Senior Clinical Scientist, Rare Disease (PhD)

Senior Clinical Scientist, Rare Disease (PhD)

We are seeking a PhD-level Clinical Scientist to support the Biologics License Application (BLA) submission for a Breakthrough Therapy-designated biologic targeting a neurodegenerative rare disease.

This role will contribute to late-stage clinical development, supporting clinical data interpretation, regulatory documentation, and cross-functional collaboration as the program advances toward potential approval.

Key Responsibilities

• Support late-stage clinical development and BLA submission activities

• Interpret and synthesize clinical efficacy, safety, and biomarker data in pediatric populations

• Author and review clinical sections of regulatory documents (Modules 2 and 5)

• Collaborate cross-functionally with Regulatory, Clinical Operations, Biostatistics, and Medical Affairs

• Support FDA interactions and regulatory meetings as needed

Qualifications

• PhD in a life sciences discipline

• 5+ years of clinical development experience in biotech or pharma

• Experience supporting late-stage programs and regulatory submissions (BLA/NDA/MAA)

• Strong scientific writing and cross-functional collaboration skills

• Experience in rare disease, pediatric, neurodegenerative, or biologics programs preferred

Why This Role

This is an opportunity to contribute to a Breakthrough Therapy-designated biologic at a critical regulatory milestone, advancing a program aimed at addressing serious unmet need in pediatric neurodegenerative disease.