Cold Chain Confidence: REGENXBIO’s Transport Validation Journey
Cold Chain Confidence: REGENXBIO’s Transport Validation Journey
Challenge
REGENXBIO is a leading biotechnology company focused on developing, commercializing, and licensing recombinant adeno-associated virus (AAV) gene therapy.
Their mission is to transform the lives of patients suffering from severe diseases with significant unmet medical needs through innovative gene therapy solutions based on their proprietary NAV Technology Platform.
In preparation for the Biologics License Application (BLA) filing of RGX-121, a gene therapy product, REGENXBIO identified a critical gap in their knowledge regarding transport validation and regulatory expectations.
Ensuring product stability and compliance throughout the supply chain required specialized expertise in cold chain engineering.
To address these challenges, REGENXBIO turned to Modality Solutions.
Objectives
To provide a complete transport validation package supporting the BLA submission, Modality Solutions delivered:
• Process Flow Map
• Formal Risk Assessment
• Validation Master Plan
• Transport Simulation Study
• Selection and Qualification of Thermal Packaging
• Development of Work Instructions for Packout and Shipment
• Design and Execution of Performance Qualification (PQ) Studies
• Comprehensive Validation Activity Summary
• Regulatory Support Pre- and Post-Filing
Results
REGENXBIO is on track to file their BLA supported by a comprehensive and regulatory-compliant transport validation package developed by Modality Solutions. This ensures a streamlined approval process and compliance with regulatory requirements.
By leveraging Modality Solutions’ expertise, REGENXBIO gained confidence in their cold chain logistics, ensuring the safe and effective transport of RGX-121.
With a regulatory decision expected before the end of 2025, RGX-121 could become the first gene therapy approved for Hunter syndrome.
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