Senior Director, Translational Medicine, Ultra-Rare Diseases

Bannockburn, IL, US
Aug 24, 2020
Science/R&D, Pathology
Required Education
Position Type
Full time

Ready to join a team committed to developing and commercializing gene therapies for patients and families devastated by rare neurological genetic diseases? AveXis is advancing cutting-edge science, starting with our proprietary gene therapy for the treatment of spinal muscular atrophy (SMA). We are in the midst of an incredible journey and are looking for passionate individuals to join us on this important mission.

AveXis, a Novartis company, is dedicated to developing and commercializing novel treatments for patients suffering from rare and life-threatening neurological genetic diseases. Our initial product is a proprietary gene therapy approved by the US Food and Drug Administration for the treatment of pediatric patients with SMA. In addition to developing a treatment for SMA, AveXis also plans to develop other novel treatments for rare neurological diseases, including Rett syndrome and a genetic form of amyotrophic lateral sclerosis caused by mutations in the superoxide dismutase 1 (SOD1) gene.

AveXis is working to extend the impact of its gene therapy platform to develop treatments that target ultra-rare diseases (e.g. those with patent populations < 1 / 1,000,000) - via a new "Project Leap." Ultra-rare diseases have long been underserved by the pharmaceutical industry not only because their small patient populations challenge commercial viability, but also due to biological, technical, and operational complexity. Gene therapies presents an opportunity to address these technical challenges for certain diseases, and to bring hope to patients and their families.

AveXis is seeking a Senior Director, Translational Medicine (TM) who will be responsible for the successful translation of gene therapy products for ultra-rare diseases from the pre-clinical proof of concept stage in R&D to first-in human trials. As new indications are considered for the ultra-rare disease pipeline, the Senior Director, TM will conduct diligence together with colleagues in Research and Business Development. The Senior Director will develop the translational plan ensuring trial readiness with regards to evaluation of natural history datasets, 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.

  • Contribute to the scientific evaluation of new indications or opportunities to advance the ultra-rare disease franchise.
  • Lead 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 treatments.
  • Optimize trial readiness including evaluation of natural history datasets, registries, clinical outcomes, biomarkers and pathways to rapid genetic diagnosis.
  • Coordinate the design of first-in-human studies, considering innovative designs appropriate for ultra-rare diseases, and support AveXis in building shared approaches across ultra-rare disease programs for an increasingly efficient platform.
  • Ensure that translational medicine contributions to regulatory documents including FDA meetings and IND submissions are planned and executed in a timely manner.
  • Work in cross-functional teams and serve as the primary Translational Medicine representative to teams in the research, clinical development, clinical operations, regulatory, CMC, project management, commercial, and business development functions.
  • Contribute to the development of the overall project vision and strategy for the ultra-rare disease program.
  • Serve as a leader to represent the ultra-rare disease program together with the Translational Program Head at internal and external meetings.
  • Maintain scientific awareness regarding advances in the ultra-rare disease space, indications of interest, regulatory awareness regarding new guidance and rules, and industry awareness regarding trends and competition through literature, conferences and stakeholder interactions.

  • Advanced degree (Doctorate, or similar degree) in Life Sciences, Biology, or similar discipline, or commensurate experience.
  • 7 years Translational or clinical research experience is required with drug development experience in gene therapy preferred.
  • Expertise in neurology or neuromuscular diseases is required with experience in ultra-rare diseases preferred.
  • Demonstrated experience in communicating and working together collaboratively with key stakeholders in the disease community such as clinicians, researchers, patients/families, and patient advocacy organizations.
  • Proven ability to thrive in a fast-paced, multidisciplinary environment while remaining flexible, creative, proactive, resourceful and efficient.
  • Ability to analyze complex issues to develop relevant and realistic plans, programs and recommendations.
  • Excellent understanding of translational medicine, drug development and regulatory issues is desirable.
  • Demonstrated excellence in coordination of translational and/or clinical research projects.
  • True care and passion for improving the lives of people living with ultra-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.

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.