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Beam Therapeutics Presents Preclinical Data Highlighting Utility of BEAM-302 to Correct an Alpha-1 Antitrypsin (AAT) Deficiency Disease-Causing Mutation
First Preclinical Data for BEAM-302 Demonstrate Increased Levels of Corrected AAT and Reduced Mutant PiZ AAT in Multiple In Vivo Rodent Disease Models
About this update from Beam Therapeutics Inc.
[{"type":"text","content":"First Preclinical Data for BEAM-302 Demonstrate Increased Levels of Corrected AAT and Reduced Mutant PiZ AAT in Multiple In Vivo Rodent Disease Models\nCAMBRIDGE, Mass., Sept. 07, 2023 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported new preclinical data demonstrating the ability of its in vivo drug candidate, BEAM-302, to directly correct the PiZ mutation, the primary disease-causing mutation associated with severe alpha-1 antitrypsin deficiency (AATD). The data are featured in an oral presentation titled “BEAM-302: Targeting AATD Liver and Lung Disease with Base Editing” at the Alpha-1 Antitrypsin Deficiency 2023 Meeting in Naples, Italy. “AATD, one of the most common genetic conditions, predominantly results in lung and liver disease, and we believe base editing is uniquely suited to address the underlying drivers of disease progression in both organs,” said Giuseppe Ciaramella, Ph.D., president of Beam. “In today’s presentation, we shared – for the first time – a full summary of preclinical in vivo data for BEAM-302, our lead candidate for the potential treatment of AATD. In two rodent models of AATD, one-time treatment at clinically relevant dose levels of BEAM-302 led to significant increases in circulating total corrected AAT and corresponding reductions in circulating mutant PiZ AAT. These data support the continued advancement of BEAM-302 as a potential treatment option for AATD-related lung and liver disease, and we remain focused on the planned submission of our regulatory application in the first quarter of next year.” AATD is caused by mutations in the SERPINA1 gene, with >95% of severe clinical cases homozygous for the PiZ mutation (known as the PiZZ genotype). BEAM-302 is a liver-targeting lipid-nanoparticle (LNP) formulation of base editing reagents designed to precisely correct the PiZ mutation, a single-letter genetic error. 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 lung and liver disease. Correction is expected...