Senior Principal Scientist

Role Summary

Senior Principal Scientist role in Cambridge, MA within Sanofi's Rare Diseases Research team. You will lead transformative drug discovery for patients with rare Mendelian disorders, serving as a strategic scientific leader from target identification to clinical candidate selection. Leverage deep expertise in metabolic disease biology to shape our rare disease pipeline and translate mechanistic insights into breakthrough therapeutic strategies at the intersection of academia and pharmaceutical development.

Responsibilities

• Build drug discovery programs employing multiple modalities (small molecules, biologics, antisense oligonucleotides, gene therapy, etc.) targeting metabolic and mitochondrial disorders.
• Define research strategy and drive decision-making for rare disease programs from target validation through IND-enabling studies.
• Serve as the scientific voice for programs in governance forums, providing strategic recommendations that shape portfolio priorities.
• Identify and evaluate emerging therapeutic opportunities in metabolic and mitochondrial diseases through competitive intelligence and scientific landscape analysis.
• Design and oversee the execution of sophisticated in vitro and in vivo studies to elucidate disease mechanisms, validate targets, and establish proof-of-concept for novel interventions.
• Drive mechanistic understanding of rare metabolic disorders.
• Integrate multi-omics approaches and systems biology to identify disease drivers and therapeutic intervention points.
• Translate preclinical findings into clinical development strategies in collaboration with translational medicine teams.
• Lead or contribute to cross-functional project teams spanning discovery research, translational medicine, clinical development, and regulatory affairs.
• Build and maintain strategic relationships with leading academic researchers, clinician-scientists, and patient advocacy organizations in the rare metabolic disease community.
• Manage external collaborations including CROs, academic partnerships, and biotech alliances to accelerate program advancement.
• Represent Sanofi's scientific leadership at conferences, advisory boards, and collaborative research forums.
• Generate rigorous data analyses, interpretations, and documentation to support program milestones, regulatory submissions (IND/CTA), patent filings, and publications.
• Communicate complex scientific concepts clearly and persuasively to diverse audiences including senior leadership, external collaborators, and the broader scientific community.
• Mentor and develop junior scientists, fostering a culture of scientific excellence and innovation.

Qualifications

• Required: Ph.D. or equivalent advanced degree in biochemistry, metabolism, mitochondrial biology, molecular/cellular biology, genetics, or related discipline.
• Required: Minimum of 7+ years of post-graduate experience in academic or biopharmaceutical research, with substantial focus on metabolic and/or mitochondrial disorders.
• Required: Deep expertise in neurological, neuromuscular, metabolic, and/or mitochondrial disorders.
• Required: Proven drug discovery leadership experience in industry with a track record of contributions to multidisciplinary teams and advancing therapeutic programs toward clinical development.
• Preferred: Demonstrated contributions to IND/CTA-enabling activities, including target validation, mechanism-of-action studies, biomarker development, and translational research.
• Preferred: Strong publication record in high-impact journals demonstrating scientific thought leadership in relevant disease areas.
• Preferred: Established network within the academic, clinical, and patient advocacy communities focused on rare metabolic and mitochondrial diseases.
• Preferred: Experience with relevant disease models including patient-derived cells, organoids, and animal models.
• Preferred: Expertise designing and incorporating metabolomics, lipidomics, or other specialized experimental modalities.
• Preferred: Familiarity with computational biology, bioinformatics, or analysis of large-scale 'omics datasets.
• Preferred: Experience with multiple therapeutic modalities and platform technologies.
• Preferred: Prior involvement in regulatory document preparation and interactions with health authorities.
• Preferred: Understanding of the rare disease drug development landscape, including regulatory pathways and reimbursement considerations.
• Preferred: Experience presenting at scientific conferences and contributing to the external scientific discourse in rare diseases.

Skills

• Exceptional scientific problem-solving abilities with capacity to integrate complex datasets and drive mechanistic insights.
• Strong experimental design skills with rigorous attention to scientific quality and reproducibility.
• Deep understanding of drug discovery processes from target identification through clinical candidate selection.
• Proven ability to lead, influence, and inspire multidisciplinary teams in a matrixed organization.
• Strategic mindset with capability to balance scientific rigor with business priorities and timelines.
• Outstanding written and verbal communication skills with ability to tailor messages for diverse audiences.