Module 9 2024

03/09/2024

IFN-y may Prompt MAS rather than enabling cell killing

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Non-Clinical

In vivo behavior

Extent of non-clinical studies • Pre-exisiting clinical data •

Biologics ATMPs

Previous non-clinical data with the same product

MABEL

Effective dose

Duration of studies •

p450 degradation system

Difficult to establish in long life resident products • Dependant to long term expected effects • Large animals longer life expectancy than smaller animals • GvHD hinders long tern studies for CAR+T Choice of relevant animal model • Homologous / disease animal model for PoC studies • Immunosuppressed animals Full GLP compliance

Long time resident

PK and TK

Biodistribution

Carcinogenicity, mutagenesis

Tumuorigenicity and insertional mutagenesis

Developmental toxicity

Vertical transmission

Safety assessment from integrated analysis of in-vitro and in-vivo data

The Organisation for Professionals in Regulatory Affairs

40

20