We provide pharmaceutical expertise on gene therapy products and technologies including preclinical studies, quality controls, development of production processes and industrial transfer.
LENTIVIRAL VECTOR PRODUCTION
Standardized upstream and downstream production processes for preclinical lots, from small scale to large scale. Process innovation and automation to maximize capacity, yields and reduce costs. Industrial transfer towards cGMP.
EIC Transition Project TREM2MEDS. Headed by University of Padua, towards the clinical implementation of TREM2 Microglia Engineering for treating DementiaS
Base editing hematopietic stem cells for the gene therapy of sickle cell disease is a highly innovative approach that we develop in collaboration with Institut Imagine, Genethon, APHP and IMRB.
EIC Transition Project TREM2MEDS. Headed by University of Padua, towards the clinical implementation of TREM2 Microglia Engineering for treating DementiaS
OUR PROJECTS
Various projects are conducted in partnership with international or national teams and enterprises.
Click on images to read some project's descriptions.
New gene therapies targeting immune or hematopoietic cells (B or T lymphocytes, CD34+ cells) based on technologies for gene addition or CRISPR/Cas gene editing. Preclinical studies, characterization, potency and safety assays.
DEVELOPMENT OF NEW PRODUCTS
ANALYTICS
Our laboratory is hosting the Genomics and Genome Editing Quality Control Platform of the Genother Biocluster in partnership with Institute Imagine and Genethon. Our laboratory is specialized in LV analytics (VCN, Vector Insertion Site Analyses) and in vivo evaluations.