Associate Director, Regulatory CMC

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. 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.

Oversees regulatory CMC activities and maintains the regulatory CMC applications. Responsible for executing the regulatory CMC strategy of submissions to the US FDA, EMA, and other regulatory authorities as well as managing interactions with regulatory authorities on CMC issues. Aid in development of a CMC Regulatory function for AveXis.

Responsibilities

• Implement regulatory CMC strategy for early and late stage clinical product candidates to attain rapid regulatory approvals.
• Lead and drive CMC content development for submission activities (planning, authoring, reviewing, coordination, submission) for original INDs, IND amendments, investigational medicinal product dossiers (IMPD) for inclusion in Clinical Trial Applications, BLAs and MAAs.
• Serve as RA CMC liaison with various internal functions (e.g., Manufacturing, MS&T, R&D, etc.).
• Participate in multidisciplinary efforts to prepare CMC updates for regulatory documents including IND CMC updates, Regulatory Authority meetings briefing documents, etc.
• Aid in development of regulatory dossier in support of AveXis products in development for worldwide submissions.
• Respond to CMC queries to advance AveXis clinical programs.
• Aid in establishing, managing, and maintaining a knowledge base of current and emerging CMC regulatory requirements, guidelines, and best practices.
• Assure compliance with regulatory standards and guidance documents.
• Ensure that CMC-related changes are reported to authorities in accordance with regulatory requirements.
• Conduct risk assessments of global CMC regulatory issues.
• Help in building a world class Regulatory CMC function to meet AveXis growing pipeline.
• Maintains and continues to build AveXis' excellent reputation with the regulatory authorities.
• Indirectly supervises employees through a dotted line structure or via other subordinate managers.

Qualifications

• BS/MS degree in related discipline and 10 years of related experience with at least 5 years at a manger or above role.
• Experience and a proven track record of successes in biological drug submissions (e.g., IND, BLA, MAA), is preferred.
• Experience with biologics required with experience in cell/gene therapy drug development highly desirable.
• Knowledge of the drug development process and global regulations and requirements required.
• Strong analytical skills, problem solving ability and presentation skills required.
• Flexibility and ability to prioritize and manage multiple tasks simultaneously.
• Effective oral and written communication
• Develops departmental policies and oversees and/or authorizes their implementation.
• Applies strong analytical and business communication skills.

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.