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Beam Therapeutics Announces First Patient Dosed in the Phase 1/2 Study of BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD)
CAMBRIDGE, Mass., June 26, 2024 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines
About this update from Beam Therapeutics Inc.
[{"type":"text","content":"CAMBRIDGE, Mass., June 26, 2024 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced the first patient was treated with BEAM-302, an investigational in vivo base editing medicine designed to precisely correct the underlying cause of severe alpha-1 antitrypsin deficiency (AATD), that is currently being evaluated in a Phase 1/2 clinical trial. “Treating the first patient with BEAM-302 is an important milestone for Beam and the AATD patient community, who are in need of novel and more effective treatment options that can address the full spectrum of disease manifestations,” said John Evans, chief executive officer at Beam. “Using the power and precision of base editing technology, BEAM-302 is the first clinical program in the gene editing field designed to directly correct a genetic disease-causal mutation back to a normal functional gene sequence with a one-time in vivo therapy. We believe BEAM-302 has the potential to be a best-in-class treatment of both AATD-associated lung and liver disease through the correction of the PiZ allele, the most common gene variant associated with severe AATD. Advancing this Phase 1/2 study is a top priority for Beam, and we look forward to continued site activation, patient enrollment and dosing.” BEAM-302 is being evaluated in an open-label, dose-escalation Phase 1/2 clinical trial that will investigate the safety, pharmacodynamics, pharmacokinetics and efficacy of BEAM-302. The study design includes a dose exploration portion followed by a dose expansion portion to identify the optimal dose to take forward in a pivotal study. About BEAM-302BEAM-302 is a liver-targeting lipid-nanoparticle (LNP) formulation of base editing reagents designed to correct the PiZ mutation. Patients homozygous for this mutation (PiZZ) represent the majority of patients living with severe AATD disease. A one-time A-to-G correction of the PiZ mutation with Beam’s adenine base editor has the potential to simultaneously reduce the aggregation of mutant, misfolded AAT protein that causes toxicity to the liver and increase circulating levels of corrected and functional AAT protein, thus addressing the underlying pathophysiology of both the liver and lung disease. In addition, the reduction in circulating PiZ aggregates (...