Senior Director, Rare Disease Gene Therapy

The Jackson Laboratory•Bar Harbor, ME

19h•Onsite

About The Position

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. The position is located full time in Bar Harbor, ME, with relocation assistance available.

Requirements

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.

Nice To Haves

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.

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.
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.