Scientist II, Platform Development

San Diego, CA, US
Sep 23, 2020
Biotech Beach
Required Education
Bachelors Degree
Position Type
Full time

Ready to join a team committed to developing and commercializing gene therapies for patients and families devastated by rare neurological genetic diseases? Since 2013, Novartis Gene Therapies (formerly AveXis) has had one focus: bringing change to those devastated by genetic diseases.

The Novartis Gene Therapies culture embraces this mission. As a compassionate and dedicated team, we are enthusiastic about the science behind our work and finding answers to difficult questions. We are dedicated to communities affected by rare diseases, and these patients and families are the motivation for everything we do.

We have built a team with exceptional depth of experience, unified by a common vision; to develop gene therapies with the potential to positively impact the lives of the patients and families devastated by rare and life-threatening neurological genetic diseases. Though we are proud of what we have achieved to date, we remain relentlessly focused on making that vision a reality.

The Scientist II, Platform Development, contributes to AAV technology advancement, evaluation and implementation to enable safe and efficacious gene therapy strategies for Novartis Gene Therapies portfolio programs.

  • Contribute to design, planning and execution of non-GLP animal studies (rodent and non-rodent) to assess efficacy, stability, PK/PD and safety/toxicology of novel AAV gene therapies.
  • Implement experimental approaches to investigate innate and adaptive immune responses and cell stress and toxicities in a tissue-specific manner. Evaluate novel immunomodulation and AAV efficacy/safety improvement strategies in in vitro and in vivo studies.
  • Conceive and support strategies to optimize potency, efficacy and safety of gene therapy vectors. Establish/execute primary/iPS 2D/3D cell models for protein/RNA- and activity-based AAV function assessment.
  • Coordinate and execute studies in collaboration with other functional groups (e.g. histology/pathology, molecular biology) within GTx and with external CROs and academic investigators.
  • Leads and directs the activities supporting platform characterization and development.
  • Designs and executes experiments to address vector host interactions including routes of infection and immunology.
  • Presents experimental design and results to internal senior management and incorporate feedback.
  • Leverage internal functional teams to plan, coordinate, and monitor the timely execution and completion of various research projects.
  • Execute experiments via hands on work or leveraging internal capabilities, Novartis capabilities, CRO work or academic collaboration.
  • Identify and direct research personnel to execute experiments within the Platform Development group.
  • Oversee the maintenance of accurate, comprehensible, and current laboratory notebooks.
  • Presentation of data at external scientific presentation at conferences and meetings.
  • Timely provision of data and documentation in reports to support intellectual property filings and regulatory submissions.
  • Support regulatory submissions and interactions with the FDA around new products.
  • Other related job duties as assigned.

  • Bachelor's, in biomedical scientific area, with 6 years' experience including gene therapy, neurobiology, immunology, virology, cell biology, biochemistry, and/or synthetic biology. or Master's with 4 or PHD with 2.
  • Experience with conducting studies in laboratory animal models, handling and processing tissues for downstream histological and biochemical analysis.
  • Experience in general and advanced wet-lab methods such as molecular cloning, PCR, qPCR/ddPCR, 2D/3D primary and iPS cell culture and differentiation, IF/confocal microscopy, MSD/ELISA/other protein assays, plate-based reporter assays, western/bot/southern blotting, gene expression assays, immunological assays (e.g. flow cytometry, ELISPOT), NGS sample/library prep methods, genome analysis assays.
  • Proven record of ability to be adaptable, comfortable and productive in a dynamic, fast-paced and cross functional team-based environment.
  • Self-motivated to conceive and contribute to planning of studies, and to implement new experimental strategies and techniques.
  • Ability to organize, multi-task and work independently.
  • Ability to follow good research laboratory and documentation practices, and to comply with all company policies.
  • Advanced oral, presentation and written communication skills.

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.

Novartis Gene Therapies 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.