Scientist II, Potency Assay Development

San Diego, CA, US
Aug 24, 2020
Biotech Beach
Required Education
Associate Degree
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 (RTT), a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene and Friedreich's Ataxia (FA).

AveXis is seeking a highly motivated and capable scientist to join our Research and Early Development team. The position focuses on design, development, optimization, troubleshooting, and execution of cell-based assays supporting AveXis programs. The incumbent will work with Scientists across the Research and Development team and report to the function head of the Research Potency Assay Team.

  • Demonstrate innovative design, development and execution of pipeline projects and other proof of concept studies, through literature review and scientific teamwork.
  • Lead both from the bench (hands-on laboratory work) and from the desk (designing studies, analyzing data, preparing reports and documents, presenting in meetings).
  • Independently plan and execute experiments.
  • Support research initiatives such as new technology and platform development.
  • Leverage strong understanding and knowledge of gene expression and mechanism of action (MOA) for cell-based potency assay development and optimization:
    • Cell line development and characterization, cell culture and cell banking.
    • Genome editing, molecular cloning & DNA delivery methods (transduction, transfection).
    • Flow cytometry analysis and single cell sorting.
    • Protein expression and detection.
    • Cell-based reporter assays and protein expression assays by immunocytochemistry.
    • Technology platforms subject matter expert, including; Western blot, RT-qPCR, enzymatic assays, CRISPR, dsiRNA, ICC, ELISA, MSD, SIMOA, FACS, FISH, 10x.
    • Human IPSC-derived neurons, fibroblasts and murine primary cell culture.
  • May manage associates in assay development, assay execution, troubleshooting, optimization and data interpretation.
  • Adapt readily to changing project needs and support technical transfer of methods to internal groups, as needed.
  • Adhere to good laboratory conduct, documentation practices, and safety guidelines.

  • PhD degree or equivalent experience in biology, cell biology, pharmaceutical sciences, or related field with a minimum 2 years biopharmaceutical experience; 5+ years post-doc with related experience.
  • Extensive working knowledge of cell-based assays for potency (expression and MOA).
  • Strong background in neuroprogenitor cell and primary neuronal tissue culture preferred.
  • Knowledge of viral gene therapy (adeno associated viral vectors) preferred.
  • Experience with basic molecular biology and biochemistry methods: PCR, qPCR, ddPCR, DNA cloning, Southern blot and Western blot.
  • Experience with protein expression, quantification and detection by immunostaining and high content imaging.
  • Experience with quantitative and qualitative methods, including ELISA, MSD, TR-FRET. colorimetric and luminescence assays, enzymatic assays, reporter assays.
  • Advanced cell culture expertise (immortalized, primary cells, stem cells or iPS cells), including passaging, transfection, clonal selection, mass production, and cryobanking.
  • Ability to work in a fast-paced small but growing team environment and to prioritize work from multiple projects. Must be highly reliable and goals/deadlines oriented.
  • Up to 10% travel may be 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.