Dive Brief:
- Plasticell, a developer of stem cell technologies and therapies based in Stevenage, U.K., is heading up a consortium of academic gene therapy groups at two London hospitals.
- Teams from University College London's Institute of Child Health and Great Ormond Street Hospital will work on advanced technologies for manufacturing ex vivo gene therapies.
- To start, the group will target chronic granulomatous disease and other rare inherited immune disorders, with funds from a non-dilutive grant of £740,000 (around $973,000) from the government's Innovate UK agency, as part of a life sciences competition.
Dive Insight:
As gene therapies move onto the market, manufacturing and creating a safe, secure and cost-effective supply chain will be a challenge for both small and large companies.
The consortium is Plasticell's attempt to tap into academia to boost its manufacturing approaches. The group will use Plasticell's combinatorial cell culture screening platform, CombiCult, to improve lentiviral delivery to hematopoietic stem cells, with the aim of expanding the number of corrected cells outside the body.
"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," said Yen Choo, founder and executive chair of Plasticell, in a statement.
"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," said Choo.
Plasticell has already worked with Adrien Thrasher at University College London's Institute of Child Health to develop gene therapies for severe combined immunodeficiencies (SCIDs), Wiscott-Aldrich syndrome (WAS) and chronic granulomatous disease.
The biotech has a history of collaborations with research groups. In March 2017, the biotech signed agreements with the Agency for Science, Technology and Research (A*STAR) and the Nanyang Technological University (NTU), both in Singapore, to create engineered cell lines for cell therapy development and for drug discovery screening.
In April 2017, Plasticell partnered with King's College London to move its artificial blood platelet product, manufactured from pluripotent stem cells, forward through preclinical development. In December 2017, Anthony Nolan, a research organization working on blood cancers, signed a deal to work with Plasticell to move the biotech's ex vivo expanded cord blood-derived hematopoietic stem cell product forward through clinical development.
The deals aren't just academic. Also in 2017, GlaxoSmithKline and Plasticell hooked up. The British pharma giant is using CombiCult to optimize the differentiation of induced pluripotent stem cells (iPSCs) into specific blood cells.