Principal Scientist - Rare Diseases

Job title: Principal Scientist - Rare Diseases

• Location: Cambridge, MA

About the job

As Principal Scientist - Rare Diseases within our R&D team, you will join Sanofi's Rare Diseases Research team and lead transformative drug discovery efforts for patients with rare Mendelian disorders. In this role, you will serve as a strategic scientific leader, driving innovation from target identification through clinical candidate selection. This position offers the unique opportunity to shape our rare disease pipeline by leveraging your deep expertise in metabolic disease biology and proven track record of drug discovery.

At Sanofi, we are committed to pursuing the miracles of science to improve people's lives. You will translate cutting-edge mechanistic insights into breakthrough therapeutic strategies, working at the intersection of academic innovation and pharmaceutical development. Your leadership will be instrumental in advancing novel treatments for patients with limited or no therapeutic options, including those suffering from inborn errors of metabolism, mitochondrial diseases, and related rare metabolic conditions.

Join the engine of Sanofi’s mission — where deep immunoscience meets bold, AI-powered research. In R&D, you’ll drive breakthroughs that could turn the impossible into possible for millions.

About Sanofi

We’re an R&D-driven, AI-powered biopharma company committed to improving people’s lives and delivering compelling growth. Our deep understanding of the immune system – and innovative pipeline – enables us to invent medicines and vaccines that treat and protect millions of people around the world. Together, we chase the miracles of science to improve people’s lives.

Main Responsibilities

Strategic Leadership & Program Direction

• Build drug discovery programs employing a variety of therapeutic modalities (small molecule, 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

Scientific Execution & Innovation

• Design and oversee the execution of sophisticated in vitro and in vivo studies to elucidate disease mechanisms, validate therapeutic 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

Cross-Functional Collaboration & External Partnerships

• 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

Scientific Communication & Documentation

• 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

About You

Required Qualifications

• Ph.D. or equivalent advanced degree in biochemistry, metabolism, mitochondrial biology, molecular/cellular biology, genetics, or related discipline

• Minimum of 7+ years of post-graduate experience in academic or biopharmaceutical research, with substantial focus on metabolic and/or mitochondrial disorders.

• Deep expe