Module 9 2024

03/09/2024

Manufacturing

Small Molecule, Biologics and ATMPs: Key Quality Differences

Biologics

ATMPs

Produced by host cells

Cell or virus is the product

Process product specific

Can be patient specific

Large batches

Small batches

Terminal sterilization

No terminal sterilization

Structure and characterization

Biologics

ATMPs

High M.W.

No M.W. defined

Mass activity Balance ( μ g, IU)

Cell/kg, MOI, Viability

Product degradation

Unwanted cell population

The Organisation for Professionals in Regulatory Affairs

27

In-Vivo and Ex-Vivo GT

The Organisation for Professionals in Regulatory Affairs

28

14