Principal Investigator, Program Lead Research and Development

Overview

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 seeking a highly motivated and talented Program Leader with a successful track record of leading multi-functional teams to drive product development. This candidate will report to the Directors of Research and Development and work closely with Translational Medicine and Program Management to align research activities with timelines.

Responsibilities

• Oversee the pre-clinical aspects of up to 2 IND programs from gene to FIH studies.
• Contribute to the development, internal review and approval of the investigator's brochure.
• Primary responsibility for preparation of IND pre-clinical section.
• Scientific leadership in support of translational research activities to generate gene therapies.
• Will serve as the face of the program, including being the subject matter expert.
• Responsible for overseeing research, experimental design, and efficient implementation in cooperation with functional heads.
• Coordinates timelines for projects; tracks progress of cross-functional core team activities.
• Identifies risks, potential bottlenecks or delays and proposes options to overcome these.
• Manages 3rd party contracts and services aligned with program objectives (CRO, CLIA Labs, etc.).
• Participate in academic outreach and partnership to support existing and potential new 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 products.

Qualifications

• MD, PhD, or equivalent advanced biology degree.
• Experienced scientist with 5+ years' experience in the drug development process and 2+ years' people management experience.
• Strong proficiency in fundamental aspects of drug development and demonstrated ability to advance pipeline programs as required.
• Excellent oral and written communication skills across all levels within and external to the organization.
• Experience with gene therapy preferred.
• Experience with CNS disorders preferred.

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.

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