Beam Therapeutics’ BEAM-302 Granted FDA Orphan Drug Designation for Alpha-1 Antitrypsin Deficiency

CAMBRIDGE, Mass. and AMSTERDAM, May 29, 2025 – Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced its investigational therapy, BEAM-302, has received Orphan Drug Designation (ODD) from the U.S. FDA for Alpha-1 Antitrypsin Deficiency (AATD). BEAM-302 is a liver-targeting lipid-nanoparticle (LNP) formulation of a guide RNA and an mRNA encoding a base editor, designed to directly correct the disease-causing mutation in AATD patients, a disorder leading to severe lung and/or liver disease with high unmet need.

“This ODD is an important milestone, reinforcing the urgency and potential of BEAM-302 to directly correct the DNA mutation, the root cause of AATD,” said Giuseppe Ciaramella, Ph.D., president of Beam Therapeutics. “Following our recent RMAT designation, we are committed to advancing this potentially curative, one-time treatment.”

ODD provides benefits like tax credits and potential market exclusivity for rare disease treatments.

Positive initial safety and efficacy data from the ongoing Phase 1/2 trial of BEAM-302, reported in March, demonstrated the first clinical proof of concept for in vivo base editing. Preliminary results showed BEAM-302 was well tolerated, with single doses of the LNP leading to durable, dose-dependent correction of the mutation and AAT protein levels above the therapeutic threshold in the 60 mg cohort.

Beam has begun dosing in the 75 mg cohort and expects updated data in H2 2025. They also plan to dose the first patient in Part B (including AATD patients with mild to moderate liver disease) in H2 2025. BEAM-302 previously received U.S. IND clearance in March 2025 and RMAT designation in May 2025.

About BEAM-302:

BEAM-302 is a liver-targeting LNP formulation of base editing reagents designed to correct the PiZ mutation, the most common severe form of AATD. This one-time A-to-G correction aims to reduce toxic protein aggregation, generate functional AAT, and increase total AAT in circulation, addressing both liver and lung disease pathophysiology with durable, physiological AAT levels.

About Alpha-1 Antitrypsin Deficiency (AATD):

AATD is an inherited genetic disorder causing early-onset emphysema and liver disease due to misfolded AAT protein. The PiZZ genotype (affecting ~100,000 in the U.S., many undiagnosed) leads to very low circulating AAT and organ damage. There are currently no curative treatments.