Beam Therapeutics Inc. (NASDAQ:BEAM) on Saturday presented additional data from the Phase 1/2 clinical trial of BEAM-302 in patients with alpha-1 antitrypsin deficiency (AATD) at the 2025 Alpha-1 Foundation 7th Global Research Conference and 10th Patient Congress.

AATD is an inherited genetic disorder that can cause early onset emphysema and liver disease.

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Initial safety and efficacy data from the Phase 1/2 trial of BEAM-302 were previously reported in March 2025, establishing clinical proof of concept as a potential treatment for AATD and in vivo base editing.

Preliminary results from the first three single-ascending dose cohorts demonstrated that BEAM-302 was well tolerated.

Single doses of BEAM-302 led to durable dose-dependent correction of the disease-causing mutation and total AAT protein levels above the therapeutic threshold in the 60 mg dose cohort.

These previously reported data were included in the presentation, alongside updated biomarker data from the 60 mg cohort showing levels of corrected protein (M-AAT) and the mutant form of alpha-1 antitrypsin protein (Z-AAT) out to Day 28 for all three patients.

At Day 28, the proportion of corrected M-AAT reached a mean of 91% of total AAT in circulation, surpassing levels observed in patients with the MZ genotype where circulating M-AAT is typically ~80%.

In addition, treatment with BEAM-302 led to a mean decrease of 79% of circulating mutant Z-AAT from baseline as of Day 28.

Beam plans to continue the dose-escalation portion of Part A of the ongoing Phase 1/2 trial, including enrolling and dosing a fourth dose cohort of 75 mg, and expects to report further data at a medical conference in the second half of 2025.

In addition, the company plans to dose the first patient in Part B, including AATD patients with mild to moderate liver disease, in the second half of 2025.

The U.S. Food and Drug Administration recently cleared Beam Therapeutics’ investigational drug application for BEAM-302, enabling the company to activate sites in the U.S. for its ongoing Phase 1/2 trial.

Price Action: BEAM stock is down 6.75% at $14.24 at the last check on Monday.

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This article Beam Therapeutics Presents Biomarker Data For BEAM-302 Gene Therapy For Inherited Disease originally appeared on Benzinga.com

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