Cellectis' off-the-shelf CAR T-Cells treat 11-month old with leukaemia

By Dan Stanton

- Last updated on GMT

Related tags T cell B cell Major histocompatibility complex

An ‘off-the-shelf’ CAR (chimeric-antigen receptor) T-Cell therapy from Cellectis has shown to have successfully treated a baby girl with leukaemia.

A paper being presented by Cellectis during the upcoming American Society of Hematology (ASH) Annual Meeting is set to show how its Talen Engineered Universal CAR19 T Cells exhibit powerful anti-leukemic effects in patients with B cell malignancies, following the successful treatment of an 11-month old girl.

Despite chemotherapy, the girl’s diseased progressed, an abstract of the paper​ says. But the girl received a single dose (4.5x106/kg) of Cellectis’ allogeneic engineered T-cell product UCART19 and two months later the girl was cleared of leukaemia.

“Within the short period of follow up available, our intervention comprising lymphodepletion and infusion of UCART19 T cells has induced molecular remission where all other treatments had failed,”​ the paper says.

“This first-in-man application of Talen engineered cells provides early proof of concept evidence for a ready-made T cell strategy that will now be tested in early phase clinical trials.”

The product is, of course, still in its early stages, and this case shows data from a single patient, but the results is likely to have caught the attention of other investors in CAR-T Cell therapies such as Novartis, Juno Therapeutics and Amgen-partnered Kite Pharma.

Cellectis – itself subjected to takeover rumours in May by its partner Pfizer​ - describes its UCART (Universal Chimeric Antigene Receptor T-cells) candidates as “off-the-shelf”​ allogeneic products.

If successful, this would mean production of its therapies can be industrialised and standardised with consistent pharmaceutical release criteria, over time and from batch to batch, making manufacture cheaper and easier than rival CART candidates.

While Swiss Pharma Giant Novartis would not comment on data presented by other companies, spokesperson Dana Kahn Cooper told Biopharma-Reporter.com its lead CAR-T candidate is in Phase II multi-centre global studies.

Furthermore, “we have the first FDA-approved Good Manufacturing Practices quality site for a cell therapy site in Morris Plains, and are processing and re-programming cells to support our ongoing clinical program.”

Cellectis, Juno and Kite did not respond when contacted by this publication for comment.

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