Scientist, Process Science - Upstream

AveXis Inc.
San Diego, CA, US
Apr 16, 2019
Biotech Beach
Required Education
Bachelors Degree
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 (ZOLGENSMA®). 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.

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

The Scientist is responsible for contributing to the development and improvement of the upstream manufacturing platform processes used to manufacture AveXis gene therapy products. This individual will lead and support activity with cross-functional organizations to plan, execute, and document experiments and early manufacturing that define the process, and method of delivery to the clinical and commercial sites. The Scientist will provide support for process, analytical, and characterization knowledge related to the production of Avexis gene therapy products, and the raw materials needed to make them.

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 Development to GMP manufacturing. The successful candidate will take responsibility to ensure all documentation and reports are accurate, complete, and suitable for using in support of production, characterization, and regulatory filings. The candidates should have extensive experience with production of virus or biologics from mammalian expression systems. Hands on experience with different cell lines (adherent and suspension), bioreactors, and scale-down model is required. The individual will have an organized and systematic approach to viral or biologic production, as well as a track record of collaborative relations with groups such as research & development, analytical development, and pilot scale operations.


  • Design and apply DOE and QbD studies to develop, refine, optimize and characterize cell culture and vector production processes. Execute experiments and troubleshoot process and equipment as needed.
  • Conduct laboratory studies to enhance AAV manufacturing technologies, capabilities and processes (such as media development, bioreactor fed-batch and perfusion process development, optimization and scale-up).
  • 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.
  • Create and revise SOPs for equipment and process operations as needed.
  • Lead the authoring of technical reports and CMC sections for regulatory filings.
  • Support initiatives for new technology development and continuous improvement projects.
  • Maintain constant awareness of novel biochemical and biophysical technologies for AAV production and characterization. Keep up to date with the scientific literature and developments in the field.
  • Applies understanding of regulatory expectations to process development strategies. Contributes to process risk assessments. Justifies development strategies and experiment designs.


  • Minimum B.S. degree in biochemistry, chemical engineering, bioengineering, or related technical field; Master's degree or PhD preferred.
  • 6-10 years of experience in biopharmaceutical based GMP manufacturing operations including direct experience in cell culture.
  • Proven expertise with a variety of cell culture and bioreactor technologies. Experience with development of cell culture systems for the production of viral vectors or vaccines is preferred.
  • Experience in cellular metabolism and metabolic flux analysis is a plus.
  • 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.
  • Ability to multi-task and meet tight timelines is essential.
  • 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.