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Data Integrity Assessment & Remediation

Cell & Gene Therapy

Overview

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

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 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.

Life Impact

  • 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

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