Europe – EMA : Guideline on quality, non-clinical and clinical aspects of medicinal products containing genetically modified cells

This guideline defines scientific principles and provides guidance for the development and evaluation of medicinal products containing genetically modified cells intended for use in humans and presented for marketing authorisation. Its focus is on the quality, non-clinical aspects, safety and efficacy requirements of genetically modified cells.

The quality section addresses the requirements specific to the genetic modification of the target cell population and to the genetically modified cell product resulting from the manufacturing process.

The non-clinical section addresses non-clinical studies required to assess the proof-of-concept and biodistribution of the product, to identify potential target organs of toxicity, and to obtain information on dose selection for clinical trials, to support the route of administration and dosing schedule…