Beam Therapeutics’ BEAM-101 Receives FDA Orphan Drug Designation for Sickle Cell Disease

CAMBRIDGE, Mass. June 03, 2025 (GLOBE NEWSWIRE)  – Beam Therapeutics Inc., a biotechnology company specializing in precision genetic medicines through base editing, announced on June 3, 2025, that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to BEAM-101. This investigational genetically modified cell therapy is being developed for the treatment of severe sickle cell disease (SCD).

Dr. Amy Simon, Beam’s chief medical officer, highlighted the significance of this designation, noting that SCD is a devastating disorder affecting approximately 100,000 people in the U.S. She stated that BEAM-101, the lead program in the company’s hematology franchise, has the potential to offer a differentiated, best-in-class treatment and expressed commitment to progressing the BEACON Phase 1/2 clinical trial.

The FDA’s Orphan Drug Designation aims to support the development of treatments for rare diseases affecting fewer than 200,000 individuals in the U.S., offering benefits such as tax credits, user fee exemption, and potential market exclusivity.

Initial clinical data for BEAM-101, presented at the 66th American Society of Hematology (ASH) Annual Meeting in December 2024 and accepted for presentation at the European Hematology Association (EHA) 2025 Congress in June, showed promising results from seven treated patients. The data demonstrated robust and durable increases in fetal hemoglobin (HbF) and reductions in sickle hemoglobin (HbS), along with rapid engraftment of neutrophils and platelets, and normalized or improved markers of hemolysis. The initial safety profile was consistent with busulfan conditioning and autologous hematopoietic stem cell transplantation.

BEAM-101 is a one-time therapy using autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) that are base-edited to increase the production of non-sickling fetal hemoglobin (HbF). This process mimics naturally occurring variants that lead to hereditary persistence of fetal hemoglobin.

Beam is currently enrolling the pivotal part of its BEACON Phase 1/2 study, expecting to dose 30 patients by mid-2025. The company anticipates reporting topline data in early 2026 and plans to apply for FDA approval in mid-2026.