Principal Scientist, Analytical Development

AveXis Inc.
San Diego, CA, US
Jan 10, 2019
Biotech Beach
Required Education
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. 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.

He/she will collaborate closely with the internal R&D team to co-develop prototype bioassays and identify suitable assay formats for further optimization with demonstrated independence and with ability to make solid scientific and technical judgements. He/she will lead from the lab with hands-on experiments and data interpretation and may represent Bioassay group in cross-functional teams.

Main responsibilities also include method qualification and validation under phase-appropriate GMP as well as method transfer to internal or external QC laboratories under the guidance by his/her supervisor.

The individual will have the ability to supervise 1 or more junior members in Bioassay group to guide appropriate and effective experimental designs, execution, trouble-shooting as well as data analysis and interpretation. Responsibilities will also include supporting the director of Bioassay group on authoring tech reports, SOPs and qualification/validation protocols and reports.


  • Key driver to develop, optimize and qualify/validate MOA-reflective, bioassays suitable for lot disposition and stability study based on thorough understanding of the scientific background of the products, applicability of the emerging or existing bioassay platforms and phase-appropriate GMP requirements.
  • Independently design and execute effective experiments with clear goals of answering key questions to drive assay development and troubleshooting in a very face paced environment. Interpret obtained data for follow-up studies and decision making with guidance of his/her supervisor.
  • One of the key liaison to work closely with R&D team for knowledge and methodology learning and transfer to ensure win-win collaboration in co-developing bioassays and other functional assays suitable for characterization, stability, and release testing purpose.
  • Represent the Bioassay group to attend cross-functional CMC and project meetings if needed and actively participate in strategic plan for the projects, execution, data presentation and follow-up.
  • Authors technical reports and SOPs and assists director of Bioassay to prepare relevant sections of regulatory filings if needed.
  • Mentor and guide junior members of the group on daily laboratory work, experimental design, and data interpretation.


  • Ph.D. or M.S. in biology discipline is required.
  • 5-8 years (Ph.D) or 12-15 years (M.S) of biopharma industry experience with demonstrated leadership and track record of successfully developing different spectrum of bioassays by utilizing appropriate methodologies (i.e cell imaging systems, flow cytometry, ELISA, MSD and Luminex etc)
  • Minimum 1 year of GxP experience is preferred but not required
  • Experience in working on antibodies, vaccines, viruses or gene therapy drugs are preferred but not required
  • Good understanding of high-content imaging system and image analysis is highly preferred.
  • Technical writing skills and experience in authoring development reports and SOPs with detail-oriented trait are required.
  • Ability to work in a fast-paced team environment with highly goal-oriented approaches and to prioritize work from multiple projects with can-do attitude is required.
  • Ability to bring in and assess value-added platforms based on special needs associated with characterization of the gene therapy drug products.
  • Proven track record of solid scientific achievement to allow in-depth understanding of mechanism of action of the investigational drugs for the development of the MOA-reflective bioassays.
  • 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.
  • Excellent written and verbal communication skills.
  • Intelligent individual who respects others and is open to new/different ideas.
  • Prior supervisory experience is preferred.

This position is located out of our La Jolla area facility and may require up to 10% travel between La Jolla, third party vendors (domestic) and our HQ and Manufacturing sites located in the Chicago area.

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.