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Medical Product Safety & Effectiveness Institute

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07

First-Principles Safety & Effectiveness Engineering for Cell & Gene Therapies

Apply the first-principles safety and effectiveness confidence framework to cell and gene therapy development, manufacturing, clinical delivery, and lifecycle management by integrating product and process understanding, patient-specific considerations, analytical characterization, manufacturing science, risk management, clinical evidence, and long-term learning.

Challenges

Real-world challenges addressed

  • Complex product characterization
  • Potency assay development
  • Variability in starting materials and patient-derived products
  • Product and process interdependence
  • Autologous and allogeneic manufacturing variability
  • Chain of identity and chain of custody
  • Comparability following process changes
  • Manufacturing scale-up and technology transfer
  • Storage, transport, and administration controls
  • Limited patient populations
  • Durability of clinical benefit
  • Uncertain long-term effects
  • Long-term patient follow-up
  • Lifecycle knowledge management

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