(Reuters) - Cellectis has won U.S. regulatory approval to run an early clinical trial using its gene edited cell therapy product UCART123 for blood cancers, boosting the French biotech firm’s ambitions in the hot area of cancer research.
Following approval from the Food and Drug Administration, Phase I clinical trials will start in the first half of this year, the company said on Monday.
It marks the first time that U.S. regulators have approved clinical testing of an allogeneic, or “off-the-shelf”, gene-edited CAR T cell treatment.
The idea of genetically altering immune cells called T cells so that they can attack cancers more effectively has attracted interest from a range of drugmakers.
But while rivals such as Novartis, Juno and Kite have treatments that use modified T cells extracted from individual patients, Cellectis products are derived from healthy donors and aim to be universal.
Its first such “off-the-shelf” cell therapy UCART19, which is being developed with Servier and Pfizer, is now being tested in Phase I trials in Britain for acute lymphoblastic leukaemia and chronic lymphocytic leukaemia.
It has already rescued two babies treated at London’s Great Ormond Street Hospital from previously incurable cancer.
UCART123, which is still wholly owned by Cellectis, is designed to help patients with acute myeloid leukaemia and blastic plasmacytoid dendritic cell neoplasm.
Reporting by Ben Hirschler; Editing by Ruth Pitchford