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Shares of Beam Therapeutics Inc (BEAM) jumped more than 10% in pre-market trading on Wednesday, after the biotech firm reported promising early-stage data for its gene-editing therapy (BEAM-302) that showed the candidate with alpha-1 antitrypsin deficiency (AAT) could sustain protective protein levels for up to one year. This reinforces the case for durable one-time genetic therapies for rare liver diseases.
According to the report, a single 60 mg dose raised average AAT levels to 16.1 microns (µM), with all patients staying above the protective threshold for up to 12 months. The treatment converted 94% of AAT in the blood into the healthy form and reduced the harmful mutant protein by 84%.
Patients also retained the ability to boost AAT levels during infections, the company added. The treatment was well tolerated across doses up to 75 mg, with no serious safety concerns reported.
Based on the results, Beam selected 60 mg as the optimal dose and announced plans to begin a global trial in the second half of 2026.
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