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Sickle cell disease cell therapy BEAM-101 named FDA orphan drug
BEAM-101, a genetically modified cell therapy for sickle cell disease (SCD) that’s being tested in clinical trials, has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). The FDA gives the designation to experimental therapies designed to treat conditions that affect fewer than 200,000 people in the U.S. The designation seeks to provide extra economic incentives to companies developing treatments for rare diseases, w…
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