Investing.com -- Beam Therapeutics Inc (NASDAQ:BEAM) stock gained 1.7% in premarket trading Monday after the biotechnology company presented updated data from its BEACON Phase 1/2 clinical trial of ristoglogene autogetemcel (risto-cel) for sickle cell disease (SCD).
The data, presented at the 67th American Society of Hematology Annual Meeting, showed mean hemoglobin F induction of over 60% and hemoglobin S reduction to resolution of anemia that remained durable for up to 20 months in 31 adult and adolescent SCD patients. Patients required a median of just one cell collection cycle and experienced rapid neutrophil and platelet engraftment.
The Cambridge, Massachusetts-based company reported that no patients experienced any investigator-reported severe vaso-occlusive crises (VOCs) post-engraftment. Mean peripheral blood editing was 67.4% at Month 6 and increased to 72.8% by Month 12.
"Risto-cel continues to demonstrate how base editing can fulfill its promise as a next-generation precision genetic medicine that could significantly improve the treatment process and outcomes for patients with SCD," said John Evans, chief executive officer of Beam Therapeutics.
The safety profile remained consistent with busulfan conditioning, autologous hematopoietic stem cell transplantation, and underlying SCD. The most common treatment-emergent adverse events included stomatitis, febrile neutropenia, and decreased appetite.
Beam also presented data showing that a tiered fixed-dose plerixafor mobilization regimen led to higher CD34+ cell yields, faster collection, and fewer collection cycles compared to traditional weight-based dosing.
The company indicated it’s on track to efficiently dose the remaining patients enrolled in the study and advance toward a regulatory filing.
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