Beam to seek OK of gene-edited cell therapy risto-cel for sickle cell disease

The BEACON clinical trial showed therapy's lasting benefits in 31 patients

Beam Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) to approve ristoglogene autogetemcel, or risto-cel, its single-dose, gene-edited cell therapy candidate for people with sickle cell disease (SCD), as early as year’s end.

The application is expected to be supported by now-published interim data from the ongoing Phase 1/2 BEACON clinical trial (NCT05456880), which demonstrated the therapy’s lasting benefits in 31 severe SCD patients, with milder symptoms and no severe vaso-occlusive crises (VOCs) reported so far.