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Senior Scientist, Research (Potency Assay)

Company: AveXis Inc.
Location: San Diego
Posted on: September 15, 2019

Job Description:

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 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. Responsibilities Demonstrates innovative design, development, and execution of pipeline projects through literature review and scientific teamwork. Leverages strong understanding of cell-based assays, from early development through qualification and validation. Cell line development, cell culture, and cell banking Develop cell-based reporter and potency assays Support assay development/optimization and other proof of concept studies Establish technology platforms (e.g. western blot, ddPCR, enzymatic assays) and serve as SME for these platforms. Develop assays and build testing capacity as needed. Molecular cloning, protein expression and detection Mammalian expression systems to support protein expression studies Cell line generation by transient/stable transfection or viral transduction May manage associates in either assay development or assay execution. Reviews data and supports troubleshooting. Leads both from the bench (hands-on laboratory work) and from the desk (designing studies, analyzing data, preparing reports and documents). Readily adapt to changing project needs; independently plan and execute experiments. Leads and supports research initiatives such as new technology development and continuous improvement projects. Adheres to good laboratory conduct, documentation practices, and safety guidelines. Qualifications PhD degree in biology, cell biology, pharmaceutical sciences, or related field with 2+ years biopharmaceutical experience. Extensive working knowledge of cell-based assays for potency. Strong background in assay qualification/validation, with 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, colorimetric assays, enzymatic assays and 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. #LI-EC1

Keywords: AveXis Inc., San Diego , Senior Scientist, Research (Potency Assay), Other , San Diego, California

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