Senior Director, Discovery & Translational De

Key Responsibilities

• Lead and manage a team responsible for advancing multiple discovery and translational development programs from early concept through IND-enabling readiness.
• Provide scientific and strategic leadership across programs in rare neuromuscular and cardiac genetic diseases, including Friedreich’s ataxia, CPVT, emerging genetic cardiomyopathy programs, and additional pipeline opportunities.
• Partner with scientific leads to define therapeutic hypotheses, translational strategies, development plans, critical path activities, and key decision points.
• Drive disciplined program progression through clear go/no-go criteria, risk assessment, scenario planning, and milestone-based decision-making.
• Ensure discovery and translational strategies are aligned with clinical development, regulatory expectations, CMC considerations, and portfolio priorities.
• Help translate early scientific opportunities into development-ready programs with clear strategic rationale and execution plans.

Team Leadership & Organizational Development

• Lead, mentor, and develop a high-performing team of scientific leads and translational development professionals.
• Mentor directly the clinical assay leadership function and ensure clinical assay strategy is integrated into program and portfolio plans.
• Foster a culture of scientific rigor, accountability, collaboration, transparency, and disciplined decision-making.
• Help build scalable processes, expectations, and operating models for the growing DTD organization.
• Serve as a trusted partner to the Head of Discovery & Translational Development in managing team priorities, portfolio execution, and organizational growth.

Translational Strategy & Development Execution

• Oversee translational development strategies, including mechanism-of-action studies, biodistribution, dose-response, pharmacology, biomarker strategy, and proof-of-concept studies.
• Ensure preclinical models, endpoints, and assays are fit-for-purpose and aligned with the intended clinical and regulatory strategy.
• Integrate discovery, nonclinical, clinical, assay, and emerging translational data to inform program direction and portfolio decisions.
• Anticipate scientific, translational, regulatory, and execution risks and develop mitigation strategies.
• Ensure programs are designed with a clear line of sight to IND-enabling development, clinical translation, and future regulatory interactions.

Clinical Assay & Biomarker Oversight

• Provide strategic and managerial oversight of clinical assay and biomarker efforts supporting advancing and clinical-stage programs.
• Ensure clinical assay strategies are scientifically rigorous, operationally feasible, and aligned with clinical development and regulatory expectations.

Cross-Functional & Executive Leadership

• Serve as a senior DTD representative in cross-functional program teams, governance forums, and executive-level discussions.
• Partner closely with Clinical Development, Regulatory Affairs, CMC, Program & Portfolio Management, Business Development, Finance, and external collaborators.
• Develop clear, concise, and decision-enabling scientific narratives for senior leadership and governance forums.
• Frame program options, risks, tradeoffs, and recommendations to support disciplined portfolio decisions.
• Represent DTD externally with academic collaborators, CROs, KOLs, and strategic partners.

Regulatory & Documentation Support

• Contribute to IND-enabling strategy, briefing documents, regulatory interactions, and scientific content supporting regulatory submissions.
• Ensure nonclinical and translational packages are coherent, rigorous, and aligned with agency expectations.
• Support preparation of high-quality internal and external documentation, including study reports, executive summaries, data packages, and governance materials.