Principal Investigator, Ultra-Rare Disease Program Lead, R&D

San Diego, CA, US
Nov 21, 2020
Science/R&D, Pathology
Biotech Beach
Required Education
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.

AveXis is working to extend the impact of its gene therapy platform to develop treatments that target ultra-rare diseases (e.g. those with patent populations < 1 / 1,000,000) - via a new "Project Leap." Ultra-rare diseases have long been underserved by the pharmaceutical industry not only because their small patient populations challenge commercial viability, but also due to biological, technical, and operational complexity. Gene therapies present an opportunity to address these technical challenges for certain diseases, and to bring hope to patients and their families.

AveXis is seeking a highly motivated and talented Principle Investigator who will be responsible for the successful pre-clinical development of gene therapy products for ultra-rare diseases. As new indications are considered for the ultra-rare disease pipeline, the Principle Investigator will help conduct diligence together with colleagues in Translational Medicine and Business Development. Candidates should have a successful track record of leading multi-functional teams to drive product development. The Principle Investigator will report to the Vice President, Ultra-Rare Disease Translational Program Head and work closely with Translational Medicine and Program Management to align research activities with timelines.

  • Oversee the pre-clinical aspects of up to 3 ultra-rare disease IND programs from gene to FIH studies.
  • Contribute to the development, internal review and approval of the investigator's brochure for ultra-rare disease programs.
  • Primary responsibility for preparation of the IND pre-clinical section.
  • Scientific leadership in support of translational research activities to generate gene therapies for ultra-rare diseases.
  • Responsible for overseeing research, experimental design, and efficient implementation in cooperation with functional heads.
  • Works with ultra-rare Program Director to coordinate timelines for ultra-rare disease projects and track progress of Research activities.
  • Identifies risks, potential bottlenecks or delays and proposes options to overcome these.
  • Manages 3rd party contracts and services aligned with ultra-rare disease program objectives (CRO, CLIA Labs, etc.).
  • Participate in academic outreach and partnership to support existing and potential new ultra-rare disease programs and aid in the company's strategic decision making for new areas of research.
  • Presentation of data and strategy to the internal senior management team and external scientific presentation at conferences and meetings.
  • Timely provision of data and contribution to the preparation of documents to support intellectual property filings and regulatory submissions.
  • Support regulatory submissions and interactions with FDA around new ultra-rare disease products.
  • Serve as a leader to represent the ultra-rare disease program together with the Translational Program Head and Translational Medicine Senior Director at internal and external meetings.
  • Maintain scientific awareness regarding advances in the ultra-rare disease space, indications of interest, regulatory awareness regarding new guidance and rules, and industry awareness regarding trends and competition through literature, conferences and stakeholder interactions.

  • MD, PhD, or equivalent advanced biology degree.
  • Experienced scientist with 5+ years' experience in the drug development process and 2+ years' people management experience.
  • Expertise in neurology or neuromuscular diseases is required with experience in ultra-rare diseases preferred.
  • Experience with gene therapy preferred.
  • Strong proficiency in fundamental aspects of drug development and demonstrated ability to advance pipeline programs as required.
  • True care and passion for improving the lives of people living with ultra-rare diseases.
  • Excellent oral and written communication skills across all levels within and external to the organization.
  • Strong problem-solving ability, and attention to timelines, detail and quality are critical to success.

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.