The Jackson Laboratory
Senior Director, Rare Disease Translational Center
Bar Harbor, Maine · Director · Full-time
Sponsorship not specified$158k-$278kDetected 97 days ago
Project ManagementStakeholder ManagementPharmacologyCRISPRMolecular BiologyResearchExperimental DesignAnimal StudiesLeadershipCommunication
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Cap-exempt (no lottery)100
Sponsors year-round90
Entry-level history60
PERM / green-card track50
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About the role
- 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.
Responsibilities
- 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.
- Build, mentor, and scale a multidisciplinary preclinical team (geneticists, molecular and cellular biologists, iPSC specialists, study operations).
- Ensure preclinical data packages meet regulatory expectations for pre-IND and IND submissions and support regulatory interactions.
- Hands on experience designing and executing mouse model studies (transgenic, humanized, conditional) and developing/using iPSC-derived disease models.
Requirements
- 10 or more years of experience with at least 8 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.
- Proven experience implementing quality systems (SOPs, GLP or GLP readiness), ensuring data integrity, reproducibility, and operational compliance.
- demonstrated ability to present scientific and operational data to senior stakeholders and regulators.
- 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.
Nice to have
- 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.
- Talent developer who recruits, mentors, and scales high performing scientific teams
Skills
- For more information, please visit www.jax.org .
- EEO Statement:
Compensation
- Pay Range for this position is $157,688 - $277,662, based on years of related experience
Company info
- 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.
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