Beam Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to BEAM-302, its investigational therapy for alpha-1 antitrypsin deficiency (AATD)—a rare and inherited condition that affects both the lungs and liver.
AATD is caused by a genetic mutation that leads to deficient or malfunctioning alpha-1 antitrypsin (AAT) protein. This deficiency contributes to early-onset emphysema and liver disease due to the accumulation of misfolded proteins. Approximately 100,000 individuals in the U.S. have the PiZZ genotype (two copies of the Z allele), though only 10% are diagnosed.
Current treatments, such as intravenous AAT protein replacement, offer limited benefit and do not prevent long-term organ damage.
BEAM-302 is a next-generation genetic therapy designed to correct the PiZ mutation, the most common and severe form of AATD. Using an adenine base editor, BEAM-302 aims to convert a single A nucleotide to a G at the gene’s native locus—correcting the disease-causing mutation at its source.
By addressing the genetic root of AATD, BEAM-302 may provide a durable therapeutic effect, surpassing the temporary benefits of protein replacement therapy.
In addition to orphan drug designation, BEAM-302 has achieved two key regulatory designations in 2025:
These designations support the drug’s clinical development and highlight its potential to address a significant unmet medical need.
The FDA’s orphan drug designation provides several incentives for the development of therapies targeting rare diseases, including:
BEAM-302 is currently advancing through preclinical and early clinical development phases. As a precision genetic therapy, it represents a potentially transformative approach for patients with AATD—offering hope for a long-lasting and meaningful improvement in both liver and lung function.
Beam Therapeutics continues to pioneer base editing approaches across a range of severe genetic diseases, with BEAM-302 now standing as one of its most advanced and promising programs.