Ultra-Rare Disease Program Director- Gene Therapy (Program Management, discovery through translation

San Diego, CA, US
Nov 21, 2020
Biotech Beach
Required Education
Masters Degree/MBA
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.

The Ultra-Rare Disease Program Director (PD) has a key organizational role to partner with the Ultra-Rare Disease Translational Program Head (TPH) and drive the ongoing alignment / refinement and execution of an integrated strategy and development plan for global gene therapy drug development programs for ultra-rare diseases, ensuring organizational alignment and seamless cross-functional execution. The Ultra-Rare Disease PD is responsible for all Program Management deliverables and is the single representative from Program Management for "Project Leap". The Ultra-Rare Disease PD manages development deliverables for all ultra-rare disease gene therapy program(s), across the asset lifecycle from discovery through translational stages, registration, and lifecycle management.

The Ultra-Rare Disease PD has excellent drug development knowledge and provides matrix and situational leadership to a multi-disciplinary team of functional experts on the ultra-rare disease team to deliver with high quality, and in accordance with approved strategy. The Ultra-Rare Disease PD may deputize for the Ultra-Rare Disease Translational Program Head as leader and spokesperson for "Project Leap" at AveXis / Novartis internal meetings.

  • As operational leader for the program, this role is specifically responsible to:
    • Influence cross-functional strategy for development of ultra-rare disease therapies to ensure innovation, productivity, and feasibility.
    • Proactively identify and manage program development risks and issues.
    • Build a deep understanding of challenges and opportunities unique to ultra-rare diseases and translate into an agile development and commercialization model to drive a cost-sustainable, impactful and enduring business model.
    • Champion enterprise thinking when planning resources and budget.
    • Generate and maintain a high-quality and accurate project plan for ultra-rare diseases and forecast in the enterprise planning systems (e.g., Horizon).
    • Ensure delivery of development activities to forecast, timelines and with high quality.
    • Ensure effective dynamics, performance, prioritization and communication in the ultra-rare disease team.
    • Regularly contributes to or occasionally leads Program Management-driven or initiated organizational improvement initiatives.
  • Programs
    • In partnership with the Ultra-Rare Disease Translational Program Head, drives ongoing evolution and refinement of ultra-rare disease program strategy and integrated development plan and ensures consistency of both the program strategy and the development plan with the target product profile.
    • Responsible for all Program Management documentation associated with the program (e.g., minutes, Target Product Profile).
    • Ensure quality of the integrated development plan, reflecting best innovative drug research and development practices, realistic planning, cross-functional alignment and ensuring consistency with individual functional plans.
    • Drives and/or coordinates development of strategic documents and prepares for phase transitions/tollgates in collaboration with the ultra-rare disease team.
    • Drives risk assessment and contingency planning by the ultra-rare disease team.
    • Communicates program status and issues horizontally and vertically in a transparent and timely manner.
    • Supports the Ultra-Rare Disease Translational Program Head in driving preparation of comprehensive program recommendations, board meeting presentations and updates for governance boards.
    • Leads ad-hoc cross-functional sub-teams and task forces to address and resolve issues.
    • May deputize for the Ultra-Rare Disease Translational Program Head as leader and spokesperson for the ultra-rare disease team and may represent the program at board meetings.
    • May lead a subpart of a program, e.g. secondary indication, pediatric program, new delivery forms or deputize for the Ultra-Rare Disease Translational Program Head at ultra-rare disease team meetings.
  • Operations
    • Organizes ultra-rare disease team meetings and prepares high team agendas and minutes in a timely manner.
    • Along with Finance, assembles and monitors financial forecasts and communicates variances/deviations to the ultra-rare disease team.
    • Responsible for program/project/financial information provided to governance boards, portfolio management, Finance, and other parties, and responsible for executive communication of program status through team minutes and as otherwise required by the organization.
    • Leads generation of complete and accurate ultra-rare disease program plans and forecasts in the enterprise planning system (e.g., Horizon). This includes liaising with partner functions to ensure a realistic plan that reflects the strategy and challenging the schedules and financial forecasts provided by the functions to ensure feasibility.
    • Collaborates with partner functions to maintain realistic ultra-rare disease program plans reflecting latest team decisions.
    • Ensures execution of cross-functional project activities and tracks progress versus integrated development plan (timelines, forecast).
    • May manage external partnership/collaborations in conjunction with the Ultra-Rare Disease Translational Program Head and other functions, such as Business Development and Research.
  • Teams
    • Partners with the Ultra-Rare Disease Translational Program Head to enable a high performing team culture based on the AveXis / Novartis values and behaviors, the expertise and contributions of the ultra-rare disease team members, shared responsibility, and the coordination of work towards a common goal.
    • Drives ultra-rare disease team objective setting, prioritization and tracking, and definition and achievement of realistic program goals with an enterprise mindset by integrating and balancing both portfolio and line function perspectives.
    • Builds trusting relationships with ultra-rare disease team members.
    • Collaborates with ultra-rare disease team members to identify key program issues and brings to the full ultra-rare disease team for deliberation and decision on resolution.
    • Resolves conflicts in good faith.
    • Coaches ultra-rare disease team members, as needed, e.g., for optimal program progression.
  • Leadership
    • Contributes drug development knowledge and project management experience to team discussions.
    • Coaches others on drug development and project management topics.
    • Provides program management expertise and leadership in the areas of planning, tracking, scenario-generation, contingency development, critical path analysis, and risk management.
    • Actively identifies and develops best practices for project management and implements accordingly.
    • Keeps up to date and acts as strong supporter of organizational change; champions implementation of changes within ultra-rare disease teams.
    • Participates in initiatives to identify, develop and implement process changes within and outside of Program Management.

  • Masters or Doctorate in life sciences (or MBA with bachelor's degree, or equivalent experience in life science).
  • 10+ years pharmaceutical industry experience.
  • 5+ years of equivalent multi-/cross-functional leadership experience.
  • Previous track record of success in working with and leading large scale and complex international and multidisciplinary drug development teams.
  • Expert knowledge in drug development (including early and late stage development).
  • Excellent project / program management skills.
  • Advanced knowledge and experience in pharma business development is a plus.
  • Advanced knowledge in international customs and business practice is a plus.
  • Expert in communication and information management.
  • Team facilitation skills that can help with objective setting for the team and the collective achievement of the objectives.
  • Leadership presence with the ability to present and interact with senior management.
  • Strength demonstrated in strategic decision making, scenario evaluation and contingency planning.
  • Strong interpersonal and communication skills for bridging between scientific and business participants, for driving a program to effective implementation and for managing effective international collaborations; strong verbal communication, expert in written communications, expert team player.
  • Advanced planning and tracking skills, ability to use proper tools in program management and capable of managing multiple programs with respect to priorities.

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