Intel stock spikes after report of possible US government stake
Investing.com -- Beam Therapeutics Inc. (NASDAQ:BEAM) stock surged 12% after the company announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BEAM-101, its investigational cell therapy for sickle cell disease.
The RMAT designation, which follows the orphan drug designation received earlier this year, provides Beam with opportunities for enhanced FDA collaboration, potential accelerated approval pathways, and possible priority review of future biologics license applications. This regulatory milestone recognizes BEAM-101’s potential as a one-time treatment 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 clinical data from its BEACON Phase 1/2 trial at the European Hematology Association Congress in June. Results from 17 treated patients showed robust increases in fetal hemoglobin, reductions in sickle hemoglobin, rapid cell engraftment, and improved markers of hemolysis and oxygen delivery. Notably, no vaso-occlusive crises were reported post-engraftment.
Beam Therapeutics, which specializes in precision genetic medicines through base editing, has now dosed 30 patients in the BEACON trial and expects to present additional data later this year. The company’s manufacturing process for BEAM-101 utilizes an advanced, largely automated system that has consistently demonstrated high yields and viability.
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