Associate Director, Research and Development

AveXis Inc.
San Diego, CA, US
Nov 30, 2018
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. 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 Associate Director will be responsible for generating reports to update senior management on all aspects of the research and development function, while maintaining appropriate documentation of all studies. The position will serve as a knowledge resource to the company on the latest gene therapy research and a subject matter expert on the biology of neuromuscular diseases. The successful candidate will take accountability to ensure all projects requiring research and development resources are adequately resourced and tracking to timelines.

The Associate Director will also be responsible for implementing the research program for AveXis, particularly for future programs, and guidance for the team in research and development.


  • Facilitate research required for future AveXis gene therapy programs.
  • Provide feedback and subject matter expertise on any new product or manufacturing process.
  • Participate in the hiring and training of key research and development personnel.
  • Provide hands on technical leadership to the research staff members.
  • Support Vice President, Research and Development to meet information requirements as needed for quality, compliance, and management reporting.
  • Manage the overall day-to-day research and development activities in the laboratory and assist with the creation and on-going maintenance of all pertinent equipment, policies, and procedures.


  • Minimum PhD degree in neuroscience, molecular biology or related field.
  • Record of productive post-doctoral experience in neuroscience.
  • Publication record of high impact research in gene therapy of neuromuscular disease
  • Demonstrated technical ability in molecular, cellular, and animal models of neuromuscular disease
  • 3+ years of supervisory experience with demonstrated effectiveness in providing input, guidance, mentorship and support to staff.
  • Ability to motivate peers and staff, foster a culture of intellectual curiosity and timely delivery of critical results.
  • Experience with 3 rd party outsourcing of research and development.
  • Demonstrated ability to partner and collaborate with academic and industry scientists.
  • Recognized as a thought leader in spinal muscular atrophy.
  • Excellent oral and written communication skills. Strong technical writing ability required.

Approximately 25% travel required

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.