Beam Therapeutics (BEAM) shares increased by 5% on Thursday following the FDA’s approval of its investigational new drug application for BEAM-304, a gene-editing therapy targeting phenylketonuria (PKU). This milestone is significant for the company’s liver-targeted genetic disease portfolio.
PKU affects approximately 20,000 individuals in the U.S. and is characterized by the inability to metabolize phenylalanine, leading to toxic accumulation and serious neurological issues without treatment. Currently, no curative therapies are available for this condition. Beam plans to initiate a Phase I/II clinical study to assess the safety and efficacy of BEAM-304, which has demonstrated promise in preclinical models.
Year to date, Beam’s stock has risen 23.2% compared to a 1% decline in the broader biotech industry.
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