Director, Discovery & Translational Development

Role Summary

Director, Discovery & Translational Development will provide scientific and strategic leadership for AAV-based gene therapy programs targeting neuromuscular diseases, with a primary focus on Duchenne Muscular Dystrophy (DMD). This role sits at the nexus of discovery, translational science, and early clinical development and is accountable for driving data-driven decisions that advance programs from preclinical development through IND and into the clinic. The Director will lead translational strategy, oversee preclinical and IND-enabling studies, and serve as a key scientific partner to Clinical, Regulatory, CMC, Bioanalytics, and Program Management. Location: Charlestown, MA.

Responsibilities

• Scientific & Translational Leadership
  • Own and drive the translational strategy for neuromuscular gene therapy programs, ensuring alignment between preclinical data, biomarker strategy, and clinical interpretation.
  • Provide scientific leadership for mechanism of action, biodistribution, transgene expression, durability, and functional efficacy across relevant disease models.
  • Define and oversee biomarker frameworks that link nonclinical data to clinical endpoints and regulatory expectations.
  • Establish data standards, success criteria, and decision points to support confident program advancement.
• Program Ownership & IND-Enabling Execution
  • Provide scientific oversight and accountability for IND-enabling studies, including study design, CRO execution, data review, and interpretation.
  • Contribute to and review regulatory-facing documents (e.g., study reports, investigator brochures, briefing packages, responses to agency questions).
  • Partner with Program Management to ensure timelines, risks, and dependencies are clearly defined and proactively managed.
  • Support development and refinement of Target Product Profiles (TPPs) and program risk-mitigation strategies.
• Cross-Functional Leadership
  • Serve as a core scientific representative on cross-functional program teams, working closely with Clinical Development, Regulatory Affairs, CMC, Bioanalytics, and Quality.
  • Communicate complex scientific concepts and data clearly to senior leadership and executive audiences, enabling informed decision-making.
  • Represent DTD in internal governance forums and, as appropriate, external scientific or regulatory interactions.
• Team Leadership & Development
  • Provide scientific mentorship and leadership to scientists within DTD, fostering rigor, accountability, and translational thinking.
  • Guide experimental planning and data interpretation across the team without necessarily being hands-on at the bench.
  • Contribute to building scalable DTD processes, platforms, and best practices.

Qualifications

• PhD in Muscle Biology, Neuroscience, Molecular Biology, Cell Biology, Genetics, Pharmacology, or a related life sciences discipline.
• Postdoctoral training in neuromuscular disease or gene therapy is preferred.
• 8–12+ years of relevant experience, with substantial experience in biotechnology or pharmaceutical industry settings.
• Direct experience leading or significantly contributing to neuromuscular disease programs, preferably DMD.
• Demonstrated experience advancing AAV gene therapy programs through preclinical development and IND submission.
• Experience engaging with or supporting regulatory interactions (FDA, EMA) is strongly preferred.
• Deep familiarity with neuromuscular disease models and translational endpoints.
• Expertise in molecular, histological, and functional assays relevant to gene therapy.
• Strong data interpretation and critical assessment skills; able to integrate complex datasets across modalities.
• Experience overseeing CROs and external collaborators.

Skills

• Deep expertise in neuromuscular disease biology, with a strong emphasis on DMD.
• Demonstrated leadership in AAV-based gene therapy development, including translational and IND-supporting work.
• Strong translational mindset with the ability to bridge preclinical data to clinical hypotheses and regulatory strategy.
• Strategic thinking combined with scientific rigor and attention to detail.
• Proven ability to influence across functions and operate effectively in a matrixed organization.
• Clear, concise scientific communication tailored to diverse audiences.