Scientist II, Process Science (Upstream)

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.

The Scientist will be an integral part of a fast paced and rapidly growing group of scientists and engineers developing processes for the manufacture of our proprietary adeno-associated virus (AAV) based gene therapies.

The candidate will responsible for contributing to the development and improvement of the AAV upstream manufacturing platform. This individual will lead and support activity with cross-functional organizations to plan, execute, and document experiments that define the process.

Candidate primary responsibility will be the design and execution of time-sensitive experiments, studies, and capturing related data and knowledge, to advance the development of Avexis products from Research to GMP manufacturing. The candidates should have extensive molecular experience to enhance virus production from mammalian expression systems. The individual will have an organized and systematic approach to viral production, as well as a track record of collaborative relations with groups such as discovery research, analytical development, and pilot scale operations. Ability to multi-task and meet tight timelines is essential.

Responsibilities

• Design and apply DOE and QbD studies to generate, optimize and characterize expression systems for vector production.
• Conduct laboratory studies to optimize AAV platforms such as cell line development, plasmid construction, subcloning, media development, bioreactor optimization and scale-up etc.
• Ability to effectively and efficiently analyze and interpret data to further progress development strategies.
• Write detailed experimental protocols, develop Bill of Materials (BOM), execut and documente experimental studies according to Standard Operating Procedures (SOPs) or established practices, review and report data.
• Author technical reports of studies in support of Process Development.
• Support initiatives for new technology development and continuous improvement projects
• Maintain constant awareness of novel molecular and cell culture technologies for AAV production and characterization. Utilize literatures and external resources to support internal studies.

Qualifications

• Minimum B.S. degree in biochemistry, molecular biology, chemical engineering, bioengineering, or related technical field, Master's degree or PhD preferred.
• Extensive experience on cell line development, including knock-out/knock-in, transient and stable expression, especially with in-depth knowledge of CRISPR/Cas9, TALEN, transposons, etc. gene editing technology.
• Broad and extensive knowledge of molecular biology, biochemistry and cell biology assays, including but not limited to: molecular cloning, cell culture, DNA/RNA transfection, virus production & infection, flow cytometry, immunoblot, qPCR, protein purification, enzyme activity and kinetics etc.
• Proven track record of vector biology, codon optimization, and cell line genetic characterization.
• Proven expertise with a variety of cell culture and bioreactor technologies.
• Should be highly motivated, have excellent organizational and communication skills, and be able to multitask in a fast-paced environment with changing priorities.
• Must demonstrate a keen knowledge of experimental design, underlying scientific principles, and the ability to analyze and assess results
• Demonstrated ability to work effectively in a cross-functional team.
• Excellent oral and written communication skills.
• Approximately 10% to 15% 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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