Scientific Director | tRNA Biotherapeutics

Scientific Director | tRNA Biotherapeutics – Germany

This is a rare opportunity to take a leading scientific role at the forefront of next-generation RNA-based therapeutics, shaping a pioneering platform aimed at treating rare genetic and neuromuscular diseases with no current therapeutic options. You will operate at the intersection of discovery science, translational research, and strategic company development, driving programs from early proof‑of‑concept through IND‑enabling studies. In this role, you will define the scientific vision and directly influence the direction of a cutting‑edge tRNA therapeutic platform with both elevation and suppression modalities. You will collaborate closely with executive leadership, academic partners, and external experts to translate breakthrough RNA biology into clinically meaningful therapies. The environment is highly entrepreneurial, science‑driven, and globally collaborative, with frequent engagement across Europe’s leading research institutions. This position offers the opportunity to make a tangible impact on the future of genetic medicine while helping build a platform with broad therapeutic reach.

Accountabilities

• Define and lead the scientific strategy and R&D roadmap, guiding the tRNA therapeutic platform from discovery through preclinical development and IND‑enabling studies.
• Oversee advancement of dual tRNA modalities (elevation and suppression) across multiple therapeutic programs targeting rare and neuromuscular diseases.
• Lead translational research efforts, ensuring alignment between RNA biology discoveries and clinically relevant therapeutic applications.
• Supervise and guide preclinical studies, including in vivo and in vitro work, biomarker development, and functional validation in relevant disease models.
• Drive optimization of AAV‑based constructs, vector design, capsid selection, and delivery approaches, as well as development of synthetic delivery systems.
• Oversee IND‑enabling activities including toxicology, biodistribution, dose‑finding, and regulatory study design.
• Identify and evaluate new clinical indications to expand the platform’s therapeutic reach across additional disease areas.
• Collaborate cross‑functionally with CMC, regulatory, clinical, IP, and business development teams to align scientific execution with company milestones.
• Build and maintain relationships with academic partners, key opinion leaders, and scientific advisory boards to support research and validation efforts.
• Represent the organization in scientific conferences, investor discussions, publications, and external stakeholder engagements.
• Contribute to building a high‑performance scientific culture and, over time, support recruitment and mentorship of an expanding R&D team.

Requirements

• PhD or equivalent advanced degree in Molecular Biology, Genetics, Neuroscience, or a related life sciences field.
• 8–12+ years of experience in biotech or pharmaceutical R&D, with progressive scientific leadership responsibilities.
• Strong expertise in RNA biology, translational mechanisms, gene therapy, or related therapeutic modalities.
• Hands‑on experience with AAV vector design, gene delivery systems, and preclinical development workflows.
• Familiarity with LNP or alternative delivery technologies for nucleic acid therapeutics.
• Proven track record working with in vivo and in vitro disease models, including rodent models and ideally non‑human primates.
• Experience advancing programs toward IND‑enabling stages, including toxicology and regulatory study design.
• Strong ability to translate early‑stage scientific discoveries into therapeutic development strategies.
• Demonstrated success in leading cross‑functional collaboration in biotech or high‑growth environments.
• Strong communication skills with experience engaging scientific, clinical, and business stakeholders.
• Entrepreneurial mindset with a hands‑on, execution‑oriented approach to scientific leadership.
• Experience in rare diseases or platform‑based therapeutic development is highly desirable.

Benefits

• Opportunity to lead the development of a pioneering tRNA‑based therapeutic platform with broad disease applicability.
• High‑impact scientific leadership role shaping programs from discovery through early clinical readiness.
• Flexible, remote‑first working model with international collaboration across leading research institutions.
• Close partnership with experienced biotech leaders, academic experts, and external scientific networks.
• Significant strategic influence over platform development, research direction, and pipeline expansion.
• Opportunity to contribute to publications, patents, and high‑visibility scientific outputs.
• Entrepreneurial environment with direct involvement in company growth, partnerships, and fundraising activities.
• Strong mission focus on developing transformative therapies for patients with high unmet medical needs.