Pre-clinical Assessment Platform

Genethon has set up evaluation platform in order to generate and compile all pre-clinical data for assessment of efficacy, toxicity and side effects of a gene therapy product. These platforms are divided between the Evry site and the Boisbonne animal testing center (located in Nantes, in collaboration with the Ecole Nationale Vétérinaire de Nantes). This set of data will serve as a key element to the regulatory file, submitted to the regulatory agencies in the framework of an IND or an IMPD.

Functional Studies

The main role of Genethon’s Functional Studies Platform is to assess functional efficacy of gene therapy strategies in animal model of pathologies of interest.

Key parameters in myopathy models include strength, motor and heart function, measured by ultrasound analysis.

The aim of these studies is to define the treatment window and the mode of administration in order to be able to predict human therapeutic doses.

Imaging and Histology

Imaging, histology and cytometry are used to assess the therapeutic effects and any adverse reactions at cell, tissue and whole animal level. The Genethon teams who carry out this work are also responsible for ensuring the transfer of medical imaging technologies in preparation for the clinical trials.