Director, Translational Medicine Expert (Neuromuscular)
About The Position
The Director, Translational Medicine (Neuromuscular) will lead the end to end translational strategy for neuromuscular 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
Requirements
- MD, clinical subspecialty training in neurological diseases
- Experience in translational medicine, early clinical development, or experimental therapeutics.
- Proven ability to shape development strategies, execute early clinical studies, and influence regulatory/packages supporting differentiation.
Nice To Haves
- Experience working in pharmaceutical/biotech company strongly preferred.
- PhD, PharmD, or equivalent in: Neurology / Neuroscience, Neuromuscular disease
Responsibilities
- Define and execute integrated translational strategies across neuromuscular programs (e.g., gene therapy, RNA therapeutics)
- Lead translational components from preclinical → Phase 1/2 clinical studies
- Drive program progression through key decision gates (e.g., IND, TDP, IDPA)
- Ensure strong mechanistic linkage between target biology, biomarkers, and clinical endpoints
- Collaborate with Biomarker Development colleagues toward development and validation of: 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
- Co‑lead design and execution of early clinical studies
- Define: 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
- Serve as core Translational Medicine representative within global project teams
- Partner closely with: Clinical Development, Biomarker Development, Clinical Pharmacology (PKS), Preclinical Safety (PCS), Regulatory
- Drive alignment between research, TM, and development organizations
- Build and lead collaborations with: Academic KOLs, Patient advocacy groups (critical in rare neuromuscular disease), Technology partners (imaging, omics platforms)
- Contribute to scientific strategy and external positioning of programs
- Integrate the patient voice into translational and clinical strategies
- Advance: Meaningful functional endpoints, Patient-relevant outcome measures, Innovative trial approaches (e.g., decentralized models where feasible)
- Contribute to: 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
Benefits
- health, life and disability benefits
- a 401(k) with company contribution and match
- a variety of other benefits
- a generous time off package including vacation, personal days, holidays and other leaves
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What This Job Offers
Job Type
Full-time
Career Level
Director
Education Level
Ph.D. or professional degree
