Senior Scientist, Potency Assay

Overview

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 seeking a highly motivated and capable Senior Scientist to join our Research and Early Development team. The position entails leading activities in the Cell Potency Assay group, including developing and mentoring the team and supporting the advancement of research projects. The incumbent will work with Scientists across the Research and Development team, as well as external teams and report to the function head of the Research Potency Assay Team.

Responsibilities

• Leverage strong understanding and knowledge of gene expression and mechanism of action (MOA) for cell-based potency assay development.
• Design and execute experiments to identify and optimize key parameters for cell potency assays.
• Lead activities within the functional group for pipeline program research & development from both the bench and the desk.
• Coordinate group efforts and oversee ongoing projects to facilitate teamwork.
• Develop team members, ensuring staff receive mentoring and constructive feedback in order to develop in the role and learn new capabilities.
• Partner with project leaders to plan, coordinate, and monitor the timely execution and completion of various research projects.
• Oversee the maintenance of accurate, comprehensible, and current laboratory notebooks.
• Present data to the internal senior management team and external collaborators.
• Timely provision of data and documentation in reports to support intellectual property filings, regulatory submissions and interactions with FDA around new products.

Qualifications

• Bachelor's in scientific area (biology, cell biology, pharmacology or related) with 8 years' experience or Master's with 6 or PHD with 4.
• Expert knowledge of scientific principles and concepts.
• Extensive laboratory and working knowledge of cell-based assays for potency (expression and MOA).
• Publications and demonstrable experience desired.
• Background in neuroscience.
• Specific laboratory experience desired includes, but is not limited to:
  • Knowledge of viral gene therapy (adeno associated viral vectors).
  • Cell line development and characterization, cell culture and cell banking activities.
  • Genome editing, molecular cloning & DNA delivery methods.
  • Reporter and protein expression assays.
  • Human IPSC-derived neurons, fibroblasts and murine primary cell culture.
  • Biotechnology methods: High content imaging, Western blot, RT-qPCR, enzymatic assays, CRISPR, dsiRNA, ICC, ELISA, MSD, SIMOA, flow cytometry, NGS, RNA-seq, 10x.
• 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.

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