Data Integrity Assessment & Remediation for Pharmaceutical Compliance
Cell & Gene Therapy
Novartis manufactures a gene therapy designed to replace the function of the nonworking or missing gene that causes Spinal Muscular Atrophy (SMA). The 170,000-square-foot facility in Durham primarily produces drug substance.
Sequence Scope: Pharmaceutical Compliance
This project involved assessment and remediation of Manufacturing, Operational and Analytical Equipment system audit trails at two Novartis Gene Therapy locations. Sequence resources developed a project plan and established a structured schedule for the initiation and completion of the required tasks. Audit trail review assessments were led by Sequence resources to ensure all regulatory requirements and pharmaceutical compliance were assessed. Where gaps were identified during the assessments, CAPA(s) were initiated and guided to closure. Equipment/platforms for this project included Compliance Wire, OSI PI Historian, and Empower.
What is Pharmaceutical Compliance?
Life sciences and pharmaceutical compliance programs ensure that the products manufactured in these industries are safe for consumers, meet their intended usage specifications and that the processes used to produce them adhere to certain quality standards. These compliance regulations can cover the manufacturing, control, storage and distribution of the products. Typically compliance programs will outline several aspects of the manufacturing process including: implementing policies and procedures, training and education of employees, and monitoring and auditing of processes. Pharmaceutical compliance is an important and potentially life-saving component to any manufacturing process in the life sciences industry. Let Sequence help you promote a culture of early risk mitigation and accountability into your compliance project plan and management.
Spinal Muscular Atrophy (SMA) is a rare genetic disease that leads to progressive muscle weakness, paralysis and, when left untreated in its most severe form, permanent ventilation or death for most patients by age 2. The manufactured gene therapy is designed to replace the function of the nonworking or missing gene that causes Spinal Muscular Atrophy (SMA) in infants, children and adults by addressing the genetic root cause of SMA by replacing the defective or missing SMN1 gene to halt disease progression with a single, one-time infusion. Source