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Beam Therapeutics Says Potential Sickle Cell Therapy Receives FDA Orphan Drug Designation
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Beam Therapeutics Says Potential Sickle Cell Therapy Receives FDA Orphan Drug Designation
Jun 3, 2025 5:29 AM

08:11 AM EDT, 06/03/2025 (MT Newswires) -- Beam Therapeutics ( BEAM ) said Tuesday that the US Food and Drug Administration has granted orphan drug designation to BEAM-101, an investigational cell therapy for sickle cell disease.

The designation provides tax credits for development and a potential seven years of market exclusivity for the drug, which is currently being tested in a phase 1/2 clinical trial, the company said.

Sickle cell disease is an inherited ailment that can lead to anemia, severe pain, stroke, and early death.

Shares of the company were up 5% in premarket activity Tuesday.

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