Consortium to Advance Methods for Manufacture of Gene Therapies

Pioneering gene therapy groups at UCL’s Institute of Child Health (ICH) and Great Ormond Street Hospital (GOSH) are working with stem cell technologies and therapies company Plasticell to develop methods for the manufacturing 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 commercialise 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” commented Dr. Yen Choo, founder and Executive Chairman of Plasticell.

Plasticell’s CombiCult combinatorial screening technology will be utilised in the studies for enhancing lentiviral gene delivery to hematopoietic stem cells and expanding the numbers of corrected cells ex vivo. The collaboration builds on the company's work with Professor Adrian Thrasher from UCL ICH, who has led clinical development of gene therapies for Severe Combined Immunodeficiencies (SCIDs), Wiscott-Aldrich Syndrome (WAS) and Chronic Granulomatous Disease. GMP-compliant 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, obtained from Innovate UK as part of its ‘Innovation in Health and Life Sciences’ competition.

“We are delighted to extend our collaboration with Plasticell. Gene Therapies are showing real benefit in patients. This is an excellent opportunity to improve the manufacturing technology as we move towards drug licensing”, commented Professor Adrien Thrasher, Fellow at the UCL Great Ormond Street Institute of Child Health.

“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.