inspired by our mission to edit RNA, treat disease and change lives. Are you up for the challenge?
Please contact Silvia Catellani via [email protected] if you have any questions.
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