First base editing therapeutic for T-ALL/T-LL enters trials

CAR-T therapy

The first quadruplex-edited, allogeneic CAR-T cell therapy candidate has entered clinical trials in the US.

The first patient has been treated with Beam Therapeutics’ BEAM-201 as part of a Phase I/II clinical study for the treatment of relapsed/refractory T-cell acute lymphoblastic leukaemia/T-cell lymphoblastic lymphoma (T-ALL/T-LL).

“As the first patient dosed with a Beam therapeutic candidate and the first patient in the US to receive a base editing therapeutic, this represents a major milestone for the company, the scientists that made this possible, and the patients we hope to serve,” said John Evans, Chief Executive Officer of Beam.

“We believe that the full therapeutic potential of CAR-T therapies, including the ability to utilise an allogeneic source of T cells, will only be unlocked through higher levels of cellular engineering enabled by multiple simultaneous genetic edits. Base editing is especially well-suited to this challenge, as it is designed to deliver highly efficient multiplex edits in cells without the double stranded breaks that can lead to frequent chromosomal rearrangements and loss of cell viability.”

The primary objectives of the Phase I portion of the trial are the assessment of safety, tolerability, and identification of the recommended Phase II dose and lymphodepletion regimen.

Key safety endpoints for the trial include treatment-emergent and treatment-related adverse events, and key efficacy endpoints include proportion of patients with complete or partial responses, proportion eligible for hematopoietic stem cell transplant, and proportion achieving minimal residual disease negative status.

Multiplexed base editing is designed to eliminate expression of the CD7, TRAC, PDCD1 and CD52 genes. This approach has the potential to reduce fratricide, graft-versus-host disease and CAR-T cell exhaustion.

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