Plasticell, a developer of stem cell technologies and therapies, is leading a consortium of pioneering gene therapy groups at UCL’s Institute of Child Health (ICH) and Great Ormond Street Hospital (GOSH) to develop advanced technologies for the manufacture of ex vivo gene therapies. The initial focus is on rare inherited disorders of the immune system including Chronic Granulomatous Disease. 
 
“There are a number of highly promising gene therapy targets currently under investigation globally but in order to commercialize these potential cures, the industry urgently needs to find better ways of manufacturing therapeutic products. It is a really exciting project as we test innovative solutions for some of the most advanced medical technologies in collaboration with leading groups in the field,” said Dr. Yen Choo, founder and executive chairman of Plasticell.
 
Plasticell will use its combinatorial screening technology, CombiCult, to develop novel methods for enhancing lentiviral gene delivery to hematopoietic stem cells, and expanding the numbers of corrected cells ex vivo. GMP manufacturing of the therapeutic products will be overseen by Great Ormond Street Hospital, where these and other gene therapies have been produced and delivered clinically.
 
The consortium will fund this project partly through a competitive, non-dilutive grant of £740,000 from Innovate UK as part of its ‘Innovation in Health and Life Sciences’ competition.
  
“We have previously used CombiCult to develop cell culture media that expand hematopoietic stem cells and, separately, methods that enhance lentiviral delivery to target cell types. This new collaboration provides us with the opportunity to perform advanced screens using peripheral blood-derived target stem cells and clinical-grade lentivirus to develop an efficient and cost-effective platform for autologous ex vivo gene therapy applications,” added Dr. Choo.