Plasticell, the UK-based biotech, revealed that the initial targets for the newly-formed consortium will be to work on rare inherited disorders of the immune system, including chronic granulomatous disease. This particular condition sees those living with the disease struggle to create reactive oxygen compounds the immune system needs to kill certain pathogens.
The consortium will focus on two key facets: with Plasticell and the University College London's Institute of Child Health focusing on developing methods of enhancing delivery of the gene therapy and Great Ormond Street Hospital will have the responsibility of manufacturing the eventual products.
Plasticell will use its combinatorial screening technology to improve the lentiviral gene delivery to hematopoietic stem cells (HSCs) and increase the number of correct cells ex vivo.
Yen Choo, executive chairman of Plasticell, responded to questions explaining the aims of the project: “The main goal is to increase the number of long-term engrafting HSCs transduced with a therapeutic transgene. This could be achieved by increasing transduction efficiency, or amplifying transduced, or both.”
Choo explained why this is necessary by outlining the current difficulties in producing gene therapies, saying: “First, the low numbers of HSCs present in peripheral blood mean that there is a danger there will be no long-term engraftment and the therapy will not ‘take’ i.e. will fail.
“Second, the low efficiency of viral transduction means a vast excess of virus has to be used in the therapeutic protocol. As the cost of the therapy is driven by cost of virus, we hope through our methods we can dramatically reduce the cost of the therapy.”
Facilitating the project is the funding, which has been agreed through Innovate UK, that will provide the group with £740,000 (€966,467). The grant was provided after the group applied to Innovate UK’s ‘Innovation in Health and Life Sciences’ competition, set up to encourage 'quality and sustainability or improved healthcare outcomes'.
The news builds on the previous link up that Plasticell had announced, at the end of last year, with the Anthony Nolan charity.