Investing.com -- Shares of Beam Therapeutics Inc . (NASDAQ: BEAM ) climbed 3.7% today following the announcement that the U.S. Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for BEAM-302, aimed at treating alpha-1 antitrypsin deficiency (AATD). This genetic disorder, which can cause early onset emphysema and liver disease, currently lacks approved curative treatments.
Beam Therapeutics’ President, Giuseppe Ciaramella, Ph.D., expressed that the FDA’s clearance marks a significant milestone in advancing the development of BEAM-302 as a potential breakthrough treatment for AATD patients in the United States. Earlier in March 2025, the company revealed promising initial safety and efficacy data from the global Phase 1/2 study of BEAM-302, which demonstrated the first ever clinical genetic correction of a disease-causing mutation.
BEAM-302, using a liver-targeting lipid-nanoparticle (LNP) formulation, is designed to correct the PiZ mutation responsible for AATD. The ongoing Phase 1/2 clinical trial is assessing the safety, tolerability, pharmacodynamics, pharmacokinetics, and efficacy of the treatment. The trial is divided into two parts: Part A focuses on AATD patients with lung disease, while Part B will include patients with mild to moderate liver disease, with or without lung disease.
Beam Therapeutics plans to press on with the dose-escalation phase of Part A, including the enrollment and dosing of a fourth cohort, and anticipates presenting further data in the latter half of 2025. The company also expects to dose the first patient in Part B during the same period. BEAM-302 has already received clinical trial authorization in several countries, including the United Kingdom (TADAWUL: 4280 ), New Zealand, Australia, the Netherlands, and Ireland.
Supporting the positive market response, BofA Securities analyst Alec Stranahan upgraded Beam’s stock rating from Neutral to Buy and maintained a $42.00 price target. Stranahan commented, "We see this data as a key value unlocking event. Thus, BEAM’s platform is increasingly derisked, in our view, with an attractive valuation and limited near-term dilution risk following a recent share offering."
Investors appear encouraged by the FDA’s clearance and the optimistic outlook from analysts, reflected in the uptick of Beam Therapeutics’ stock price. As the company continues its clinical trials and prepares for further updates in the second half of 2025, the market will closely watch for the potential impact of BEAM-302 on the treatment landscape for AATD.
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