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

Beam Therapeutics (BEAM) announced that the United States Food and Drug Administration, FDA, has granted orphan drug designation, ODD, to BEAM-101, an investigational genetically modified cell therapy for the treatment of sickle cell disease. “Sickle cell disease is a devasting disorder that affects approximately 100,000 people in the U.S., leading to anemia, severe pain, stroke and even early death. Receiving orphan drug designation from the FDA emphasizes the importance of new treatment options for this debilitating disease, and our clinical data suggest that BEAM-101, the lead program in our hematology franchise, has the potential to offer a differentiated, best-in-class treatment,” said Amy Simon, M.D., chief medical officer of Beam. “We look forward to continuing to progress our BEACON Phase 1/2 clinical trial of BEAM-101 in patients with severe sickle cell disease and to working closely with the FDA with the goal of bringing BEAM-101 to patients as safely and quickly as possible, embodying our mission of providing lifelong cures to patients suffering from severe diseases.”