Senior Director, Rare Disease Translational Center (Gene Therapy)

Location: Onsite full time in Bar Harbor, Maine, United States; relocation assistance is available.

Salary: $157,688–$277,662 annually, based on years of related experience.

Company

hirify.global is an independent, nonprofit biomedical research institution focused on genetics, genomic solutions, disease research, and translational biomedical science.

What you will do

• Lead scientific strategy, milestones, and go/no-go decisions across preclinical programs from phenotyping and target validation through pre-IND submission.
• Design, oversee, and interpret efficacy, safety, biodistribution, DMPK, PK/PD, and dose-ranging studies for small molecules and genomic therapies.
• Direct the use of genetically engineered mouse models, patient-derived and engineered iPSC models, and organoid systems for efficacy, mechanism of action, toxicity, and biomarker development.
• Establish SOPs, study-design standards, GLP readiness, data-integrity practices, reproducible workflows, and quality metrics.
• Build, mentor, and scale a multidisciplinary preclinical team.
• Manage timelines, risks, reporting, executive presentations, regulatory interactions, and pre-IND/IND data packages.

Requirements

• Ph.D. in Pharmacology, Toxicology, Genetics, Molecular Biology, or a related life-science discipline.
• 10+ years of experience, including at least 8 years in leadership.
• Industry experience in preclinical drug development, with hands-on work across small-molecule and genomic therapy programs.
• Strong expertise in mouse genetics, in vivo pharmacology, DMPK, PK/PD modeling, safety pharmacology, preclinical toxicology, and gene or biologic therapies.
• Experience designing mouse-model studies and developing or using iPSC-derived disease models.
• Experience implementing quality systems, GLP or GLP readiness, data integrity, reproducibility, project management, and regulatory communication.

Nice to have

• Therapeutic experience in neurodegenerative, neurodevelopmental, cardiac, or rare genetic diseases.
• Direct experience preparing pre-IND or IND packages, particularly for gene therapies.
• Publications, patents, or recognized contributions in gene therapy, iPSC modeling, translational pharmacology, or preclinical safety.
• Experience with AAV vector engineering, manufacturing considerations, nanoparticles, or other delivery modalities.

Culture & Benefits

• Work within an independent nonprofit research institution with nearly 3,000 employees across the United States, Japan, and China.
• Collaborate across genetics, translational research, clinical application, and biomedical data initiatives.
• Relocation assistance is available for the Bar Harbor position.
• Equal employment opportunities are provided in accordance with applicable law.