Updated data from 17 patients treated with BEAM-101 demonstrated robust and durable increases in fetal hemoglobin (HbF) and reductions in sickle hemoglobin (HbS), rapid neutrophil and platelet ...

The BEAM-101 edit is designed to inhibit the transcriptional repressor BCL11A from binding to the promoter without disrupting BCL11A expression, leading to increased production of non-sickling and ...

BEAM-101 is a gene therapy using base editing to increase fetal hemoglobin production and reduce sickle hemoglobin levels. What safety profile has been reported for BEAM-101?

In preclinical studies using clinically-translatable human cell models, CLY-124 increased fetal hemoglobin above 20%, with no evidence of cytotoxicity.

What's sickle cell disease, and how did gene therapy cure it? Sickle cell disease is a genetic blood disorder that impacts hemoglobin, the protein that carries oxygen in red blood cells.

RBC indices such as mean corpuscular volume, mean corpuscular hemoglobin concentration, and red cell distribution width are laboratory parameters that are frequently overlooked in clinical practice.

SCD can be diagnosed with a simple blood test. Confirmatory tests like hemoglobin electrophoresis and genetic testing provide definitive diagnosis and help plan treatment.