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Beam Therapeutics Enrolls First Patient in BEACON Clinical Trial of BEAM-101 Base Editing Therapy Candidate for the Treatment of Sickle Cell Disease
BEAM-101 BEACON Trial Represents the First Clinical Trial of a Base Editor in the United States CAMBRIDGE, Mass., Nov. 14, 2022 (GLOBE NEWSWIRE) -- Beam
About this update from Beam Therapeutics Inc.
[{"type":"text","content":"BEAM-101 BEACON Trial Represents the First Clinical Trial of a Base Editor in the United States\nCAMBRIDGE, Mass., Nov. 14, 2022 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the first patient has been enrolled in the company’s BEACON trial. BEACON is an open-label, single-arm, multicenter, Phase 1/2 clinical trial designed to evaluate the safety and efficacy of BEAM-101 in adult patients with severe sickle cell disease (SCD). BEAM-101 is a patient-specific, autologous hematopoietic stem cell (HSC) investigational therapy, which incorporates base edits that are designed to mimic single nucleotide polymorphisms seen in individuals with hereditary persistence of fetal hemoglobin. BEAM-101 aims to potentially alleviate the effects of mutations causing SCD by leading to increases in fetal hemoglobin (HbF), which inhibits hemoglobin S (HbS) polymerization. Using base editing, a next-generation form of CRISPR, BEAM-101 in preclinical studies featured high levels of HSC editing (over 90% of alleles edited), high and consistent levels of upregulation of HbF (over 60% of total hemoglobin), and significant reductions in the disease-causing protein HbS (less than 40% of total hemoglobin) – levels that are similar to sickle cell trait carriers, who do not have SCD. Unlike nuclease editors, the BEAM-101 base editor is designed to avoid double stranded breaks during editing, which can result in unwanted chromosomal abnormalities and genotoxic stress. Following enrollment in the BEACON trial, each patient will undergo a transfusion and mobilization process for HSC retrieval. The cells are then edited, creating an autologous BEAM-101 investigational drug product. Following the drug product manufacturing, patients in the trial will receive pre-treatment conditioning using a standard-of-care chemotherapy regimen, after which the edited cells are transplanted back into the patient. The trial is expected to include an initial “sentinel” cohort of three patients, treated one at a time to confirm successful engraftment, followed by dosing in up to a total of 45 patients. “The enrollment of the first patient in our BEACON trial is a significant step forward for Beam and for the field of base editing,” said John Evans, chief executive ...