Executive Medical Director, Clinical Development

AveXis Inc.
Bannockburn, IL, US
Oct 07, 2018
Required Education
Position Type
Full time

Ready to join a team committed to moving gene therapies into the clinical setting 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 at the beginning of an incredible journey, and are looking for passionate individuals to join us on this important mission.

AveXis, Inc. 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.

We are seeking to recruit a physician executive who is experienced in neurology drug development as Executive Medical Director, Clinical Development. Reporting to VP, Clinical Development, this executive will be an integral member of the AveXis drug development organization and will contribute significantly to the advancement of programs into and through the clinic. The near-term priority for this individual is to champion the development of AVXS-101 for the treatment of SMA. The direct responsibilities of this position will cover Phase 1 through Phase 3 activities, expanding and advancing the company's programs from early clinical trial development to proof-of-concept and market approval. This role will require collaboration with the clinical operations, business strategy and commercial teams to optimize the success of AVXS-101. This will be a high profile and high impact position, responsible for executing and delivering on critical clinical milestones. The individual hired for this position will, therefore, have significant influence shaping the future of the company.


  • Evaluate and understand the unmet medical needs in SMA and other rare neurologic disorders.
  • Establish and approve scientific methods underlying the design and implementation of clinical protocols.
  • Design and manage clinical studies in collaboration with the Biometrics, Clinical Operations, and Medical Affairs groups, including supervising or writing protocols and managing the clinical programs.
  • Recruit, guide, and motivate clinical investigators.
  • Act as and/or supervise Medical Monitor for AveXis sponsored clinical trials as needed and provide adverse event safety monitoring and reporting.
  • Author or supervise writing relevant sections of Clinical Study Reports, BLA reports, investigator brochures, and other clinical/regulatory/safety documents.
  • Educate AveXis staff and external audiences on the science and clinical applications of the program.
  • Present technical information to internal teams; represent AveXis externally where necessary through publications, presentations at scientific meetings and congresses.
  • Assist in medical affairs activities which may include medical information efforts, FAQ document creation, health economic efforts, etc.
  • Assist with business development and pipeline activities.


  • MD or MD/PhD in Neurology or Pediatrics is strongly preferred; pediatric neurology training would be ideal. Physicians with training in other medical specialties will be considered, but must have substantial industry experience in design, oversight and reporting of CNS clinical trials.
  • Minimum four to ten years' experience in the design, execution and evaluation of clinical trials, with an emphasis on phases 2-3.
  • Substantive experience in the pharmaceutical/biotechnology industry and in-depth understanding of pharmaceutical regulatory requirements and their impact on the development of clinical trials and BLAs.
  • An active network in key segments of the CNS field is strongly preferred. This will include KOLs, key research centers, patient advocacy groups and a variety of patient care organizations.
  • Able to write clearly and succinctly in a variety of communication settings; open, transparent and succinct communication skills, both verbal and written.
  • Experience designing and managing clinical trials.
  • Familiarity with statistics.
  • Advanced knowledge of Microsoft Word, Excel and PowerPoint preferred (knowledge of Gmail, Google Apps and Adobe Acrobat desired).
  • Proven leadership and critical decision making skills.
  • Exceptional analytical, critical thinking, and problem solving abilities; strong attention to detail.
  • Demonstrated organizational and collaboration skills.
  • Ability to deliver professional and quality work products while working on multiple projects.
  • Desire and ability to work in a fast-paced, dynamic start-up environment.

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.