Principal Scientist

Develop and lead biomarker strategies for neuromuscular preclinical programs, ensuring alignment with program objectives.

Serve as a biomarker and translational science subject matter expert on cross-functional program teams, providing scientific guidance on biomarker selection, implementation, and interpretation.

Integrate biological hypotheses and mechanism of action into fit-for-purpose biomarker approaches that inform target engagement, pharmacodynamic response, and disease progression.

Identify scientific and technical opportunities, risks, and gaps within program biomarker plans and develop strategies to address them.

Identify and manage CRO’s, design and execute rodent pharmacology studies to enable translation of PK/PD/efficacy to the clinic.

Lead the design, development, qualification, and implementation of fit-for-purpose biomarker assays across relevant matrices including fluid, tissue, imaging, molecular, and cellular biomarkers.

Maintain an active laboratory presence, personally designing, executing, and troubleshooting experiments to develop and evaluate biomarker assays, as needed.

Collaborate closely with Discovery, Bioanalytical, and Clinical Pharmacology to translate mechanistic findings and preclinical biomarker hypotheses into clinically feasible and informative approaches.

Critically analyze and integrate complex biomarker datasets, clearly communicating conclusions, limitations, and implications for program decision-making.

Maintain awareness of emerging biomarker technologies and scientific advances relevant to the portfolio.

PhD in skeletal muscle biology, immunology, neuroscience, pharmacology, or related discipline.

8+ years of experience in biotech/pharma R&D, preferably in neuromuscular disease, with a strong track record in biomarker discovery and development.

Experience implementing biomarker strategies that support drug discovery and preclinical drug development.

Deep expertise in one or more biomarker disciplines with working knowledge across relevant technologies such as immunoassays, bioinformatics, imaging, flow cytometry, and molecular assays.

Experience providing managerial and scientific oversight of CROs, external laboratories, technology vendors, and/or academic collaborators.

Ability to work independently, manage multiple priorities, and drive complex scientific initiatives in a fast-paced, highly collaborative environment.

Passion for rare disease research and a commitment to translating rigorous science into meaningful benefits for patients.