CAMBRIDGE, Mass., June 03, 2025 - Beam Therapeutics Inc. (Nasdaq: BEAM) announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to BEAM-101, an investigational genetically modified cell therapy for treating sickle cell disease $(SCD)$. This designation underscores the critical need for innovative treatments for SCD, a debilitating disorder affecting approximately 100,000 people in the U.S. BEAM-101, the lead program in Beam's hematology franchise, has shown promise in clinical trials, demonstrating significant increases in fetal hemoglobin and reductions in sickle hemoglobin among patients. The FDA's orphan drug designation offers potential benefits, including tax credits for clinical trials and a possible seven years of market exclusivity post-approval. Beam Therapeutics continues to advance its BEACON Phase 1/2 clinical trial, with plans to dose 30 patients by mid-2025.
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