Beam Therapeutics announces U.S. FDA granted RMAT to BEAM-101

Beam Therapeutics (BEAM) announced that the United States Food and Drug Administration, FDA, has granted Regenerative Medicine Advanced Therapy, RMAT, designation to BEAM-101, an investigational genetically modified cell therapy for the treatment of sickle cell disease, SCD. “We are thrilled that the FDA has granted RMAT designation to BEAM-101, following orphan drug designation earlier this year, reinforcing its potential as a one-time, best-in-class therapy for severe sickle cell disease,” said Giuseppe Ciaramella, Ph.D., president of Beam Therapeutics. “These designations not only recognize the promise of BEAM-101 but also enable enhanced collaboration with the FDA as we advance toward a BLA filing. With 30 patients now dosed in the BEACON Phase 1/2 trial and additional data expected later this year, we remain focused on delivering a transformative treatment to people living with sickle cell disease.”