Our researchers use sophisticated laboratory techniques and “big data” analytic methods to examine genetic influences on health and disease, and assess the safety of novel gene therapies.
Case Study: Screening for Gene Therapy Safety and Efficacy
Pharmaceutical companies including GlaxoSmithKline (GSK), Novartis, AstraZeneca and Pfizer are investing heavily in gene therapy virus technology, bringing several new products to market to provide treatments for acquired diseases and genetic disorders including cancer. However, no standardised platform has been available to test vector safety. Following the unexpected development of leukaemia in several children treated with gene therapy, the Brunel team developed a method to test the safety of the gene therapy vectors. The Brunel team, with colleagues from UCL, KCL, GeneWerk, and NMI in Germany, won the Small Business Research Initiative InMutaGene CRACK-IT Challenge, funded by the National Centre for the Replacement, Refinement and Reduction of Animal Research, sponsored by GSK and Novartis. The team generated the first human-induced pluripotent stem cell (iPSC) directly from a cell-based platform that evaluates the safety of gene therapy products. The technology will also enable pre-treatment screening to identify individual patient tolerance to gene therapy and profiling of vector side effects. With patents filed, TestaVec Ltd, a spin-out of Brunel University London, will enable researchers and industry to bring their products safely into the clinic.
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Dr Michael Joseph
Business Development Manager
“The InGetox platform is an exciting new model developed by Brunel University London, UK and GeneWerk, Germany with the potential to offer safety profiling of gene therapy vectors before entering the clinic”
Silvana Libertini, Laboratory Head of Pre Clinical Safety, Novartis, Basel, Switzerland and Jan Klapwijk, Director of Pathology at GSK, UK