BEAM Up on FDA's Orphan Drug Status for Gene-Editing Therapy

Core Insights - Beam Therapeutics (BEAM) has received orphan drug designation from the FDA for its investigational genome-editing candidate, BEAM-101, aimed at treating sickle cell disease (SCD) [1][7] - The orphan drug designation provides Beam with seven years of market exclusivity post-approval, along with exemptions from FDA application fees and tax credits for qualifying clinical studies [2][7] - Following the announcement, Beam's shares increased by 4.3%, although the stock has seen a 32% decline year-to-date compared to a 2.9% decline in the industry [2][3] Development Activities - BEAM-101 is the lead candidate in Beam's hematology franchise, currently undergoing evaluation in the phase I/II BEACON study for adult SCD patients [4] - Preliminary data from the BEACON study indicated that BEAM-101 treatment resulted in a significant and lasting increase in fetal hemoglobin and a decrease in sickle hemoglobin [5][7] - Updated data from the BEACON study is anticipated to be presented at an upcoming European Hematology Association conference [8] Competitive Landscape - Other companies utilizing CRISPR/Cas9 technology include CRISPR Therapeutics and Intellia Therapeutics, both of which are developing therapies for blood disorders [9][10] - CRISPR Therapeutics, in collaboration with Vertex Pharmaceuticals, has received approvals for its CRISPR/Cas9 gene-edited therapy, Casgevy, for SCD and transfusion-dependent beta thalassemia [10] - Intellia Therapeutics is advancing its investigational therapies, including NTLA-2001 for ATTR amyloidosis and NTLA-2002 for hereditary angioedema, both in late-stage development [11]