Director, Translational Medicine Expert (Neuromuscular)

Band Level 6 Job Description Summary #LI-Hybrid Location: San Diego, CA 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 Job Description Key Responsibilities: 1. Translational Strategy & Program Leadership 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 2. Biomarker & Endpoint Leadership 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 3. Early Clinical Development (Phase 1/2) 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 4. Cross-Functional Leadership 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 5. External Scientific Leadership 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 6. Patient-Centered Development 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) 7. Portfolio Contribution & Strategic Input 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 Essential 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. Desirable Requirements: Experience working in pharmaceutical/biotech company strongly preferred. PhD, PharmD, or equivalent in: Neurology / Neuroscience Neuromuscular disease The salary for this position is expected to range between: $248,500-$461,500/year The final salary offered is determined based on factors like, but not limited to, relevant skills and experience, and upon joining Novartis will be reviewed periodically. Novartis may change the published salary range based on company and market factors. Your compensation will include a performance-based cash incentive and, depending on the level of the role, eligibility to be considered for annual equity awards. US-based eligible employees will receive a comprehensive benefits package that includes health, life and disability benefits, a 401(k) with company contribution and match, and a variety of other benefits. In addition, employees are eligible for a generous time off package including vacation, personal days, holidays and other leaves. To learn more about the culture, rewards and benefits we offer our people click here. EEO Statement: The Novartis Group of Companies are Equal Opportunity Employers. We do not discriminate in recruitment, hiring, training, promotion or other employment practices for reasons of race, color, religion, gender, national origin, age, sexual orientation, gender identity or expression, marital or veteran status, disability, or any other legally protected status. We strive to create an inclusive workplace that cultivates bold innovation through collaboration and empowers our people to unleash their full potential. Accessibility and reasonable accommodations The Novartis Group of Companies are committed to working with and providing reasonable accommodation to individuals with disabilities. If, because of a medical condition or disability, you need a reasonable accommodation for any part of the application process, or in order to perform the essential functions of a position, please send an e-mail to View email address on click.appcast.io call View phone number on click.appcast.io and let us know the nature of your request and your contact information. Please include the job requisition number in your message. Salary Range $248,500.00 - $461,500.00 Skills Desired Clinical Trials, Cross-Functional Teams, Data Analysis, Data Monitoring, Drug Development, Drug Discovery, Medical Research, Medical Strategy, People Management Improving the lives of people living with disease takes more than innovative science. It takes a focus on the needs of people and a community committed to meeting them. It takes a team of people like you. Working together. Learning together. Thriving together. Discover how you can join us in changing people’s lives. 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