Sr Director, Clinical Development (Rare Disease)

Senior Director, Clinical Development

We are seeking an experienced and innovative Senior Director, Clinical Development to provide strategic and operational leadership for early-stage rare disease drug development programs. This role will play a pivotal part in shaping preclinical-to-clinical strategy, preparing for regulatory engagement, and ensuring the successful transition into first-in-human studies. Ideal for candidates who thrive in agile, entrepreneurial biotech environments, this position offers the opportunity to impact multiple high-potential programs from the ground up.

Key Responsibilities

• Serve as the medical lead for rare disease programs from pre-IND through early clinical phases.

• Partner with preclinical and translational teams to define target product profiles and clinical development pathways.

• Lead the design of early-stage clinical development strategies, including biomarker selection, patient population definition, and study endpoints.

• Oversee or contribute to authoring key clinical and regulatory documents, including INDs, clinical protocols, investigator brochures, and regulatory briefing packages.

• Collaborate with Regulatory Affairs to prepare for FDA, EMA, and other agency interactions.

• Engage and build relationships with key opinion leaders, patient advocacy organizations, and external collaborators.

• Provide medical expertise in evaluating new opportunities for portfolio expansion.

• Maintain up-to-date knowledge of scientific, clinical, and regulatory developments in rare disease research.

Qualifications

Education

• MD or MD/PhD required; board certification in a relevant specialty preferred.

Experience

• 5+ years of clinical development experience, with a focus on rare disease or orphan indications.

• Direct experience in early-stage (pre-IND through Phase 1/2) drug development.

• Demonstrated ability to operate effectively in small, fast-paced biotech or startup environments.

• Familiarity with global regulatory requirements and experience preparing for agency meetings.

Skills

• Deep understanding of clinical trial design and translational medicine principles.

• Excellent written and verbal communication skills; comfortable presenting to scientific and non-scientific audiences.

• Entrepreneurial mindset with the ability to adapt to shifting priorities.