BioPharma Dive March 10, 2025
Initial results from a small study suggest Beam’s medicine can directly correct the disease-causing mutation behind alpha-1 antitrypsin deficiency.
Preliminary results from a small clinical trial suggest that a cutting-edge genetic medicine developed by Beam Therapeutics can repair the damaged DNA that gives rise to a rare liver and lung disorder.
The data, from the first nine patients treated in the trial, are an important proof point for Beam, which specializes in a form of CRISPR gene editing that can precisely rewrite misspelled DNA sequences by changing individual nucleotides, or “letters.” Although the biotechnology company has previously released data for other experimental editing medicines, Monday’s findings are the first from a therapy designed to directly correct a disease-causing genetic mutation.
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