Consortium préindustriel des vecteurs de thérapie génique

GENETHON and EFS-ABG, each, were running a clinical-grade vector production site that complies with Good Manufacturing Practice (GMP) and were both granted and registered by regulatory bodies as pharmaceutical manufacturer sites in 2013. To our knowledge, both PGT partners are the first not-for-profit entities in Europe to obtain such registration. Practically and with respect to PGT’s main mission, it allows us to manufacture, export and release advanced-therapy medicinal products to any European investigators. In November 2016, the existing bioproduction platform of Genethon (GBP) was integrated in the new project supported by the AFM-Telethon and the « SPI – Sociétés de Projets Industriels » equity fund, headed by Bpifrance as part of the Investments Program for the Future. The two subjects decided to co-fund the creation of the largest European center, named YposKesi, for the development and manufacturing of gene and cell therapies. In October 2017, EFS ABG has opened an extension of its facilities with new rooms dedicated to gene therapy production and characterization.- DISSEMINATION: A dedicated website was launched in 2013 (www.pgt-consortium.fr). The PGT consortium was present at the ESGCT congress in Barcelona from 22-25 October 2019 and at the ASGCT congress in Washington, DC, April 29 -May 2, 2019. ONGOING PROJECTS: The developments presented and agreed in 2017 aim at scaling-up and improving the production processes as well as the analytical methods, maintaining process improvement and implementation of new technologies in order to meet the needs of the gene therapy market. EFS-ABG continues with the extension of its production platform, as well as the development of ex vivo gene therapy. – PRECLINICAL AND CLINICAL VECTOR PRODUCTION: In 2019, 15 large scale preclinical and clinical vector productionswere produced, both for academic and non academic needs.