Director, Translational Medicine

San Diego, CA
Nov 12, 2018
Required Education
Masters Degree/MBA
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. 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 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. This position can be located in Bannockburn, IL or San Diego, CA.

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 that translational medicine contributions to IND submissions are planned and executed in a timely manner.
  • Work in cross-functional teams and serve as the primary Translational Medicine coordinator to teams in the clinical development, clinical operations, regulatory, CMC, project management, and business development functions.
  • Coordinate the early engagement 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.
  • Provide project management for work towards trial readiness including natural history datasets, registries, clinical outcomes, biomarkers, pathways to rapid genetic diagnosis, observational study protocols, and standard of care guidelines.
  • Coordinate the design of first-in-human studies, considering innovative designs appropriate for rare diseases, and support AveXis in building shared approaches across programs for an increasingly efficient platform.
  • Organize translational medicine projects including coordination of meetings, pre-reads, agendas, minutes, technical documents such as protocols, reports, Translational Medicine-related regulatory submissions, and requests for proposals, and contracts. Track and manage document timelines, workflows for the affiliated group and provide status updates for R&D and AveXis leadership meetings.
  • 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.


  • 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 manager and team member across several project teams.
  • Ability to analyze complex issues to develop relevant and realistic plans, programs and recommendations.
  • Advanced degree (Masters, Doctorate, or similar degree) in Life Sciences, Biology, or similar discipline, or commensurate experience.
  • 2-5 years of translational or clinical research experience or relevant program management experience is required.
  • Knowledge of neurology or rare diseases is preferred.
  • Strong understanding of translational medicine, drug development and regulatory issues is desirable.
  • Demonstrated excellence in coordination translational and/or clinical research projects.
  • Background and interest in relevant science and technology are preferred, as are bio/pharmaceutical industry or IND experience.
  • True care and passion for improving the lives of people living with rare diseases.
  • Diplomacy and professionalism, able to command respect from peers, and 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.