January 31, 2025 expert reaction to NICE final draft guidance on exagamglogene autotemcel (exa-cel) for severe sickle cell disease . Scientists comment on final draft guidance fro ...

In sickle cell disease, a gene mutation causes the hemoglobin ... It does so by suppressing the gene BCL11A, which normally shuts down fetal hemoglobin production after birth.

BEAM-101 is an investigational genetically modified cell therapy for the treatment of severe sickle cell disease ... the transcriptional repressor BCL11A from binding to the promoter without ...

Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced the company will encore data from the BEACON Phase 1/2 ...

Advances in CRISPR technologies promise to accelerate the burgeoning pipeline of gene-editing therapies and broaden access to these disease-altering therapies.

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 ...