Beam Therapeutics Inc. (BEAM), on Monday, announced positive initial data from its Phase 1/2 trial of BEAM-302, a potential treatment for Alpha-1 Antitrypsin Deficiency or AATD, a genetic disorder that affects the lungs and liver.
The trial demonstrated the first-ever clinical genetic correction of the disease-causing PiZ mutation, showing durable, dose-dependent increases in functional Alpha-1 Antitrypsin or AAT and a reduction in the harmful mutant Z-AAT in circulation.
In the trial's first three dose cohorts (15 mg, 30 mg, and 60 mg), BEAM-302 was well-tolerated with no serious adverse events.
The 60 mg dose achieved a mean total AAT level of 12.4µM, exceeding the protective therapeutic threshold and reducing mutant Z-AAT by up to 78 percent.
This promising data suggests that BEAM-302 has the potential to be a one-time therapy addressing both the lung and liver disease aspects of AATD.
Beam Therapeutics plans to continue dose escalation and expects to present updated data at a medical conference in the second half of 2025.
BEAM closed Friday's (Mar. 07, 2025) trading at $28.48, up 4.51 percent. In pre market trading Monday, the stock is up by 5.34% at $30.00.
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