Chief Science Officer

About the foundation

NIPBL Therapeutics Foundation is a nonprofit advancing therapeutic development for NIPBL-related Cornelia de Lange syndrome (CdLS). Pathogenic variants in NIPBL, which encodes the cohesin-loading factor, are the most common cause of CdLS, a multi-system developmental diagnosis with no approved treatment.

We set a focused research agenda, fund critical science, and coordinate academic laboratories, clinicians, and biotech partners to advance the most promising approaches toward the clinic. Learn more at nipbl.org.

The opportunity

Lead the scientific engine of our therapeutic programs. You will translate NIPBL and cohesin biology into a disciplined development program focused on drug repurposing and small-molecule validation, hold research partners to decision-ready results, and build the evidence base toward an Investigational New Drug (IND) application.

What you will own

• Scientific strategy and roadmap. Build an integrated 18-month scientific roadmap with milestones, budgets, and explicit go/no-go criteria; recommend which studies to start, expand, pause, or stop.
• Partner-led research. Direct academic laboratories, biotech partners, and CROs; define scopes of work, evidence standards, and deliverables; review primary data and resolve bottlenecks.
• Drug repurposing and validation. Guide the expansion of patient-based compound screening, independently assess computational predictions, and prioritize compounds for orthogonal validation.
• Disease biology and models. Oversee variant-function studies and iPSC, organoid, and animal-model work; evaluate reproducibility, genotype coverage, and NIPBL dosage sensitivity.
• Path toward the clinic. With regulatory, toxicology, and manufacturing advisors, identify evidence gaps for a lead candidate and build a staged, costed pre-IND plan.
• Translational and patient-data alignment. With the Clinical Program & Community Manager, connect preclinical questions to patient outcomes, natural history data, and biomarkers.
• Scientific governance. Run scientific reviews with the Scientific & Strategic Advisory Board, maintain a program risk register, and work with counsel on data access, publication, and IP terms.
• Stakeholder reporting and communications. Deliver scientific updates, risk assessments, and milestone forecasts; support grant proposals, donor diligence, and partner discussions.

Requirements

• Typically 10+ years in translational research and drug development, including scientific leadership roles.
• A PhD, MD, or equivalent advanced training in a relevant life-science or medical field.
• A record of advancing a therapeutic program from discovery or preclinical work toward clinical development.
• Experience directing academic labs, biotechs, and CROs, including scopes of work, deliverables, and study budgets.
• Rigorous evaluation of functional assays, transcriptomic data, and patient-derived models, and clear communication with scientists and funders.

Preferred experience

• Rare genetic or neurodevelopmental disease, especially pediatric.
• Small-molecule drug repurposing.
• Cohesin, chromatin, or gene-regulation biology.
• iPSC, organoid, or animal-model programs.
• Pre-IND meetings or other FDA interactions.