Director, Translational Medicine Expert; Neurodegeneration
Listed on 2026-09-28
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Research/Development
Clinical Research
Job Description Summary #LI-Hybrid Internal
Title:
Director
Location:
Cambridge, MA, USA
The Director, Translational Medicine Expert (Neurodegeneration) will lead the end to end translational strategy for neurodegenerative programs, bridging preclinical research to early clinical development (Phase 1–2). This role is accountable for biomarker strategy, clinical endpoint selection, and early phase trial design, enabling efficient, data driven decision-making through key development milestones (e.g., Transition Decision Points) and ensuring competitive differentiation of neuromuscular assets.
In addition, he/she oversees the planning, execution, and interpretation of clinical trials research, data collection activities and clinical operations. Establishes and approves scientific methods for design and implementation of clinical protocols, data collection systems and final reports. Support new and ongoing clinical research and clinical trials and ensure efficient and timely processing of confidentiality agreements and clinical agreements
Key Responsibilities:
Molecular biomarkers (tissue, blood, CSF where applicable) Functional endpoints (strength, mobility, disease-specific measures) Patient-reported outcomes (PROs) Establishing quantitative biomarkers to support:
Target engagement Disease modification Dose selection and PoC decisions Integrate biomarker strategy into clinical protocols and operational plans Drive early biomarker de‑risking, a key priority in neuromuscular programs
Dose escalation strategy Exposure–response framework Translational endpoints (biopsy, imaging, functional) Ensure TM leadership within Clinical Trial Teams (CTTs) for:
Trial design Execution oversight Data interpretation and decision-making
Clinical Development Biomarker Development Clinical Pharmacology (PKS) Preclinical Safety (PCS) Regulatory Drive alignment between research, TM, and development organizations
Academic KOLs Patient advocacy groups (critical in rare neuromuscular disease) Technology partners (imaging, omics platforms) Contribute to scientific strategy and external positioning of programs
Meaningful functional endpoints Patient‑relevant outcome measures Innovative trial approaches (e.g., decentralized models where feasible)
Asset prioritization and portfolio strategy Competitive differentiation strategy (e.g., vs RNA or gene therapy competitors) Provide scientific input to:
Business development / due diligence Indication expansion strategies
- MD, neurologists, clinical training in neurodegenerative diseases
- Experience in translational medicine, early clinical development, or experimental therapeutics.
- Ability to shape development strategies, execute early clinical studies, and influence regulatory/packages supporting…
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