Hosted on MSN1mon
Could a pill replace gene therapy for sickle cell disease? Research uncovers potential path to small-molecule drug
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.
manilatimes23d
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) and reductions in sickle ... stem cell transplant procedure. The BEAM-101 edit is designed to inhibit the ...