Cellectis gets US ok to test its universal donor derived cancer cell therapy

By Gareth Macdonald

- Last updated on GMT


Related tags Leukemia

US regulators have cleared French biotech Cellectis to trial its “off-the-shelf” blood cancer cell therapy, UCART123.

The product consists of T-cells modified to target the CD123 antigen on the surface of cancerous cells.

Jennifer Moore, VP of Communications at Cellectis, told us: "CELLforCURE, the largest commercial industrial facility for the production of innovative therapeutic cell therapies in Europe, a subsidiary of the biopharmaceutical group LFB, will perform the cGMP manufacturing of clinical batches of Cellectis’ allogeneic CART cells.​"

Unlike autologous cell therapies that are made from the specific patients’ own cells, UCART123 is composed of lymphocytes harvested from an unrelated, so called “universal” donor.

The advantage of this allogenic approach is that developers can make and store stocks of cell therapies rather than having to produce a specific batch for each patient, which makes such treatments cheaper according to Loan Hoang-Sayag, Cellectis Chief Medical Officer.

Hoang-Sayag said: “Cellectis’ allogeneic UCART products have the potential to create an important shift with regard to availability, and cost-effectiveness, to make these therapies widely accessible to patient population across the world​.”

Gene editing

UCART123 - which Cellectis claims is the first allogenic cell therapy to be approved for trials by the US Food and Drug Administration (FDA) - will be tested as a treatment for patients with acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN). The Phase I clinical studies due to start in the first half of the year.

Like other therapies in Cellectis’ pipeline, UCART123 is produced using Talen gene editing to inset genes that encode a chimeric antigen receptor (CAR) that targets the CD123 antigen.

The gene editing method is also used to prevent the T-cells of which UCART123 is composed from interacting with non-target proteins, thereby reducing side-effects

UCART therapies

One of Cellectis UCART therapies was used to successfully treat a baby girl with leukaemia whose disease had progressed despite chemotherapy.

According to the results of a study published in 2015​, 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.

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