Health

    Beam Therapeutics shares rally as FDA clears experimental drug for rare genetic disorder

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    Shares of biotech firm Beam Therapeutics (NASDAQ:BEAM) climbed 5.6% to $23.43 in premarket trading on Friday after the company announced a significant regulatory milestone.

    Late Thursday, Beam revealed that the U.S. Food and Drug Administration (FDA) had cleared its marketing application for BEAM-302, an experimental drug designed to treat alpha-1 antitrypsin deficiency (AATD), a rare genetic disorder.

    AATD is characterized by a substantial risk of lung and liver disease, with no curative treatments currently approved.

    Beam Therapeutics highlighted the unmet need for patients suffering from this condition, positioning BEAM-302 as a potential breakthrough.

    The FDA’s clearance marks a key step forward for the drug, which now moves closer to potentially reaching the market.

    Analysts have responded positively to the news.

    BofA Global Research described early-stage clinical trial data for BEAM-302 as “potentially practice-changing,” underscoring its promise in addressing AATD.

    The brokerage also expressed optimism about the market potential for AATD treatments, estimating unadjusted peak sales could exceed $5 billion.

    Of the 17 brokerages covering Beam Therapeutics, 13 rate the stock as a “buy” or higher, while four assign a “hold” rating, reflecting broad confidence in the company’s trajectory.

    Despite the upbeat outlook, Beam’s stock has faced challenges over the past year, declining approximately 35% as of its last close.

     

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