Senior Scientific Director, Rare Disease Translational Center

The Senior Scientific 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.

Bar Harbor:

The Jackson Laboratory's 43-acre scenic Bar Harbor campus is nestled between the Gulf of Maine and the mountains of Acadia National Park. Some of the best hiking and biking in the world (over 50 square miles of mountains, lakes, views, and dramatic coastline) is accessible from our campus. We are one mile from downtown Bar Harbor, which offers a vibrant downtown district with a plethora of restaurants, shops, museums, and galleries.

Ellsworth:

The Charles E. Hewett Center in Ellsworth, Maine, is a state-of-the-art facility that serves researchers world wide. The facility represents the culmination of nine decades of unmatched experience and leadership in breeding and caring for laboratory mice. Ellsworth, Maine, is one of Maine's fastest growing cities. With a growth rate of nearly 20%, Ellsworth is a bustling year-round community and acts as a hub for surrounding towns. It is located nearby Acadia National Park and hosts numerous special events, concerts and festivals each year. Residents have access to fabulous shopping, recreation, museums, galleries, and dining at a variety of independent restaurants.

Farmington:

JAX Genomic Medicine is transforming medicine by improving patient care, lowering costs, and increasing life span and health span. JAX Genomic Medicine's research focuses on the complex genetic causes of disease and on the development of genomic solutions tailored to each person's unique genetic makeup. JAX Genomic Medicine sits on a 17-acre site on the campus of the University of Connecticut Health Center. The 183,500-square-foot facility opened in the fall of 2014. Now, it houses over 300 biomedical researchers, technicians, and support staff in state-of-the-art computing facilities and laboratories. In Connecticut, JAX resides in the scenic town of Farmington, in the state's capitol region. The Hartford region, which offers some of the best public schools in the country, is made up of both bigger cities and smaller, charming historic New England towns. JAX Genomic Medicine is also geographically located within 2 hours of Boston and New York and is close to multiple transportation systems, including bus lines, highways, railroads and international airports.

Sacramento:

The JAX® Mice, Clinical and Research Services facility is located in Sacramento, the state capital of California. Sacramento is less than a two-hour drive from San Francisco, Napa Valley, and scenic Lake Tahoe, and a short flight to both Los Angeles and Orange County. Sacramento is the birthplace of the nation's Farm to Fork movement, and offers excellent restaurants, gastro-pubs, wineries, theaters, and museums.

Outdoor activities are plentiful:

Boating, white water rafting, and riding the scenic bicycle trails are popular. Housing and other costs of living are more affordable in Sacramento and its surrounding suburbs than in most other neighboring areas in the North Bay, making Sacramento one of the hottest housing markets in the country year after year.

Augusta:

JAX's Maine Cancer Genomics Initiative (MCGI) is located in Augusta, Maine, in the Harold Alfond Center for Cancer Care (HACCC), at Maine General Hospital. It began operations in 2016 with three overarching goals: to provide cancer genomic testing for Maine cancer patients; to develop and deliver physician education about clinical genomic cancer diagnostics; and to build a network in Maine of oncology practices conducting studies in genomic medicine.