San Raffaele Telethon Institute for Gene Therapy
Process Development Lab
The Process Development Lab (PDL) is dedicated to the optimization, scale-up and pre-clinical validation of protocols for the preparation and control of gene and cell therapy products generated in SR-Tiget research laboratories, with the aim of making them suitable for transfer to specialized contract manufacturing organizations operating according to Good Manufacturing Practices (GMP). All PDL activities are carried out in a context of Good Scientific Practices and Quality by Design in order to ensure that the development of products and processes is conducted in a systematic, accurate and reproducible way, following internal procedures and best practices recognized in the scientific community.
PDL personnel has documented experience in the following areas, which represent two key activities within the facility:
- Manipulation and genetic modification of hematopoietic stem cells and T lymphocytes (selection, transduction, genome editing, expansion; analytical methods to evaluate cell quality in terms of identity, purity, potency and safety)
- Manufacturing of lentiviral vectors at a pre-GMP level (upstream and downstream process; analytical methods to evaluate vector quality)
Soldi M, Sergi Sergi L, Unali G, Kerzel T, Cuccovillo I, Capasso P, Annoni A, Biffi M, Rancoita PMV, CantoreA, Lombardo A, Naldini L, Squadrito ML, Kajaste-Rudnitski A.Laboratory-Scale Lentiviral VectorProduction and Purification for Enhanced Ex Vivo and In Vivo Genetic Engineering. Mol Ther MethodsClin Dev. 2020 Oct 20;19:411-425. doi: 10.1016/j.omtm.2020.10.009. eCollection 2020 Dec 11.