Beam Therapeutics Inc. (NASDAQ:BEAM) shares jumped 12% following the announcement that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BEAM-101, the company’s investigational cell therapy for sickle cell disease.
The RMAT designation, which comes after an earlier orphan drug designation, provides Beam with opportunities for closer FDA collaboration, potential accelerated approval pathways, and possible priority review of future biologics license applications. The designation underscores BEAM-101’s potential as a one-time therapy for severe sickle cell disease.
“We are thrilled that the FDA has granted RMAT designation to BEAM-101, following orphan drug designation earlier this year, reinforcing its potential as a one-time, best-in-class therapy for severe sickle cell disease,” said Giuseppe Ciaramella, Ph.D., president of Beam Therapeutics.
The company recently presented updated data from its BEACON Phase 1/2 trial at the European Hematology Association Congress in June. Among 17 treated patients, the results showed robust increases in fetal hemoglobin, reductions in sickle hemoglobin, rapid cell engraftment, and improvements in hemolysis and oxygen delivery. Importantly, no vaso-occlusive crises were reported post-engraftment.
Beam, which focuses on precision genetic medicines using base editing, has now dosed 30 patients in the BEACON trial and plans to release additional data later this year. Its manufacturing process for BEAM-101 employs an advanced, largely automated system that has consistently delivered high yields and cell viability.
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