Medical Director, Translational Medicine

Bannockburn | US-CA-San Diego, IL, US
Oct 08, 2019
Biotech Beach
Required Education
Bachelors Degree
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. It has been granted Orphan Drug and Fast Track Designations by the FDA, and is currently under review by the FDA, EMA and PMDA. We have product candidates for other diseases that are approaching first in human studies. AveXis is a subsidiary of Novartis that is being run as an independent entity, leveraging resources from Novartis where it makes sense.

AveXis is seeking a Medical Director who will, as part of the Translational Medicine team in R&D, be responsible for the successful translation of gene therapy products from the pre-clinical proof of concept stage in R&D to first-in human trials. This includes ensuring trial readiness with regards to the optimization of natural history dataset, biomarkers and clinical outcomes. This also includes stakeholder engagement with clinicians, researchers and patient groups, as well as the design of potentially label-enabling first-in-human trials. Building on AveXis gene replacement therapy technology platform, the Translational Medicine team drives the company's IND engine to accelerate the development of novel treatments for patients suffering from one of the many rare and life-threatening neurological genetic diseases.

The current focus of the role is on the company's programs that are nearing IND (AVXS-201 for Rett syndrome and AVXS-301 for genetic amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene). Starting in 2019, it is anticipated that there will be 2 - 5 gene therapy programs in the clinic each year.


  • Ensure milestone-gated IND submissions are planned and executed including alignment across the different modules, and responsiveness to FDA guidance relevant to gene therapy and rare diseases.
  • Work in cross-functional teams and serve as the primary Translational Medicine interface to peers in the clinical development, clinical operations, regulatory, CMC, project management, and business development functions.
  • Engage early with patients, families, researchers and clinicians, to better understand disease burden, unmet needs, meaningful endpoints, acceptable and feasible clinical development strategies, and opportunities to partner towards the shared goal of finding better treatment.
  • Optimize trial readiness in any given indication by pro-actively identifying and evaluating available tools and datasets, and by strategically fostering research to fill critical gaps including in natural history datasets, registries, clinical outcomes, biomarkers, pathways to rapid genetic diagnosis, observational study protocols and standard of care guidelines.
  • Provide strategic input to maximizing the IND engine's efficiency by building a platform that shares approaches between programs, including optimized delivery, formulation, monitoring and managing immune responses, IND submission processes, and innovative trial designs for rare genetic disease (e.g., adaptive, seamless Phase 1/2 approaches).
  • Maintain scientific awareness regarding advances in indications of interest, regulatory awareness regarding new guidance and rules, and industry awareness regarding trends and competition through literature, conferences and stakeholder interactions.
  • Serve as scientific point of reference, ensuring that assigned Translational Medicine programs are firmly grounded in the latest research, and that internal and external communications are consistent with this standard.


  • Proven ability to thrive in a fast-paced, innovative environment while remaining flexible, proactive, resourceful and efficient.
  • Ability to excel in a multidisciplinary environment as an integral leader and team member across several project teams.
  • Ability to analyze complex issues to develop relevant and realistic plans, programs and recommendations.
  • Medical degree and 2-5 years of translational or clinical research experience are required.
  • Background and interest in relevant science and technology, as well bio/pharmaceutical industry or IND experience are preferred.
  • Knowledge of neurology or pediatric neurology, and/or rare diseases is strongly preferred.
  • Demonstrated excellence in translational, clinical research, drug development,
    and/or regulatory issues are desirable.
  • True care and passion for improving the lives of people living with rare diseases.
  • Diplomacy and professionalism, able to command respect from peers and subordinates alike, capable of highly-independent work as well as being a team player and role model.
  • Excellent verbal and written communication skills, allowing for an open and effective dialogue throughout the company, and for effectively representing the company externally.
  • Strong problem-solving ability, and attention to timelines, detail and quality are critical to success.
  • Highly motivated and results-oriented individual with the flexibility and creativity to excel in and contribute to multiple projects simultaneously in a rapidly growing company.
  • Must have the passion and ability to forge and nurture key partnerships and collaborations with the external scientific community, including academia, governmental organizations, nonprofit organizations and CROs.

Travel is estimated to be 30%, most being domestic and some international.

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.