Beam Therapeutics Announces Orphan Drug Designation For BEAM-101

(RTTNews) - Beam Therapeutics (BEAM) announced that the FDA has granted orphan drug designation to BEAM-101, an investigational genetically modified cell therapy for the treatment of sickle cell disease.

Amy Simon, chief medical officer of Beam, said: "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."

Shares of Beam Therapeutics are up 1% in pre-market trade on Tuesday.

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