Associate Director, Development Program Quality Lead

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.

The Associate Director is responsible for preclinical and clinical QA activities that drive the IND engine at AveXis, for novel viral gene therapies. This person will be responsible for decision-making regarding all quality assurance topics and for collaboration with core team members to ensure quality and compliance for the product candidate(s) managed.

A successful candidate will provide critical strategic quality expertise to transition a novel gene therapy into a commercially approved product. This individual must have a strong knowledge and application of the CFRs and international regulations and have been involved in multiple regulatory inspections (familiar with multiple regulatory jurisdictions both domestically and international).

This candidate may describe themselves as passionately devoted to their patients. And, if you are the kind of person who dares to dream of something more than traditional treatments and instead wants to be a critical part of making transformational therapies a reality for patients who need it the most, then this might be the right place for you.

Responsibilities

• Provide leadership and quality expertise to project teams on the development and implementation of novel quality pathways for all aspects of the manufacturing process and method development including starting materials, plasmids, vectors and method qualification/validation for testing through the development product lifecycle (preclinical and clinical studies).
• Provide strategic quality leadership on the input into the IND of major investigational product and commercial submissions and response to HA queries, assuring that they are complete, scientifically accurate, of high quality and are presented in a way that facilitates both internal and agency review.
• Evaluate program(s) providing strategic quality decisions and documentation approvals as required.
• Evaluate and communicate risks to the development program(s) you manage to ensure successful launch of life changing products.
• Ensure filing and inspection readiness from an end-to-end product lens, and as needed, support both site readiness and any applicable interactions with regulatory agencies.
• Monitor and interpret cGMP regulatory guidelines and trends in the breakthrough therapy regulatory space and incorporate into phase appropriate control strategies.
• Serve as the quality lead to support product risk assessments, supporting the identification of quality risks and appropriate mitigation plans to achieve compliant solutions.
• Ensure the product team's understanding of quality and compliance standards and market specific regulations for aligned programs, serving as the escalation lead on behalf of Quality to guide and manage challenges to achieve risk-based solutions as challenges occur.
• Identify and drive continuous improvement and standard practices for Quality to achieve both top performance and consistency, where appropriate, across our portfolio.
• Define the stage appropriate gates required for preclinical and clinical method development programs.
• Support the definition and requirements for development programs. Manage site inspection preparation, management and response process.
• Selection of manufacturing, testing, and distribution contract suppliers (support audits as needed).
• Provide quality input during manufacturing runs, product specifications, analytical testing and release at CMOs and internal AveXis sites.
• Ensure all stability studies are designed and executed to enable IND and eventual commercialization.

Qualifications

• Bachelors' and/or Masters/PhD' degree in scientific discipline with a technical understanding of biopharmaceutical production and/or understanding of cell or gene therapy and drug product development and manufacturing and testing.
• Minimum of 10-12 years of biologics or gene therapy industry experience in technical, quality, and/or regulatory role(s) supporting programs in pre-clinical and clinical phase programs, with at least one or more role (5+ years) in Quality (QA, QC).
• Comprehensive knowledge of FDA and EMA regulations and experience in US and international regulatory agency inspections.
• Direct experience reviewing and/or authoring IND/BLA sections of regulatory filings and partnering with operations on product related investigations and deviations.
• Strong project management skills and ability to communicate effectively across matrix functions at all levels of the organization, influencing areas not under direct control to achieve shared objectives while balancing speed, quality and risk.
• Proficiency in development and execution of strategic decisions, balancing multiple priorities in a fast-paced environment while promoting a culture of quality and continuous improvement.

Approximately 20% travel required.

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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