Director, Therapeutic Area SME

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 (ZOLGENSMA®). 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 Director will support the global Medical Affairs organization in executing the consolidated Medical Affairs strategy; ensuring an accurate, robust and appropriate medical and scientific exchange of knowledge and clinical expertise with US and global stakeholders; and partnering with internal stakeholders in driving success.

This role will work closely with the field teams, Medical Communications, and Med Information, and HEOR functions, as needed, in data generation activities, publications, and presentations (whether targeted to specific individuals, institutions, or at congresses) to (1) ensure the proper capture, interpretation, and analysis of external insights; (2) address any scientific decision support needs from inquiries coming into AveXis through all of the above functions and channels; and (3) provide remote and on-site technical expertise to institutions, providers and pharmacists to support successful therapeutic administration following marketing authorization of AVXS-101.




  • Supports engagement activities with Key Opinion Leaders (KOLs) and other key external stakeholders that are aligned with the US Medical Affairs plan.
  • Maintains in-depth understanding of the disease state, product, competitors, marketplace, related medical areas, and regulatory guidelines.
  • Acts as a primary resource to external stakeholders (e.g. caregivers, patients, healthcare professionals) but also internal AveXis personnel who request information on company products, ensuring accurate and appropriate responses.
  • Captures and provides timely insights on emerging clinical / scientific trends back to the organization.
  • Delivers timely, fair balanced, objective, scientific and economic information and education to health care customers, managed markets/healthcare/payer systems.
  • Actively contributes to the execution of company's strategy at scientific meetings in support of RMD meeting/booth coverage and KOL meetings.
  • Travel as needed to the stakeholder groups to proactively execute the above responsibilities and effect the desired outcomes.








  • M.D. / Doctorate with relevant clinical expertise required.
  • Minimum of five years of experience working in rare or orphan disease space.
  • Neurology, Neurodegenerative disease and/or pediatric specialty experience, in either an industry, clinical, or laboratory setting, a must.
  • Specific spinal muscular atrophy (SMA) experience, in either an industry, clinical, or laboratory setting, preferred.
  • Experience in interacting with other providers (HCPs), opinion leaders (KOLs), and researchers (PIs) a must.
  • Overall strong ability to understand the disease state, mechanism of action, value proposition - both from an absolute and relative/competitive standpoint - combined with speaking and presentations skills (i.e. gravitas) to ensure trustful and authoritative interactions with all stakeholders sets (KOLs, payers, patients, patient advocacy groups, etc.)


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.