Medical Director

AveXis Inc.
Bannockburn, IL
Apr 14, 2019
Required Education
Position Type
Full time


Ready to join a team committed to moving gene therapies into the clinical and commercial settings for patients and families devastated by rare neurological genetic diseases? AveXis is advancing cutting-edge science to treat rare and life-threatening genetic diseases starting with our clinical-stage, proprietary gene therapy candidate, AVXS-101. We are in the midst of an incredible journey and are looking for passionate individuals to join us on this important mission.

AveXis, Inc., a Novartis Company , is a clinical-stage gene therapy company, dedicated to developing and commercializing novel treatments for patients suffering from rare and life-threatening neurological genetic diseases. Our initial product candidate, AVXS-101, is our proprietary gene therapy product candidate currently in development for the treatment of spinal muscular atrophy, or SMA, Type 1, the leading genetic cause of infant mortality, and for the treatment of SMA Type 2. The U.S. Food and Drug Administration, or FDA, has granted AVXS-101 Orphan Drug Designation for the treatment of all types of SMA and Breakthrough Therapy Designation, as well as Fast Track Designation for the treatment of SMA Type 1. In addition to developing AVXS-101 to treat SMA Type 1 and Type 2, we also plan to develop other novel treatments for rare neurological diseases, including Rett syndrome (RTT) and a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene.

The Medical Director will be responsible for assisting in the design, conduct, execution and reporting of clinical studies in rare neurologic conditions, such as frontotemporal dementia, Friedreich's Ataxia, tauopathies and other rare neurologic disorders. The Medical Director will need to work collaboratively with line management and other functional lines to assist in developing coordinated and aligned clinical plans. The Medical Director will contribute to global regulatory submissions, providing written and verbal clinical, clinical development and scientific input. The Medical Director will serve as an interface with regulatory, clinical operations, manufacturing, commercial, and medical affairs groups, depending on capabilities, to appropriately communicate information and assure coordinated delivery on goals.




  • Assists in oversight and management of clinical studies including planning, execution, completion and reporting of clinical trials according to all applicable regulations and guidance, ICH/GCP, and SOPs.
  • Partners with Clinical Operations to ensure all clinical study activities are completed efficiently and on time in accordance with applicable regulations and guidance.
  • Review clinical data from all phases of development and assist in generating study reports, publications and regulatory documents.
  • Coordinate, and provide ongoing assessment, evaluation, and communication with other departments (e.g., Regulatory, Drug Safety, and Clinical Trial Materials) and external vendors (CRO, consultants, etc.) to ensure study objectives/timelines are accomplished.
  • Serve as study medical monitor.
  • Identify and assist in managing external collaborators, investigators, key clinical academic experts and consultants.
  • Provide management personnel with timely updates on progress and proposed changes in scope, schedule, and resources as required.
  • Ensure compliance with all applicable regulatory standards related to global clinical trials and interactions with physicians.
  • Develop and maintain professional relationships with academic and community-based study sites.
  • Assist in interfacing with departments within and outside of R&D including Finance, Regulatory Affairs, Commercial, Legal Affairs and Medical Affairs.
  • Assist in incorporating Health Economics Outcomes Research requirements into protocol design.








  • Medical Degree (MD) with added qualifications in Neurology or Pediatrics and a strong scientific background.
  • At least 1 year of relevant experience in drug development at a biotech/pharmaceutical company. Candidates with Frontotemporal dementia (FTD), Friedreich's Ataxia, tauopathy, or other rare neurologic disease experience in academia but without industry experience will be considered at the Medical Director level. Candidates with at least a year of industry experience in one or more of these disorders are preferred.
  • Able to rapidly learn regulatory procedures and guidelines in the U.S.
  • Able to assist in clinical study design, monitoring and execution and results analysis.
  • Has demonstrated knowledge of designing, implementing or conducting clinical studies, to produce both timely and good quality data.
  • Proven record of working on teams and in a matrix environment.
  • Additional postdoctoral training or additional qualifications such as PhD, PharmD, certificate in Pharmaceutical Medicine.
  • Prior experience in EU/US regulatory filing.
  • Experience in development of orphan drug products, neurology products, especially orphan neurology products.
  • Demonstrable working-knowledge of regulatory procedures and guidelines in all geographic regions (global scope).


The level of this position will be based on the final candidate's qualifications.

Please note this job description is not designed to cover or contain a comprehensive listing of activities, duties or responsibilities that are required of the employee for this job. Duties, responsibilities and activities may change at any time with or without notice.

AveXis is committed to creating a diverse environment and is proud to be an equal opportunity employer. All qualified applicants will receive consideration for employment without regard to race, color, religion, gender, gender identity, national origin, genetics, disability, age, sexual orientation or veteran status.