Director, Preclinical Development (Veterinary Pathologist) - Gene Therapy

San Diego, CA, US
Aug 24, 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? 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 Director of Preclinical Development will be a member of the Research and Early Development (R&ED) leadership team and is a key leadership role in AveXis Research. The incumbent will report to the CSO and play a strategic role in the nonclinical and translational research strategy for this compelling new therapeutic modality. We are still shaping the scientific and regulatory approach to evaluation of gene therapies and the incumbent will have the opportunity to be influential in shaping the field. This role will liaise extensively with collaborators and partners across Novartis to build and execute a world leading AAV gene therapy pipeline. Intimate collaboration with Novartis line functions will be required. The pipeline therapeutic focus will be mainly in rare, monogenic neurological and muscle disorders. The incumbent will be a visible and vocal spokesperson in shaping overall R&ED strategy as well as Corporate and Business Development Strategies.

  • Member of the R&ED Leadership Team.
  • Partner and collaborate with NVS Preclinical Safety (PCS) and Pharmacokinetic Sciences (PKS) project team members and functional units to ensure well designed and executed preclinical development programs for each pipeline program.
  • Responsibility to oversee final design, protocol, execution, monitoring, and reporting of non-GLP non-rodent pharmacology, biodistribution, PK/PD and dose range finding toxicology studies with AAV gene therapies. Partnering with many collaborators and stakeholders in design and execution of these complex studies.
  • May act as the functional manager of the histology/pathology laboratories.
  • Key contributor to regulatory document creation and review.
  • Design and execute safety and tolerability aspects of translational research programs to support clinical stage programs.
  • Develop and maintain world leading expertise and recognition in AAV gene therapy as an emerging therapeutic modality.
  • Work closely with Business Development (BDL) and due diligence teams to evaluate and make strategic recommendations on external assets (technologies and programs).
  • Advance the concepts and practice of Good Research Practices and a quality culture within AveXis Research.

  • DVM Required
  • Specific expertise training and/or board certification in veterinary pathology preferred.
  • Experience as a study director, principle investigator and/or study monitor for non-rodent pharmacology/toxicology studies.
  • 5+ year's biotech/pharma industry research experience.
  • Outstanding collaboration skills required, as this is an extensive matrix collaboration role.
  • Advanced written and oral communication skills.
  • Experience working in cross-functional teams.

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.