Senior Director, Rare Disease Translational Center

The Senior Director, Rare Disease Translational Center will lead and scale preclinical programs across genomic based therapeutics (gene editing, gene replacement, RNA modalities) and small molecule portfolios. This role will provide scientific and operational leadership across in vitro and in vivo translational platforms, with emphasis on rigorous in vivo data form mouse efficacy and safety experiments involving using genetically engineered mice) as well as patient derived and engineered iPSC models, and a strong focus on operational quality, reproducibility, and regulatory readiness.

Located full time in Bar Harbor, ME – Relocation assistance available

Key Responsibilities

• Set scientific strategy, milestones, and go/no go criteria for a mixed portfolio of programs form phenotyping, to target validation through pre-IND submission.

• Design, oversee, and interpret efficacy, safety, biodistribution (vectors), DMPK, PK/PD, and dose ranging studies for both modalities, ensuring appropriate study design for translation to clinic.

• Oversee the application of iPSC derived models and organoid systems for mechanism of action, target engagement, toxicity screening, and biomarker development.

• Oversee a variety of mouse model programs (transgenic, knock in/knock out, conditional, humanized), ensuring genetic model choice, study endpoints, and statistical power align with program goals.

 Redundant with above, implied in safet• Ensure operational excellence: establish and maintain SOPs, study design standards, GLP standard readiness where required, data integrity practices, reproducible workflows, and quality metrics across internal teams.

• Build, mentor, and scale a multidisciplinary preclinical team (geneticists, molecular and cellular biologists, iPSC specialists, study operations).

• Oversee program timelines, risk management, and reporting; present program status, quality metrics, and scientific rationale to executive leadership.

• Ensure preclinical data packages meet regulatory expectations for pre-IND and IND submissions and support regulatory interactions.

Requirements, Knowledge Skills and Abilities

• Ph.D. in Pharmacology, Toxicology, Genetics, Molecular Biology or related life-science discipline

• 10 or more years of experience with at least 3 years of leadership

• Industry experience in preclinical drug development with progressive leadership responsibility, including direct hands-on involvement in both small molecule and genomic therapy programs. • Strong working knowledge of mouse genetics and mouse-based efficacy experiments.

• Demonstrated track record advancing candidates through IND enabling studies toward IND/clinical transition.

• Deep technical expertise in in vivo pharmacology, DMPK, PK/PD modeling, safety pharmacology, and preclinical toxicology for small molecules and biologics/gene therapies.

• Hands on experience designing and executing mouse model studies (transgenic, humanized, conditional) and developing/using iPSC-derived disease models.

• Proven experience implementing quality systems (SOPs, GLP or GLP readiness), ensuring data integrity, reproducibility, and operational compliance.

• Strong leadership, project management, prioritization, and communication skills; demonstrated ability to present scientific and operational data to senior stakeholders and regulators. Preferred Qualifications:

• Therapeutic-area experience aligned neurodegenerative, neurodevelopmental, cardiac, and rate genetic disease.

• Prior direct experience in providing pre IND/IND packages, particularly for gene therapies.

• Publications, patents, or recognized contributions in gene therapy, iPSC modeling, translational pharmacology, or preclinical safety.

• Experience with vector engineering, manufacturing considerations for AAV, nanoparticles and other delivery modalities.

Leadership & Competencies:

• Strategic, data driven leader who translates scientific insight into actionable development plans.

• Operationally rigorous: enforces reproducibility, continuous improvement, and measurable quality metrics.

• Collaborative cross functional partner with strong stakeholder management and governance skills.

• Talent developer who recruits, mentors, and scales high performing scientific teams

Pay Range for this position is  $157,688 - $277,662, based on years of related experience

About JAX:

The Jackson Laboratory is an independent, nonprofit biomedical research institution with a National Cancer Institute-designated Cancer Center and nearly 3,000 employees in locations across the United States (Maine, Connecticut, California), Japan and China. Its mission is to discover precise genomic solutions for disease and empower the global biomedical community in the shared quest to improve human health.

Founded in 1929, JAX applies over nine decades of expertise in genetics to increase understanding of human disease, advancing treatments and cures for cancer, neurological and immune disorders, diabetes, aging and heart disease. It models and interprets genomic complexity, integrates basic research with clinical application, educates current and future scientists, and provides critical data, tools and services to the global biomedical community. For more information, please visit www.jax.org​​​​​​​.

EEO Statement:

The Jackson Laboratory provides equal employment opportunities to all employees and applicants for employment in all job classifications without regard to race, color, religion, age, mental disability, physical disability, medical condition, gender, sexual orientation, genetic information, ancestry, marital status, national origin, veteran status, and other classifications protected by applicable state and local non-discrimination laws.