Brunel University London secures Japanese patent for gene therapy vector safety test

Gene therapy is a rapidly growing area of medical science. But there is little in the way of safety testing the vectors used to deliver gene therapy.

Lentivirus vectors (LV) work to treat genetic disease and cancer. But there is a concern that these vectors have the negative effect of insertional mutagenesis related genotoxicity. And currently available safety models, murine-based LV ones, don't reliably predict safety in humans.

Brunel University London and spin-out company TestAVec Ltd have developed hInGeTox as the first human in vitro platform that uses induced pluripotent stem cells and their hepatocyte-like derivatives to understand LV–host interaction for vector safety evaluation and design.

This testing system is the first to use human cells to assess the likelihood of a patient developing cancer when infected with a virus delivering cell or gene therapy.

“Our team is delighted that this important and novel science has been recognised worldwide by the gene therapy community," said gene therapy and genotoxicity specialist Professor Michael Themis from Brunel. "We will commercialise this opportunity along with our partners to bring greater certainty to gene therapies. This will support the safety and design of the tools used to deliver working genes to patients for their treatment.”

“Japan as well as Europe and the USA see gene therapy as a market with huge potential,” said TestAVec Ltd executive chairman, Robert Spencer. “And as the treatments grow, so the need for safety testing grows. And if we are going to sell in Japan we need the protection of a patent to stop people copying our technology.”