Leadership Guide
Beyond Toxicology
The therapy didn't fail because it was toxic. It failed because the patient's immune system decided it was a threat.
What the Framework Reveals
Safety and immunogenicity in cell and gene therapy
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living, biologically active agent — no toxicology panel was built to predict its immune interaction
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additional questions a real CGT safety strategy must answer: biodistribution and shedding
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toxicology findings behind most CGT immune reactions in the clinic
A gene therapy passes every preclinical toxicology study clean and still triggers a dangerous systemic immune reaction in the clinic — not because anything was toxic, but because the therapy is a living, biologically active agent interacting with an immune system no toxicology panel was built to predict. This guide covers immunogenicity, vector-specific safety risk, and the biodistribution and shedding questions a real CGT safety strategy must answer.
Source: Leadership Guide · Aug 2026 · MKA-20A-LG, Executive Summary
Built for: CGT safety, quality, and regulatory affairs teams.