Medical Director

About us

Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, clinical-stage gene therapy company founded in 2001 based on the work of adeno-associated virus (AAV) gene therapy pioneers, Jude Samulski, PhD, and Xiao Xiao, PhD. We are driven to change lives with every clinical advancement and turn hope into cures with new possibilities for genetic medicine. We currently have clinical trials ongoing for late-onset Pompe disease and congestive heart failure.

AskBio is headquartered in Research Triangle Park, North Carolina, a thriving biotechnology hub, and has additional research and development facilities in Scotland and France and gene therapy manufacturing in Spain.

We are looking for a Medical Director – Translational Medicine/Clinical Development to join the AskBio team.

About the role

AskBio is seeking a Medical Director in translational medicine/clinical development. Reporting to the VP of Translational Medicine, this person will be responsible for developing and championing clinical development strategies and study designs for pre-IND and IND submissions and supporting ongoing clinical trials in neuromuscular, CNS and metabolic disease indications. S/he will be responsible for integrating the knowledge base of scientific mechanisms and non-clinical studies into cogent endpoints and monitoring methods for the various indications and play a significant role in devising amendments and monitoring strategies for ongoing studies.

This is a hands-on position requiring a broad understanding of disease pathophysiology, gene therapy mechanisms and clinically relevant response elements that can be incorporated into FDA-ready strategies. A solid understanding of the regulatory process for advancing therapeutics through to approval is expected. Some interaction with senior management and business partners is expected to ensure timely decisions and commercial planning.


  • Lead delivery of clinical development activities for designated clinical-stage program(s) and ensure consistency with clinical development strategies
  • Actively contribute to cross-functional matrix teams and be a major contributor to translational and clinical components of clinical development plans
  • Help to ensure that clinical studies are designed and conducted effectively in partnership with relevant internal and external collaborators (e.g. CMC, non-clinical, clinical operations, clinical investigators and CROs, as appropriate)
  • Coordinate development plans and activities with R&D, non-clinical and translational medicine to ensure effective production of relevant regulatory documentation and supporting data
  • Participate in data review discussions and contribute to data interpretation
  • Provide clinical and translational inputs to the preparation of regulatory briefing and submission documents (IND, CTA, NDA, BLA, MAA)
  • Take active role in devising and writing of manuscripts and developing abstracts and presentation materials
  • Demonstrate knowledge and mastery of disease mechanisms, therapeutic goals and monitoring options for AskBio’s portfolio of clinical development indications and the broader pipeline
  • Thought leader for development projects within areas of expertise
  • Key translational representative on assigned discovery and pipeline projects
  • Key voice in evaluating pipeline projects and outside opportunities through due diligence and nominating process
  • Scientific reviewer of projects impacting a cross-company dimension
  • Proactively drives relevant stakeholder engagement
  • Contributes to scientific and clinical career development of scientists within therapeutic area
  • Aspires to be part of a dynamic and growing organization with the opportunity to pursue personal interests across functions and geographies

About you

  • MD or MD/PhD (will consider DVM, PharmD or PhD with right experience and background)
  • 5+ years of experience in the pharmaceutical/biotech industry
  • Leadership style with ability to navigate rapidly moving company strategies
  • Compassionate about patient care and therapeutic development for unmet medical needs
  • Strong experience in clinical program management – clinical development strategy, study design, trial management and patient reporting
  • Working knowledge of Good Clinical Practice (GCP) and other regulations governing clinical research
  • Clear understanding of disease models and their relevance to product development strategies
  • Proven track record leading cross-functional teams and ability to be an effective leader of global teams
  • Excellent verbal and written communication skills are a must

Preferred qualifications

  • Background or experience in neuromuscular, metabolic and/or neurologic diseases preferred, proficiency in immunoinflammatory conditions a plus
  • Experience working in the medical regulatory environment, including familiarity with regulatory submissions (IND, NDA, BLA, MAA)
  • Experience in both clinical and pre-clinical research (> 5 years) and experience in Ph2/3 clinical trial development
  • Ability to interpret complex biologic, non-clinical and clinical data, including pathobiology and imaging information
  • A working knowledge of gene therapy – AAV experience a plus
  • Demonstrated scientific credibility through publication of peer-reviewed studies in reputable scientific or clinical journals
  • Ability to influence project teams through sound scientific logic and convincing scientific and clinical argument
  • Demonstrated skills in networking internally and externally and communicating in situations requiring special tact and diplomacy
  • Operational knowledge of the pharmaceutical industry and broader R&D processes including scientific activity impacting project work
  • Demonstrated ability to lead peers in completing complex tasks, e.g., drafting protocols, selecting appropriate methodologies – measures, analytical plans and evaluation tools and building regulatory arguments
  • Ability to communicate and influence senior managers with strategic thinking, solution finding and streamlining plans