Beam Therapeutics Announces U.S. FDA Orphan Drug Designation Granted to BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD)

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[{"type":"text","content":"CAMBRIDGE, Mass., May 29, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the United States (U.S.) Food and Drug Administration (FDA) has granted orphan drug designation to BEAM-302, a liver-targeting lipid-nanoparticle (LNP) formulation of a guide RNA and an mRNA encoding a base editor designed to correct the disease-causing mutation in patients with alpha-1 antitrypsin deficiency (AATD). AATD is an inherited genetic disorder that affects the lungs and/or liver, leading to early onset emphysema and liver disease, and for which there is significant unmet need for effective therapies that can treat the entire spectrum of disease. “Receiving orphan drug designation for BEAM-302 is an important milestone in our efforts to bring a transformative therapy to people living with AATD, many of whom currently lack effective long-term treatment options,” said Giuseppe Ciaramella, Ph.D., president of Beam Therapeutics. “This recognition by the FDA, following the receipt of RMAT designation from the FDA just weeks ago, highlights the urgency of addressing this serious genetic disease and the potential of BEAM-302 to directly correct the DNA mutation, the underlying root cause of this illness. We are encouraged by the FDA’s continued support of this program and are committed to its advancement with the goal of delivering a one-time, potentially curative treatment to patients as quickly and safely as possible.” The FDA’s orphan drug designation is designed to support the development and evaluation of treatments for rare diseases affecting fewer than 200,000 people in the U.S. The designation comes with potential benefits for the sponsor company, including tax credits for qualified clinical trials, exemption from user fees, and a potential seven years of market exclusivity after approval. Positive initial safety and efficacy data from the ongoing Phase 1/2 trial of BEAM-302, previously reported in March, established clinical proof of concept as a potential treatment for AATD and in vivo base editing. Preliminary results from the first three single-ascending dose cohorts in Part A of the study demonstrated that BEAM-302 was well tolerated, with single doses of BEAM-302 leading to durable, dose-dependent correction of the dise...

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