MedPage Today22h
Gene Therapy May Be a Game Changer for Transfusion-Dependent Thalassemia
The FDA approved betibeglogene autotemcel (beti-cel; Zynteglo) for adult and pediatric patients with transfusion-dependent ...
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Could a pill replace gene therapy for sickle cell disease? Research uncovers potential path to small-molecule drug
Casgevy works by enabling people to produce a fetal form of hemoglobin that doesn't cause sickling. It does so by suppressing the gene BCL11A, which normally shuts down fetal hemoglobin production ...
Nasdaq16d
Beam Therapeutics to Present Data from BEACON Phase 1/2 Trial of BEAM-101 for Sickle Cell Disease at 2025 Tandem Meetings
BEAM-101 demonstrated robust and durable increases in fetal hemoglobin and reductions in ... to inhibit the transcriptional repressor BCL11A from binding to the promoter without disrupting BCL11A ...
Nasdaq16d
MaxCyte: Building the Future of Cell and Gene Therapy Innovation
Casgevy enables CRISPR/Cas-9 to edit the BCL11A gene in stem cells to produce fetal hemoglobin. The edited genes are infused into a patient's bone marrow to start producing red blood cells with ...
Vertex Pharmaceuticals: Vertex Announces CASGEVY Reimbursement Agreement for the Treatment of Sickle Cell Disease in England
Agreement means eligible sickle cell disease (SCD) patients in England now have access to CASGEVY - - Agreement for CASGEVY in transfusion-dependent beta thalassemia (TDT) was previously reached ...
Morningstar16d
Beam Therapeutics to Encore Data from BEACON Phase 1/2 Clinical Trial of BEAM-101 in Sickle Cell Disease at 2025 Tandem Meetings of ASTCT and CIBMTR
Treatment with BEAM-101 demonstrated robust and durable increases in fetal hemoglobin (HbF ... to inhibit the transcriptional repressor BCL11A from binding to the promoter without disrupting ...