Principal Scientist, Research and Development

AveXis Inc.
San Diego, CA, US
Dec 04, 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.

AveXis is seeking a Principal Scientist to execute in vitro experiments for AveXis programs. This individual will work in the laboratory, responsible for execution of experiments related to existing and pipeline products. This individual will work with scientists in the Research and Development department and report to the Director of Research and Development.


  • Cell culture experiments supporting existing and pipeline programs.
  • Molecular biology assays including DNA isolation, PCR, ddPCR.
  • Histology, including use of microtome and cryostat.
  • In vivo studies of neurological disease.
  • Drive the execution of approved research strategies.
  • Follow standard operating procedures (SOP).
  • Maintain records to comply with regulatory requirements.


  • PhD or Master's degree in Biology, Neuroscience, Stem cell biology, or related field will be considered; 5+ years industry and/or post-doctoral experience.
  • Laboratory experience in a molecular or cell biology laboratory
    • Publications and/or demonstrable experience desired
  • Specific laboratory experience desired includes, but is not limited to:
    • Culture of mammalian cells in cell culture
    • Molecular biology, including DNA isolation, PCR assay design and execution, restriction digest, cloning
    • Droplet digital PCR (ddPCR)
    • Isolation of cells from animal tissue
    • In vivo dosing and animal models of neurological disease
    • Histology and microscopy

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.