Aug 14, 12:21 PM

Beam Therapeutics Secures FDA RMAT Status for Sickle-Cell Gene Therapy

Beam Therapeutics said the U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy status to BEAM-101, its investigational one-time cell therapy for severe sickle cell disease. The RMAT designation gives the Cambridge, Massachusetts-based company earlier and more frequent interaction with the agency, the possibility of accelerated approval based on surrogate endpoints, and eligibility for rolling and priority review of a future biologics license application. BEAM-101 has already received orphan-drug status and is being studied in the open-label BEACON Phase 1/2 trial, where 30 patients have been dosed to date. Interim data presented in June showed durable increases in fetal hemoglobin and an absence of vaso-occlusive crises after engraftment. Beam said it expects to release additional clinical results later this year as it advances toward a potential regulatory filing.

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Beam Therapeutics Secures FDA RMAT Status for Sickle-Cell Gene Therapy

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