Vice President, Ultra-Rare Disease Translational Program Head

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 working to extend the impact of its gene therapy platform to develop treatments that target ultra-rare diseases (e.g. those with patent populations

The Ultra-Rare Translational Program Head is responsible for designing and implementing a new "virtual franchise" dedicated to ultra-rare diseases, including leading activities related to disease selection, licensing of programs, engaging patient advocates, and implementing strategy. This individual also has final accountability for the innovation, strategy, operations, and related development deliverables for all ultra-rare disease gene therapy program(s), across the discovery and translational stages of the asset lifecycle.

The core responsibilities are to: Set / advance ultra-rare disease strategic vision; Define an agile discovery, preclinical and clinical development model that addresses the unique challenges and opportunities of the ultra-rare disease environment; and Drive program development by leading a multidisciplinary team of functional experts (some with dedicated, direct line reporting), focused on driving toward key regulatory submissions, optimized market access, and financial sustainability of the program(s).

Responsibilities

Establish short and long-range project vision and strategy

• Establish ultra-rare disease program strategy and short- and long-term priorities.
• Identify, prioritize, and select ultra-rare diseases for development according to the strategic vision, collaborating with Research & Early Development, Translational Medicine and Business Development.
• Stay informed on the global landscape of ultra-rare disease and gene therapy research and development to anticipate and respond to opportunities and risks.

Define an agile development model tailored to ultra-rare diseases

• Build a deep understanding of challenges and opportunities unique to ultra-rare diseases across the development and commercialization lifecycle.
• Translate unique complexities of ultra-rare diseases to an agile development and commercialization model to drive a cost-sustainable, impactful and enduring business model.
• Proactively shape the external environment to advance AveXis' position in ultra-rare diseases by developing strong, evidence and trust-based relationships with key stakeholders (e.g., regulators, KOLs, patient groups, scientific societies).
• Continuously assess strengths and weaknesses of the ultra-rare development model and team to improve on the model as needed.

Drive project operations to achieve superior performance

• Communicate clear strategy to program team and functions, and ensure that strategy is reflected in the operations.
• Set, enforce, and measure realistic goals for project operations.
• Ensure rigorous adherence to quality and compliance.
• Maintain P&L for ultra-rare disease program, striving for cost sustainability.
• Drive talent acquisition among team members and within their sub-teams including active selection and de-selection and performance management of the core membership of the program team.
• Partner with other functional heads across AveXis to ensure sufficient resource allocation to support the ultra-rare development model.

Contribute to the franchise and wider development organization

• Present at key management boards, gathering best practices from other teams, and contributing experience as needed to the rest of the portfolio.
• Translate development and commercialization insights from ultra-rare programs into implications for AveXis' broader portfolio.
• Actively participate in mentoring and talent management initiatives.

Qualifications

• Doctoral or Medical degree or equivalent.
• Minimum 5 years of drug development experience, ideally including experience in program teams and with dossier submission for new or supplemental indications.
• Leadership of multidisciplinary teams.
• Established networks in rare or ultra-rare diseases is a plus.
• Competent in understanding of drug development (ideally for rare or ultra-rare diseases) including key issues for compounds or programs as they transition through Development (e.g., TS / early development, clinical trials, safety, PK, biomarkers, diagnostics, data management, drug substance / development, marketing and sales).
• Demonstrated knowledge of operational issues (e.g., portfolio management, planning global studies, and risk management).
• Competent in GCP, and pharmacovigilance practice.
• Demonstrated competence in understanding of commercial and market access issues and collaborates closely with these groups, especially in commercializing rare or ultra-rare diseases.
• Has experience with health authority interactions, especially for rare or ultra-rare diseases (e.g., regulation / guidelines, HA meetings, safety reviews, registration dossier, submissions).
• Demonstrates the courage to assume personal accountability in challenging situations
• Critical thinker - able to develop and oversee implementation of a global strategy for the product. Unites formerly unrelated concepts to arrive at innovations, and calculates their complex potentials in the evolving global marketplace.
• Excellent communication skills essential, both oral and written; able to present to Health authorities. Presents information confidently and effectively to executives, peers, and direct reports, one-on-one or in groups.
• Strong leadership skills are essential.

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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