U.K. Gene Therapy Manufacturing Consortium Seeks to Address Barrier to Commercialization

Rare Daily Staff

Plasticell, a developer of stem cell technologies and therapies, is leading a consortium of United Kingdom gene therapy groups at UCL’s Institute of Child Health and Great Ormond Street Hospital to develop advanced technologies for the manufacturing of 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,” said Yen Choo, founder and Executive Chairman of Plasticell. “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.”

A number of gene therapies in which faulty cells isolated from a patient’s peripheral blood are corrected by a virus carrying a functional copy of a therapeutic gene are now either approved or in the late clinical development for a number of rare diseases. But the high cost of these therapies remains a barrier to delivering life-saving treatments to patients.

Plasticell will develop novel methods for enhancing lentiviral gene delivery to hematopoietic stem cells using its combinatorial screening technology CombiCult.

The scientific consortium will fund this project partly through a competitive, non-dilutive grant of $976,250 (£740,000) from Innovate UK as part of its Innovation in Health and Life Sciences competition.

June 29, 2018
Photo: Yen Choo, founder and Executive Chairman of Plasticell