Associate Director, Quality Assurance, Development Testing

Overview

Ready to join a team committed to moving gene therapies into the clinical and commercial settings for patients and families devastated by rare neurological genetic diseases? AveXis is advancing cutting-edge science to treat rare and life-threatening genetic diseases starting with our clinical-stage, proprietary gene therapy candidate, AVXS-101 (ZOLGENSMA®). We are in the midst of an incredible journey and are looking for passionate individuals to join us on this important mission.

AveXis, Inc., a Novartis Company , is a clinical-stage gene therapy company, dedicated to developing and commercializing novel treatments for patients suffering from rare and life-threatening neurological genetic diseases. Our initial product candidate, AVXS-101, is our proprietary gene therapy product candidate currently in development for the treatment of spinal muscular atrophy, or SMA, Type 1, the leading genetic cause of infant mortality, and for the treatment of SMA Type 2. The U.S. Food and Drug Administration, or FDA, has granted AVXS-101 Orphan Drug Designation for the treatment of all types of SMA and Breakthrough Therapy Designation, as well as Fast Track Designation for the treatment of SMA Type 1. In addition to developing AVXS-101 to treat SMA Type 1 and Type 2, we also plan to develop other novel treatments for rare neurological diseases, including Rett syndrome (RTT) and a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene.

The Associate Director is responsible for preclinical and clinical Quality Assurance (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 of AveXis 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).

Responsibilities

• In a global function, collaborate with Analytical and Development groups to support method qualification, validation and transfer activities, as well as comparability as required throughout product lifecycle.
• Assure documentation associated with R&D, vendors, and QC are complete, scientifically accurate, of high quality and are presented in a way that facilitates both internal and agency review for the development programs. The focus should be sound testing strategies (phase appropriate), on using repeatable/reproducible methods in a compliant manner.
• Perform documentation review and approvals for the Quality Assurance role including technical transfer protocol, evaluation of product specifications, methods, review and approval.
• Evaluate and communicate risks to the development programs managed (phase appropriate) to ensure successful launch.
• Ensure filing and inspection readiness and 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.
• Ensure the product team's understanding of quality and compliance standards for GLP and GCP testing requirements and market specific regulations for your programs, knowing when and how to effectively escalate issues to the IND Engine and senior leadership.
• Identify and drive continuous improvement and standard practices for Quality to achieve both top performance and consistency, across our portfolio.
• Support the definition and requirements for development programs. Manage site inspection preparation, management and response process.
• Selection of testing sites and support audits as needed.
• Provide quality input on product specifications and methods at CMOs and AveXis sites.
• Review all stability studies and ensure they 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 5-8+ years of biologics or gene therapy industry experience in technical (e.g. method development, transfer), quality, and/or regulatory role supporting programs in pre-clinical and clinical phase programs, with at least one or more role (5+ years) in Quality (QA, QC, R&D QA).
• 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 communication skills at all levels of the company, and skills in influencing areas not under direct control to achieve shared objectives while balancing speed, quality and risk.
• Proficiency in balancing multiple priorities in a fast-paced environment while continuously promoting a culture of quality and 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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