FDA Lifts Clinical Hold on Beam’s Experimental Gene Editing to Treat Certain T-Cell Cancers

The U.S. Food and Drug Administration lifted a clinical hold on Beam Therapeutics’ BEAM-201 for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL)/T-cell lymphoblastic lymphoma (T-LL).

Photo: John Evans, CEO of Beam

The FDA notified the company on July 29, 2022 that it placed a clinical hold on BEAM-201. The agency said it wanted additional control data from genomic rearrangement assessments, further analyses of certain off-target editing experiments, additional control data for a cytokine independent growth assay,  and an updated investigator brochure that includes information regarding any new nonclinical studies.

Chimeric antigen receptor T-cell (CAR-T) therapies have the potential to address a number of T-cell cancers, but their therapeutic potential is often hindered by technological limitations in their development and application. To address these limitations, Beam said it is using multiplex base editing to create donor-derived CAR-T cells by simultaneously silencing multiple target genes, thereby potentially enabling universal compatibility and resistance to host rejection, fratricide, and immunosuppression.

BEAM-201 is a potent and specific anti-CD7, multiplex-edited, allogeneic chimeric antigen receptor T-cell development candidate.

“We believe the future of cell therapy involves high levels of cell engineering, enabled by multiplex base editing technology,” said John Evans, CEO of Beam. “Combining four unique edits with high efficiency, BEAM-201 has the potential to make a substantial impact for patients diagnosed with these challenging T-cell cancers, who lack innovative, new treatment options for their disease.”

Author: Rare Daily Staff