A global biotechnology company working toward regulatory approval of a gene therapy treatment was required to perform shipping validation to support review and approval in the US market.
As the primary indication was targeted for fatal neuromuscular genetic disease with unmet medical needs, the product was granted Fast Track, Rare Pediatric Disease (RPD) and Orphan Drug designations from regulatory bodies.
While these designations highlighted the unmet medical need, the submission date and review timeline posed significant risks to internal stakeholders that needed to manage the required qualification activities.
Lean Biologix was requested to support the team to qualify the following material types:
- Master Cell Banks and Working Cell Banks (cryovials, LN2)
- Plasmids and Plasmid Cell Banks (cryovials, biotainers, Dry Ice)
- Drug Substance (flexible bag/bioshell, Dry Ice)
- Bulk DP (crystal zenith vials, Dry Ice)
- Finished Good (multi count vial box, ancillary components, Dry Ice)
Summary of Engagement
Lean Biologix Process:
- Assembled team of SMEs to support various material type qualifications
- Streamlined qualification process to perform multiple material types in parallel
- Used bracketing approach to satisfy thermal load across multiple material types without repeat testing
- Used bracketing approach to determine worst case test payload for transportation simulation across multiple material types
- Supported design team to develop and qualify state-of-the-art custom dry ice shipping system for bulk drug product and finished good distribution
- Production Deployment
- Rigid outer container, payload immobilizer, onboard temperature and distribution hazards (vibration, shock etc.) monitoring with GPS communication to cloud based tracking platform