Director/Sr. Director, Oligonucleotide Translational Science

This position is listed on behalf of a partner company, who manages all applications and next steps. Our partner is looking for a Director/Sr. Director, Oligonucleotide Translational Science based in United States.

This is a high-impact translational science leadership opportunity at the forefront of oligonucleotide therapeutics for genetic diseases. The role sits at the critical intersection of preclinical pharmacology, DMPK, safety, and IND-enabling strategy, with direct influence on how programs advance from discovery into clinical development. You will act as a scientific integrator, translating complex in vivo and in vitro datasets into coherent development narratives that guide candidate selection, regulatory readiness, and portfolio decisions. Working closely with cross-functional teams in chemistry, toxicology, DMPK, and translational biology, you will help define what data is needed for IND success and ensure execution is rigorous and efficient. This is a highly visible role where scientific leadership, hands-on expertise, and strategic thinking directly shape program direction and patient impact.

• Lead and/or directly contribute to the design, execution, and interpretation of IND-enabling preclinical studies for oligonucleotide programs, including pharmacology, efficacy, MoA, PK/PD, and immunogenicity assessments.
• Drive integration of DMPK and pharmacokinetic data, including biodistribution, tissue uptake, exposure-response relationships, and durability of effect.
• Collaborate with DMPK and translational teams to connect PK/PD findings with safety and efficacy outcomes across systemic and tissue compartments.
• Lead evaluation of preclinical safety data with a focus on off-target effects, therapeutic index, and dose-limiting toxicity risk assessment.
• Identify scientific gaps in IND packages and recommend follow-up mechanistic, pharmacology, or safety studies to support program progression.
• Provide strategic input into oligonucleotide design, including chemistry, modifications, and conjugation approaches such as GalNAc-siRNA or antibody-oligo constructs.
• Oversee CRO and external partner studies, including protocol review, study design input, data interpretation, and final deliverable validation.
• Ensure scientific rigor and consistency across bioanalytical and pharmacology datasets generated through external vendors and internal teams.
• Contribute directly to IND-enabling submissions by integrating preclinical pharmacology, DMPK, and safety data into cohesive regulatory packages.
• Partner cross-functionally with chemistry, toxicology, translational, and clinical teams to support data-driven decision-making and program advancement.
• Communicate complex scientific findings clearly to governance forums and leadership stakeholders to inform portfolio strategy.

Requirements

• Ph.D. in pharmacology, molecular biology, biochemistry, or a related scientific discipline, or equivalent advanced degree with substantial industry experience.
• 10+ years of experience in oligonucleotide drug development, with direct involvement in IND-enabling programs (siRNA and/or ASO strongly preferred).
• Demonstrated hands-on expertise in preclinical pharmacology and DMPK/ADME studies, including PK, biodistribution, tissue uptake, and exposure-response modeling.
• Proven experience contributing to IND submissions, including preclinical pharmacology, safety, and/or DMPK components.
• Strong understanding of oligonucleotide therapeutic mechanisms, including PK/PD relationships, off-target risk assessment, and therapeutic index evaluation.
• Experience designing, managing, and interpreting in vitro and in vivo studies across pharmacology and safety domains.
• Demonstrated ability to oversee CROs and external partners, including study design, execution monitoring, and data quality assessment.
• Strong analytical skills with the ability to interpret complex biological datasets and translate them into actionable development insights.
• Excellent scientific communication skills, with experience presenting to cross-functional teams and senior governance bodies.
• Experience with conjugated oligonucleotide platforms (e.g., GalNAc-siRNA or antibody-oligo conjugates) is highly desirable.
• Exposure to bioanalytical methodologies such as LC-MS/MS, ELISA, MSD ECL, qPCR, or NGS is a plus.
• Prior experience in fast-paced biotech or small-company environments is advantageous.

Benefits

• Competitive base salary ranging from $235,000 to $300,000 annually, based on experience and qualifications.
• Performance-based bonus opportunity and equity participation.
• Comprehensive health, dental, vision, and retirement benefits package.
• Flexible paid time off policy.
• Remote-first flexibility with occasional travel requirements.
• Opportunity to shape IND-enabling strategies across cutting-edge oligonucleotide programs.
• High-impact role with direct influence on pipeline progression and clinical translation.
• Collaborative, science-driven environment with strong cross-functional partnership.
• Access to professional development resources and leadership growth opportunities.
• Opportunity to work on multiple programs across genetic disease therapeutic areas over time.